                         SEQUENCE LISTING

<110>  Ospedale San Raffaele S.r.l.
 
<120>  GENE THERAPY

<130>  P117507PCT

<140>  PCT/EP2020/060627
<141>  2020-04-15

<150>  GB 1905301.6
<151>  2019-04-15

<160>  30    

<170>  PatentIn version 3.5

<210>  1
<211>  498
<212>  PRT
<213>  Homo sapiens

<400>  1

Met Ala Ala Ala Ala Ala Ala Ala Pro Ser Gly Gly Gly Gly Gly Gly 
1               5                   10                  15      


Glu Glu Glu Arg Leu Glu Glu Lys Ser Glu Asp Gln Asp Leu Gln Gly 
            20                  25                  30          


Leu Lys Asp Lys Pro Leu Lys Phe Lys Lys Val Lys Lys Asp Lys Lys 
        35                  40                  45              


Glu Glu Lys Glu Gly Lys His Glu Pro Val Gln Pro Ser Ala His His 
    50                  55                  60                  


Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu Thr Ser Glu Gly Ser 
65                  70                  75                  80  


Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala Ser Pro Lys Gln Arg 
                85                  90                  95      


Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr Asp Asp Pro Thr Leu 
            100                 105                 110         


Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser 
        115                 120                 125             


Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro Gln Gly Lys Ala Phe 
    130                 135                 140                 


Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu Lys Val Gly Asp Thr 
145                 150                 155                 160 


Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val Thr Gly Arg Gly Ser 
                165                 170                 175     


Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys Pro Lys Ser Pro Lys 
            180                 185                 190         


Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro Lys Gly Ser Gly Thr 
        195                 200                 205             


Thr Arg Pro Lys Ala Ala Thr Ser Glu Gly Val Gln Val Lys Arg Val 
    210                 215                 220                 


Leu Glu Lys Ser Pro Gly Lys Leu Leu Val Lys Met Pro Phe Gln Thr 
225                 230                 235                 240 


Ser Pro Gly Gly Lys Ala Glu Gly Gly Gly Ala Thr Thr Ser Thr Gln 
                245                 250                 255     


Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg Lys Ala Glu Ala Asp 
            260                 265                 270         


Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys Pro Gly Ser Val Val 
        275                 280                 285             


Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala Val Lys Glu Ser Ser 
    290                 295                 300                 


Ile Arg Ser Val Gln Glu Thr Val Leu Pro Ile Lys Lys Arg Lys Thr 
305                 310                 315                 320 


Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val Val Lys Pro Leu Leu 
                325                 330                 335     


Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly Leu Lys Thr Cys Lys 
            340                 345                 350         


Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro Lys Gly Arg Ser Ser 
        355                 360                 365             


Ser Ala Ser Ser Pro Pro Lys Lys Glu His His His His His His His 
    370                 375                 380                 


Ser Glu Ser Pro Lys Ala Pro Val Pro Leu Leu Pro Pro Leu Pro Pro 
385                 390                 395                 400 


Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Thr Ser Pro Pro Glu 
                405                 410                 415     


Pro Gln Asp Leu Ser Ser Ser Val Cys Lys Glu Glu Lys Met Pro Arg 
            420                 425                 430         


Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu Pro Ala Lys Thr 
        435                 440                 445             


Gln Pro Ala Val Ala Thr Ala Ala Thr Ala Ala Glu Lys Tyr Lys His 
    450                 455                 460                 


Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser Ser Met Pro Arg 
465                 470                 475                 480 


Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro Val Thr Glu Arg 
                485                 490                 495     


Val Ser 
        


<210>  2
<211>  501
<212>  PRT
<213>  Mus musculus

<400>  2

Met Ala Ala Ala Ala Ala Thr Ala Ala Ala Ala Ala Ala Pro Ser Gly 
1               5                   10                  15      


Gly Gly Gly Gly Gly Glu Glu Glu Arg Leu Glu Glu Lys Ser Glu Asp 
            20                  25                  30          


Gln Asp Leu Gln Gly Leu Arg Asp Lys Pro Leu Lys Phe Lys Lys Ala 
        35                  40                  45              


Lys Lys Asp Lys Lys Glu Asp Lys Glu Gly Lys His Glu Pro Leu Gln 
    50                  55                  60                  


Pro Ser Ala His His Ser Ala Glu Pro Ala Glu Ala Gly Lys Ala Glu 
65                  70                  75                  80  


Thr Ser Glu Ser Ser Gly Ser Ala Pro Ala Val Pro Glu Ala Ser Ala 
                85                  90                  95      


Ser Pro Lys Gln Arg Arg Ser Ile Ile Arg Asp Arg Gly Pro Met Tyr 
            100                 105                 110         


Asp Asp Pro Thr Leu Pro Glu Gly Trp Thr Arg Lys Leu Lys Gln Arg 
        115                 120                 125             


Lys Ser Gly Arg Ser Ala Gly Lys Tyr Asp Val Tyr Leu Ile Asn Pro 
    130                 135                 140                 


Gln Gly Lys Ala Phe Arg Ser Lys Val Glu Leu Ile Ala Tyr Phe Glu 
145                 150                 155                 160 


Lys Val Gly Asp Thr Ser Leu Asp Pro Asn Asp Phe Asp Phe Thr Val 
                165                 170                 175     


Thr Gly Arg Gly Ser Pro Ser Arg Arg Glu Gln Lys Pro Pro Lys Lys 
            180                 185                 190         


Pro Lys Ser Pro Lys Ala Pro Gly Thr Gly Arg Gly Arg Gly Arg Pro 
        195                 200                 205             


Lys Gly Ser Gly Thr Gly Arg Pro Lys Ala Ala Ala Ser Glu Gly Val 
    210                 215                 220                 


Gln Val Lys Arg Val Leu Glu Lys Ser Pro Gly Lys Leu Val Val Lys 
225                 230                 235                 240 


Met Pro Phe Gln Ala Ser Pro Gly Gly Lys Gly Glu Gly Gly Gly Ala 
                245                 250                 255     


Thr Thr Ser Ala Gln Val Met Val Ile Lys Arg Pro Gly Arg Lys Arg 
            260                 265                 270         


Lys Ala Glu Ala Asp Pro Gln Ala Ile Pro Lys Lys Arg Gly Arg Lys 
        275                 280                 285             


Pro Gly Ser Val Val Ala Ala Ala Ala Ala Glu Ala Lys Lys Lys Ala 
    290                 295                 300                 


Val Lys Glu Ser Ser Ile Arg Ser Val His Glu Thr Val Leu Pro Ile 
305                 310                 315                 320 


Lys Lys Arg Lys Thr Arg Glu Thr Val Ser Ile Glu Val Lys Glu Val 
                325                 330                 335     


Val Lys Pro Leu Leu Val Ser Thr Leu Gly Glu Lys Ser Gly Lys Gly 
            340                 345                 350         


Leu Lys Thr Cys Lys Ser Pro Gly Arg Lys Ser Lys Glu Ser Ser Pro 
        355                 360                 365             


Lys Gly Arg Ser Ser Ser Ala Ser Ser Pro Pro Lys Lys Glu His His 
    370                 375                 380                 


His His His His His Ser Glu Ser Thr Lys Ala Pro Met Pro Leu Leu 
385                 390                 395                 400 


Pro Ser Pro Pro Pro Pro Glu Pro Glu Ser Ser Glu Asp Pro Ile Ser 
                405                 410                 415     


Pro Pro Glu Pro Gln Asp Leu Ser Ser Ser Ile Cys Lys Glu Glu Lys 
            420                 425                 430         


Met Pro Arg Gly Gly Ser Leu Glu Ser Asp Gly Cys Pro Lys Glu Pro 
        435                 440                 445             


Ala Lys Thr Gln Pro Met Val Ala Thr Thr Thr Thr Val Ala Glu Lys 
    450                 455                 460                 


Tyr Lys His Arg Gly Glu Gly Glu Arg Lys Asp Ile Val Ser Ser Ser 
465                 470                 475                 480 


Met Pro Arg Pro Asn Arg Glu Glu Pro Val Asp Ser Arg Thr Pro Val 
                485                 490                 495     


Thr Glu Arg Val Ser 
            500     


<210>  3
<211>  1497
<212>  DNA
<213>  Homo sapiens

<400>  3
atggccgccg ccgccgccgc cgcgccgagc ggaggaggag gaggaggcga ggaggagaga       60

ctggaagaaa agtcagaaga ccaggacctc cagggcctca aggacaaacc cctcaagttt      120

aaaaaggtga agaaagataa gaaagaagag aaagagggca agcatgagcc cgtgcagcca      180

tcagcccacc actctgctga gcccgcagag gcaggcaaag cagagacatc agaagggtca      240

ggctccgccc cggctgtgcc ggaagcttct gcctccccca aacagcggcg ctccatcatc      300

cgtgaccggg gacccatgta tgatgacccc accctgcctg aaggctggac acggaagctt      360

aagcaaagga aatctggccg ctctgctggg aagtatgatg tgtatttgat caatccccag      420

ggaaaagcct ttcgctctaa agtggagttg attgcgtact tcgaaaaggt aggcgacaca      480

tccctggacc ctaatgattt tgacttcacg gtaactggga gagggagccc ctcccggcga      540

gagcagaaac cacctaagaa gcccaaatct cccaaagctc caggaactgg cagaggccgg      600

ggacgcccca aagggagcgg caccacgaga cccaaggcgg ccacgtcaga gggtgtgcag      660

gtgaaaaggg tcctggagaa aagtcctggg aagctccttg tcaagatgcc ttttcaaact      720

tcgccagggg gcaaggctga ggggggtggg gccaccacat ccacccaggt catggtgatc      780

aaacgccccg gcaggaagcg aaaagctgag gccgaccctc aggccattcc caagaaacgg      840

ggccgaaagc cggggagtgt ggtggcagcc gctgccgccg aggccaaaaa gaaagccgtg      900

aaggagtctt ctatccgatc tgtgcaggag accgtactcc ccatcaagaa gcgcaagacc      960

cgggagacgg tcagcatcga ggtcaaggaa gtggtgaagc ccctgctggt gtccaccctc     1020

ggtgagaaga gcgggaaagg actgaagacc tgtaagagcc ctgggcggaa aagcaaggag     1080

agcagcccca aggggcgcag cagcagcgcc tcctcacccc ccaagaagga gcaccaccac     1140

catcaccacc actcagagtc cccaaaggcc cccgtgccac tgctcccacc cctgccccca     1200

cctccacctg agcccgagag ctccgaggac cccaccagcc cccctgagcc ccaggacttg     1260

agcagcagcg tctgcaaaga ggagaagatg cccagaggag gctcactgga gagcgacggc     1320

tgccccaagg agccagctaa gactcagccc gcggttgcca ccgccgccac ggccgcagaa     1380

aagtacaaac accgagggga gggagagcgc aaagacattg tttcatcctc catgccaagg     1440

ccaaacagag aggagcctgt ggacagccgg acgcccgtga ccgagagagt tagctga        1497


<210>  4
<211>  1506
<212>  DNA
<213>  Mus musculus

<400>  4
atggccgccg ctgccgccac cgccgccgcc gccgccgcgc cgagcggagg aggaggagga       60

ggcgaggagg agagactgga ggaaaagtca gaagaccagg atctccaggg cctcagagac      120

aagccactga agtttaagaa ggcgaagaaa gacaagaagg aggacaaaga aggcaagcat      180

gagccactac aaccttcagc ccaccattct gcagagccag cagaggcagg caaagcagaa      240

acatcagaaa gctcaggctc tgccccagca gtgccagaag cctcggcttc ccccaaacag      300

cggcgctcca ttatccgtga ccggggacct atgtatgatg accccacctt gcctgaaggt      360

tggacacgaa agcttaaaca aaggaagtct ggccgatctg ctggaaagta tgatgtatat      420

ttgatcaatc cccagggaaa agcttttcgc tctaaagtag aattgattgc atactttgaa      480

aaggtgggag acacctcctt ggaccctaat gattttgact tcacggtaac tgggagaggg      540

agcccctcca ggagagagca gaaaccacct aagaagccca aatctcccaa agctccagga      600

actggcaggg gtcggggacg ccccaaaggg agcggcactg ggagaccaaa ggcagcagca      660

tcagaaggtg ttcaggtgaa aagggtcctg gagaagagcc ctgggaaact tgttgtcaag      720

atgcctttcc aagcatcgcc tgggggtaag ggtgagggag gtggggctac cacatctgcc      780

caggtcatgg tgatcaaacg ccctggcaga aagcgaaaag ctgaagctga cccccaggcc      840

attcctaaga aacggggtag aaagcctggg agtgtggtgg cagctgctgc agctgaggcc      900

aaaaagaaag ccgtgaagga gtcttccata cggtctgtgc atgagactgt gctccccatc      960

aagaagcgca agacccggga gacggtcagc atcgaggtca aggaagtggt gaagcccctg     1020

ctggtgtcca cccttggtga gaaaagcggg aagggactga agacctgcaa gagccctggg     1080

cgtaaaagca aggagagcag ccccaagggg cgcagcagca gtgcctcctc cccacctaag     1140

aaggagcacc atcatcacca ccatcactca gagtccacaa aggcccccat gccactgctc     1200

ccatccccac ccccacctga gcctgagagc tctgaggacc ccatcagccc ccctgagcct     1260

caggacttga gcagcagcat ctgcaaagaa gagaagatgc cccgaggagg ctcactggaa     1320

agcgatggct gccccaagga gccagctaag actcagccta tggtcgccac cactaccaca     1380

gttgcagaaa agtacaaaca ccgaggggag ggagagcgca aagacattgt ttcatcttcc     1440

atgccaaggc caaacagaga ggagcctgtg gacagccgga cgcccgtgac cgagagagtt     1500

agctga                                                                1506


<210>  5
<211>  278
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  chicken beta-actin (CBA) promoter

<400>  5
tcgaggtgag ccccacgttc tgcttcactc tccccatctc ccccccctcc ccacccccaa       60

ttttgtattt atttattttt taattatttt gtgcagcgat gggggcgggg gggggggggg      120

ggcgcgcgcc aggcggggcg gggcggggcg aggggcgggg cggggcgagg cggagaggtg      180

cggcggcagc caatcagagc ggcgcgctcc gaaagtttcc ttttatggcg aggcggcggc      240

ggcggcggcc ctataaaaag cgaagcgcgc ggcgggcg                              278


<210>  6
<211>  223
<212>  DNA
<213>  Mus musculus

<400>  6
ctttacatag agcggattgc aaagcaaacc aacaagaata aaggcagctg ttgtctcttc       60

tccttatggg tagggctctg acaaagcttc ccgattaact gaaataaaaa atattttttt      120

ttctttcagt aaacttagag tttcgtggct tcggggtggg agtagttgga gcattgggat      180

gtttttctta ccgacaagca cagtcaggtt gaagacctaa cca                        223


<210>  7
<211>  224
<212>  DNA
<213>  Homo sapiens

<400>  7
ctttacacgg agcggattgc aaagcaaacc aacaagaata aaggcagctg ttgtctcttc       60

tccttatggg tagggctctg acaaagcttc ccgattaact gaaataaaaa atattttttt      120

ttctttcagt aaacttagag tttcgtggct tcagggtggg agtagttgga gcattgggga      180

tgtttttctt accgacaagc acagtcaggt tgaagaccta acca                       224


<210>  8
<211>  171
<212>  DNA
<213>  Homo sapiens

<400>  8
ctttacacgg agcggattgc aaagcaaacc aacaagaata aaggcagctg ttgtctcttc       60

tccttatggg tagggctctg acaaagcttc ccgattaact gaaataaaaa atattttttt      120

ttctttcagt aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa a               171


<210>  9
<211>  490
<212>  DNA
<213>  Homo sapiens

<400>  9
tcgagagatc tacgggtggc atccctgtga cccctcccca gtgcctctcc tggccctgga       60

agttgccact ccagtgccca ccagccttgt cctaataaaa ttaagttgca tcattttgtc      120

tgactaggtg tccttctata atattatggg gtggaggggg gtggtatgga gcaaggggca      180

agttgggaag acaacctgta gggcctgcgg ggtctattgg gaaccaagct ggagtgcagt      240

ggcacaatct tggctcactg caatctccgc ctcctgggtt caagcgattc tcctgcctca      300

gcctcccgag ttgttgggat tccaggcatg catgaccagg ctcagctaat ttttgttttt      360

ttggtagaga cggggtttca ccatattggc caggctggtc tccaactcct aatctcaggt      420

gatctaccca ccttggcctc ccaaattgct gggattacag gcgtgaacca ctgctccctt      480

ccctgtcctt                                                             490


<210>  10
<211>  736
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  amino acid sequence of the (wild-type) AAV9 capsid

<400>  10

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro 
        115                 120                 125             


Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ala Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Ala Gln Ala Gln 
            580                 585                 590         


Thr Gly Trp Val Gln Asn Gln Gly Ile Leu Pro Gly Met Val Trp Gln 
        595                 600                 605             


Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His 
    610                 615                 620                 


Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Met 
625                 630                 635                 640 


Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala 
                645                 650                 655     


Asp Pro Pro Thr Ala Phe Asn Lys Asp Lys Leu Asn Ser Phe Ile Thr 
            660                 665                 670         


Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln 
        675                 680                 685             


Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Ser Asn 
    690                 695                 700                 


Tyr Tyr Lys Ser Asn Asn Val Glu Phe Ala Val Asn Thr Glu Gly Val 
705                 710                 715                 720 


Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
                725                 730                 735     


<210>  11
<211>  743
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  amino acid sequence of the AAV-PHP.B capsid VP1 protein

<400>  11

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro 
        115                 120                 125             


Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ala Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Arg Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Thr Leu Ala Val 
            580                 585                 590         


Pro Phe Lys Ala Gln Ala Gln Thr Gly Trp Val Gln Asn Gln Gly Ile 
        595                 600                 605             


Leu Pro Gly Met Val Trp Gln Asp Arg Asp Val Tyr Leu Gln Gly Pro 
    610                 615                 620                 


Ile Trp Ala Lys Ile Pro His Thr Asp Gly Asn Phe His Pro Ser Pro 
625                 630                 635                 640 


Leu Met Gly Gly Phe Gly Met Lys His Pro Pro Pro Gln Ile Leu Ile 
                645                 650                 655     


Lys Asn Thr Pro Val Pro Ala Asp Pro Pro Thr Ala Phe Asn Lys Asp 
            660                 665                 670         


Lys Leu Asn Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val Ser Val 
        675                 680                 685             


Glu Ile Glu Trp Glu Leu Gln Lys Glu Asn Ser Lys Arg Trp Asn Pro 
    690                 695                 700                 


Glu Ile Gln Tyr Thr Ser Asn Tyr Tyr Lys Ser Asn Asn Val Glu Phe 
705                 710                 715                 720 


Ala Val Asn Thr Glu Gly Val Tyr Ser Glu Pro Arg Pro Ile Gly Thr 
                725                 730                 735     


Arg Tyr Leu Thr Arg Asn Leu 
            740             


<210>  12
<211>  743
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  amino acid sequence of the AAV-PHP.eB capsid VP1 protein

<400>  12

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro 
        115                 120                 125             


Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ala Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Asp Gly Thr Leu Ala Val 
            580                 585                 590         


Pro Phe Lys Ala Gln Ala Gln Thr Gly Trp Val Gln Asn Gln Gly Ile 
        595                 600                 605             


Leu Pro Gly Met Val Trp Gln Asp Arg Asp Val Tyr Leu Gln Gly Pro 
    610                 615                 620                 


Ile Trp Ala Lys Ile Pro His Thr Asp Gly Asn Phe His Pro Ser Pro 
625                 630                 635                 640 


Leu Met Gly Gly Phe Gly Met Lys His Pro Pro Pro Gln Ile Leu Ile 
                645                 650                 655     


Lys Asn Thr Pro Val Pro Ala Asp Pro Pro Thr Ala Phe Asn Lys Asp 
            660                 665                 670         


Lys Leu Asn Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val Ser Val 
        675                 680                 685             


Glu Ile Glu Trp Glu Leu Gln Lys Glu Asn Ser Lys Arg Trp Asn Pro 
    690                 695                 700                 


Glu Ile Gln Tyr Thr Ser Asn Tyr Tyr Lys Ser Asn Asn Val Glu Phe 
705                 710                 715                 720 


Ala Val Asn Thr Glu Gly Val Tyr Ser Glu Pro Arg Pro Ile Gly Thr 
                725                 730                 735     


Arg Tyr Leu Thr Arg Asn Leu 
            740             


<210>  13
<211>  3649
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CBA-iMECP2 cassette sequence

<400>  13
cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt       60

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      120

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      180

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      240

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      300

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      360

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      420

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      480

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      540

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcggggagtc gctgcgacgc      600

tgccttcgcc ccgtgccccg ctccgccgcc gcctcgcgcc gcccgccccg gctctgactg      660

accgcgttac tcccacaggt gagcgggcgg gacggccctt ctcctccggg ctgtaattag      720

cccgtttagt gaaccgtcag atcgcctgga gacgccatcc acgctgtttt gacctccata      780

gaagacaccg ggaccgatcc agcctccgcg gattcgaatc ccggccggga acggtgcatt      840

ggaacgcgga ttccccgtgc caagagtgac gtaagtaccg cctatagagt ctataggccc      900

acaaaaaatg ctttcttctt ttaatatact tttttgttta tcttatttct aatactttcc      960

ctaatctctt tctttcaggg caataatgat acaatgtatc atgcctcttt gcaccattct     1020

aaagaataac agtgataatt tctgggttaa ggcaatagca atatttctgc atataaatat     1080

ttctgcatat aaattgtaac tgatgtaaga ggtttcatat tgctaatagc agctacaatc     1140

cagctaccat tctgctttta ttttatggtt gggataaggc tggattattc tgagtccaag     1200

ctaggccctt ttgctaatca tgttcatacc tcttatcttc ctcccacagc tcctgggcaa     1260

cgtgctggtc tgtgtgctgg cccatcactt tggcaaagaa ttgggattcg aacaccggtc     1320

gacgaattcg ttaacggatc cgaacgccac catgggcaag cctatcccta accctctgct     1380

gggcctggac tccacaggca gcggcaccgg tatggccgcc gctgccgcca ccgccgccgc     1440

cgccgccgcg ccgagcggag gaggaggagg aggcgaggag gagagactgg aggaaaagtc     1500

agaagaccag gatctccagg gcctcagaga caagccactg aagtttaaga aggcgaagaa     1560

agacaagaag gaggacaaag aaggcaagca tgagccacta caaccttcag cccaccattc     1620

tgcagagcca gcagaggcag gcaaagcaga aacatcagaa agctcaggct ctgccccagc     1680

agtgccagaa gcctcggctt cccccaaaca gcggcgctcc attatccgtg accggggacc     1740

tatgtatgat gaccccacct tgcctgaagg ttggacacga aagcttaaac aaaggaagtc     1800

tggccgatct gctggaaagt atgatgtata tttgatcaat ccccagggaa aagcttttcg     1860

ctctaaagta gaattgattg catactttga aaaggtggga gacacctcct tggaccctaa     1920

tgattttgac ttcacggtaa ctgggagagg gagcccctcc aggagagagc agaaaccacc     1980

taagaagccc aaatctccca aagctccagg aactggcagg ggtcggggac gccccaaagg     2040

gagcggcact gggagaccaa aggcagcagc atcagaaggt gttcaggtga aaagggtcct     2100

ggagaagagc cctgggaaac ttgttgtcaa gatgcctttc caagcatcgc ctgggggtaa     2160

gggtgaggga ggtggggcta ccacatctgc ccaggtcatg gtgatcaaac gccctggcag     2220

aaagcgaaaa gctgaagctg acccccaggc cattcctaag aaacggggta gaaagcctgg     2280

gagtgtggtg gcagctgctg cagctgaggc caaaaagaaa gccgtgaagg agtcttccat     2340

acggtctgtg catgagactg tgctccccat caagaagcgc aagacccggg agacggtcag     2400

catcgaggtc aaggaagtgg tgaagcccct gctggtgtcc acccttggtg agaaaagcgg     2460

gaagggactg aagacctgca agagccctgg gcgtaaaagc aaggagagca gccccaaggg     2520

gcgcagcagc agtgcctcct ccccacctaa gaaggagcac catcatcacc accatcactc     2580

agagtccaca aaggccccca tgccactgct cccatcccca cccccacctg agcctgagag     2640

ctctgaggac cccatcagcc cccctgagcc tcaggacttg agcagcagca tctgcaaaga     2700

agagaagatg ccccgaggag gctcactgga aagcgatggc tgccccaagg agccagctaa     2760

gactcagcct atggtcgcca ccactaccac agttgcagaa aagtacaaac accgagggga     2820

gggagagcgc aaagacattg tttcatcttc catgccaagg ccaaacagag aggagcctgt     2880

ggacagccgg acgcccgtga ccgagagagt tagctgactt tacatagagc ggattgcaaa     2940

gcaaaccaac aagaataaag gcagctgttg tctcttctcc ttatgggtag ggctctgaca     3000

aagcttcccg attaactgaa ataaaaaata tttttttttc tttcagtaaa cttagagttt     3060

cgtggcttcg gggtgggagt agttggagca ttgggatgtt tttcttaccg acaagcacag     3120

tcaggttgaa gacctaacca gatatctcta gagatatcct cgagagatct acgggtggca     3180

tccctgtgac ccctccccag tgcctctcct ggccctggaa gttgccactc cagtgcccac     3240

cagccttgtc ctaataaaat taagttgcat cattttgtct gactaggtgt ccttctataa     3300

tattatgggg tggagggggg tggtatggag caaggggcaa gttgggaaga caacctgtag     3360

ggcctgcggg gtctattggg aaccaagctg gagtgcagtg gcacaatctt ggctcactgc     3420

aatctccgcc tcctgggttc aagcgattct cctgcctcag cctcccgagt tgttgggatt     3480

ccaggcatgc atgaccaggc tcagctaatt tttgtttttt tggtagagac ggggtttcac     3540

catattggcc aggctggtct ccaactccta atctcaggtg atctacccac cttggcctcc     3600

caaattgctg ggattacagg cgtgaaccac tgctcccttc cctgtcctt                 3649


<210>  14
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  sequence from VP1 capsid protein sequence

<400>  14

Thr Leu Ala Val Pro Phe Lys 
1               5           


<210>  15
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  sequence from VP1 capsid protein sequence

<400>  15

Lys Phe Pro Val Ala Leu Thr 
1               5           


<210>  16
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  sequence from VP1 capsid protein sequence

<400>  16

Asp Gly Thr Leu Ala Val Pro Phe Lys Ala Gln 
1               5                   10      


<210>  17
<211>  4045
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-CBA-V5-iMecp2-3`pUTR-hGHpA

<400>  17
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgca acgcgctagt tattaatagt aatcaattac      180

ggggtcatta gttcatagcc catatatgga gttccgcgtt acataactta cggtaaatgg      240

cccgcctggc tgaccgccca acgacccccg cccattgacg tcaataatga cgtatgttcc      300

catagtaacg ccaataggga ctttccattg acgtcaatgg gtggagtatt tacggtaaac      360

tgcccacttg gcagtacatc aagtgtatca tatgccaagt acgcccccta ttgacgtcaa      420

tgacggtaaa tggcccgcct ggcattatgc ccagtacatg accttatggg actttcctac      480

ttggcagtac atctacgtat tagtcatcgc tattaccatg gtcgaggtga gccccacgtt      540

ctgcttcact ctccccatct cccccccctc cccaccccca attttgtatt tatttatttt      600

ttaattattt tgtgcagcga tgggggcggg gggggggggg gggcgcgcgc caggcggggc      660

ggggcggggc gaggggcggg gcggggcgag gcggagaggt gcggcggcag ccaatcagag      720

cggcgcgctc cgaaagtttc cttttatggc gaggcggcgg cggcggcggc cctataaaaa      780

gcgaagcgcg cggcgggcgg ggagtcgctg cgacgctgcc ttcgccccgt gccccgctcc      840

gccgccgcct cgcgccgccc gccccggctc tgactgaccg cgttactccc acaggtgagc      900

gggcgggacg gcccttctcc tccgggctgt aattagcccg tttagtgaac cgtcagatcg      960

cctggagacg ccatccacgc tgttttgacc tccatagaag acaccgggac cgatccagcc     1020

tccgcggatt cgaatcccgg ccgggaacgg tgcattggaa cgcggattcc ccgtgccaag     1080

agtgacgtaa gtaccgccta tagagtctat aggcccacaa aaaatgcttt cttcttttaa     1140

tatacttttt tgtttatctt atttctaata ctttccctaa tctctttctt tcagggcaat     1200

aatgatacaa tgtatcatgc ctctttgcac cattctaaag aataacagtg ataatttctg     1260

ggttaaggca atagcaatat ttctgcatat aaatatttct gcatataaat tgtaactgat     1320

gtaagaggtt tcatattgct aatagcagct acaatccagc taccattctg cttttatttt     1380

atggttggga taaggctgga ttattctgag tccaagctag gcccttttgc taatcatgtt     1440

catacctctt atcttcctcc cacagctcct gggcaacgtg ctggtctgtg tgctggccca     1500

tcactttggc aaagaattgg gattcgaaca ccggtcgacg aattcgttaa cggatccgaa     1560

cgccaccatg ggcaagccta tccctaaccc tctgctgggc ctggactcca caggcagcgg     1620

caccggtatg gccgccgctg ccgccaccgc cgccgccgcc gccgcgccga gcggaggagg     1680

aggaggaggc gaggaggaga gactggagga aaagtcagaa gaccaggatc tccagggcct     1740

cagagacaag ccactgaagt ttaagaaggc gaagaaagac aagaaggagg acaaagaagg     1800

caagcatgag ccactacaac cttcagccca ccattctgca gagccagcag aggcaggcaa     1860

agcagaaaca tcagaaagct caggctctgc cccagcagtg ccagaagcct cggcttcccc     1920

caaacagcgg cgctccatta tccgtgaccg gggacctatg tatgatgacc ccaccttgcc     1980

tgaaggttgg acacgaaagc ttaaacaaag gaagtctggc cgatctgctg gaaagtatga     2040

tgtatatttg atcaatcccc agggaaaagc ttttcgctct aaagtagaat tgattgcata     2100

ctttgaaaag gtgggagaca cctccttgga ccctaatgat tttgacttca cggtaactgg     2160

gagagggagc ccctccagga gagagcagaa accacctaag aagcccaaat ctcccaaagc     2220

tccaggaact ggcaggggtc ggggacgccc caaagggagc ggcactggga gaccaaaggc     2280

agcagcatca gaaggtgttc aggtgaaaag ggtcctggag aagagccctg ggaaacttgt     2340

tgtcaagatg cctttccaag catcgcctgg gggtaagggt gagggaggtg gggctaccac     2400

atctgcccag gtcatggtga tcaaacgccc tggcagaaag cgaaaagctg aagctgaccc     2460

ccaggccatt cctaagaaac ggggtagaaa gcctgggagt gtggtggcag ctgctgcagc     2520

tgaggccaaa aagaaagccg tgaaggagtc ttccatacgg tctgtgcatg agactgtgct     2580

ccccatcaag aagcgcaaga cccgggagac ggtcagcatc gaggtcaagg aagtggtgaa     2640

gcccctgctg gtgtccaccc ttggtgagaa aagcgggaag ggactgaaga cctgcaagag     2700

ccctgggcgt aaaagcaagg agagcagccc caaggggcgc agcagcagtg cctcctcccc     2760

acctaagaag gagcaccatc atcaccacca tcactcagag tccacaaagg cccccatgcc     2820

actgctccca tccccacccc cacctgagcc tgagagctct gaggacccca tcagcccccc     2880

tgagcctcag gacttgagca gcagcatctg caaagaagag aagatgcccc gaggaggctc     2940

actggaaagc gatggctgcc ccaaggagcc agctaagact cagcctatgg tcgccaccac     3000

taccacagtt gcagaaaagt acaaacaccg aggggaggga gagcgcaaag acattgtttc     3060

atcttccatg ccaaggccaa acagagagga gcctgtggac agccggacgc ccgtgaccga     3120

gagagttagc tgactttaca tagagcggat tgcaaagcaa accaacaaga ataaaggcag     3180

ctgttgtctc ttctccttat gggtagggct ctgacaaagc ttcccgatta actgaaataa     3240

aaaatatttt tttttctttc agtaaactta gagtttcgtg gcttcggggt gggagtagtt     3300

ggagcattgg gatgtttttc ttaccgacaa gcacagtcag gttgaagacc taaccagata     3360

tctctagaga tatcctcgag agatctacgg gtggcatccc tgtgacccct ccccagtgcc     3420

tctcctggcc ctggaagttg ccactccagt gcccaccagc cttgtcctaa taaaattaag     3480

ttgcatcatt ttgtctgact aggtgtcctt ctataatatt atggggtgga ggggggtggt     3540

atggagcaag gggcaagttg ggaagacaac ctgtagggcc tgcggggtct attgggaacc     3600

aagctggagt gcagtggcac aatcttggct cactgcaatc tccgcctcct gggttcaagc     3660

gattctcctg cctcagcctc ccgagttgtt gggattccag gcatgcatga ccaggctcag     3720

ctaatttttg tttttttggt agagacgggg tttcaccata ttggccaggc tggtctccaa     3780

ctcctaatct caggtgatct acccaccttg gcctcccaaa ttgctgggat tacaggcgtg     3840

aaccactgct cccttccctg tccttctgat tttgtaggta accacgtgcg gaccgagcgg     3900

ccgcaggaac ccctagtgat ggagttggcc actccctctc tgcgcgctcg ctcgctcact     3960

gaggccgggc gaccaaaggt cgcccgacgc ccgggctttg cccgggcggc ctcagtgagc     4020

gagcgagcgc gcagctgcct gcagg                                           4045


<210>  18
<211>  3608
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-Mecp2 promoter (1.4kb)-V5-iMecp2-3`pUTR-hGHpA

<400>  18
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggcctct agtatcgata ctcgagattt caactgctac      180

tgtcctggtt aaagccttca tcatctatct ttcttcaact gctgccagga cctctggacc      240

agccagttct tcattcttca ctggcaacat aggttttatg gtgacagcta gtgactcaaa      300

tatttatcaa gggcttctca tctcaaaata atctcctagt tcttttggtg gcctaggtct      360

ctctccagtc acactggcct ccttagtaag gcaggcatag tccttcctta gagtgtttaa      420

acttgcctag aatgttttcc ccaattaccc atattgggag acgacatgag ggcaaaagct      480

agagggtatc ataatagcac ttcttttgtc cttgccctat ctatttcaaa gtctttatct      540

ctgtgcaaaa ttttaagttc tactttcttg tatgtttagt atgactcttc cttaccagga      600

gtctagtttg tctccttgtt cagtactaaa acagtgccta gcaaataaat gaatagagag      660

gggagccaaa tttgaatcag aaagtctctt gttgcatagt gtttaaaaaa caaacaaaga      720

aagaaagtct cttgttgagc atttgtttag cacaaagagc attggatgct gactggtatc      780

agggtaaggc tgctttgaca atgctccctc tggcctcact cccttttata cgtacttcca      840

tcaaaccatc tgattcaaca atgacagacc gatctcttat gggcttggca cacaccatct      900

gcccattata aacgtctgca aagaccaagg tttgatatgt tgattttact gtcagcctta      960

agagtgcgac atctgctaat ttagtgtaat aatacaatca gtagaccctt taaaacaagt     1020

cccttggctt ggaacaacgc caggctcctc aacaggcaac tttgctactt ctacagaaaa     1080

tgataataaa gaaatgctgg tgaagtcaaa tgcttatcac aatggtgaac tactcagcag     1140

ggaggctcta ataggcgcca agagcctaga cttccttaag cgccagagtc cacaagggcc     1200

cagttaatcc tcaacattca aatgctgccc acaaaaccag cccctctgtg ccctagccgc     1260

ctcttttttc caagtgacag tagaactcca ccaatccgca gctgaatggg gtccgcctct     1320

tttccctgcc taaacagaca ggaactcctg ccaattgagg gcgtcaccgc taaggctccg     1380

ccccagcctg ggctccacaa ccaatgaagg gtaatctcga caaagagcaa ggggtggggc     1440

gcgggcgcgc aggtgcagca gcacacaggc tggtcgggag ggcggggcgc gacgtctgcc     1500

gtgcggggtc ccggcatcgg ttgcgcgcgc gctccctcct ctcggagaga gggctgtggt     1560

aaaacccgtc aatcgctagc ggatccgtta acgccaccat gggcaagcct atccctaacc     1620

ctctgctggg cctggactcc acaggcagcg gcaccggtat ggccgccgct gccgccaccg     1680

ccgccgccgc cgccgcgccg agcggaggag gaggaggagg cgaggaggag agactggagg     1740

aaaagtcaga agaccaggat ctccagggcc tcagagacaa gccactgaag tttaagaagg     1800

cgaagaaaga caagaaggag gacaaagaag gcaagcatga gccactacaa ccttcagccc     1860

accattctgc agagccagca gaggcaggca aagcagaaac atcagaaagc tcaggctctg     1920

ccccagcagt gccagaagcc tcggcttccc ccaaacagcg gcgctccatt atccgtgacc     1980

ggggacctat gtatgatgac cccaccttgc ctgaaggttg gacacgaaag cttaaacaaa     2040

ggaagtctgg ccgatctgct ggaaagtatg atgtatattt gatcaatccc cagggaaaag     2100

cttttcgctc taaagtagaa ttgattgcat actttgaaaa ggtgggagac acctccttgg     2160

accctaatga ttttgacttc acggtaactg ggagagggag cccctccagg agagagcaga     2220

aaccacctaa gaagcccaaa tctcccaaag ctccaggaac tggcaggggt cggggacgcc     2280

ccaaagggag cggcactggg agaccaaagg cagcagcatc agaaggtgtt caggtgaaaa     2340

gggtcctgga gaagagccct gggaaacttg ttgtcaagat gcctttccaa gcatcgcctg     2400

ggggtaaggg tgagggaggt ggggctacca catctgccca ggtcatggtg atcaaacgcc     2460

ctggcagaaa gcgaaaagct gaagctgacc cccaggccat tcctaagaaa cggggtagaa     2520

agcctgggag tgtggtggca gctgctgcag ctgaggccaa aaagaaagcc gtgaaggagt     2580

cttccatacg gtctgtgcat gagactgtgc tccccatcaa gaagcgcaag acccgggaga     2640

cggtcagcat cgaggtcaag gaagtggtga agcccctgct ggtgtccacc cttggtgaga     2700

aaagcgggaa gggactgaag acctgcaaga gccctgggcg taaaagcaag gagagcagcc     2760

ccaaggggcg cagcagcagt gcctcctccc cacctaagaa ggagcaccat catcaccacc     2820

atcactcaga gtccacaaag gcccccatgc cactgctccc atccccaccc ccacctgagc     2880

ctgagagctc tgaggacccc atcagccccc ctgagcctca ggacttgagc agcagcatct     2940

gcaaagaaga gaagatgccc cgaggaggct cactggaaag cgatggctgc cccaaggagc     3000

cagctaagac tcagcctatg gtcgccacca ctaccacagt tgcagaaaag tacaaacacc     3060

gaggggaggg agagcgcaaa gacattgttt catcttccat gccaaggcca aacagagagg     3120

agcctgtgga cagccggacg cccgtgaccg agagagttag ctgactttac atagagcgga     3180

ttgcaaagca aaccaacaag aataaaggca gctgttgtct cttctcctta tgggtagggc     3240

tctgacaaag cttcccgatt aactgaaata aaaaatattt ttttttcttt cagtaaactt     3300

agagtttcgt ggcttcgggg tgggagtagt tggagcattg ggatgttttt cttaccgaca     3360

agcacagtca ggttgaagac ctaaccagat atctctagag tcgacgaatt caataaaaga     3420

tctttatttt cattagatct gtgtgttggt tttttgtgtg cggccgcagg aacccctagt     3480

gatggagttg gccactccct ctctgcgcgc tcgctcgctc actgaggccg ggcgaccaaa     3540

ggtcgcccga cgcccgggct ttgcccgggc ggcctcagtg agcgagcgag cgcgcagctg     3600

cctgcagg                                                              3608


<210>  19
<211>  4033
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-CBA-V5-iMecp2-3`aUTR-hGHpA (M2c)

<400>  19
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgca acgcgctagt tattaatagt aatcaattac      180

ggggtcatta gttcatagcc catatatgga gttccgcgtt acataactta cggtaaatgg      240

cccgcctggc tgaccgccca acgacccccg cccattgacg tcaataatga cgtatgttcc      300

catagtaacg ccaataggga ctttccattg acgtcaatgg gtggagtatt tacggtaaac      360

tgcccacttg gcagtacatc aagtgtatca tatgccaagt acgcccccta ttgacgtcaa      420

tgacggtaaa tggcccgcct ggcattatgc ccagtacatg accttatggg actttcctac      480

ttggcagtac atctacgtat tagtcatcgc tattaccatg gtcgaggtga gccccacgtt      540

ctgcttcact ctccccatct cccccccctc cccaccccca attttgtatt tatttatttt      600

ttaattattt tgtgcagcga tgggggcggg gggggggggg gggcgcgcgc caggcggggc      660

ggggcggggc gaggggcggg gcggggcgag gcggagaggt gcggcggcag ccaatcagag      720

cggcgcgctc cgaaagtttc cttttatggc gaggcggcgg cggcggcggc cctataaaaa      780

gcgaagcgcg cggcgggcgg ggagtcgctg cgacgctgcc ttcgccccgt gccccgctcc      840

gccgccgcct cgcgccgccc gccccggctc tgactgaccg cgttactccc acaggtgagc      900

gggcgggacg gcccttctcc tccgggctgt aattagcccg tttagtgaac cgtcagatcg      960

cctggagacg ccatccacgc tgttttgacc tccatagaag acaccgggac cgatccagcc     1020

tccgcggatt cgaatcccgg ccgggaacgg tgcattggaa cgcggattcc ccgtgccaag     1080

agtgacgtaa gtaccgccta tagagtctat aggcccacaa aaaatgcttt cttcttttaa     1140

tatacttttt tgtttatctt atttctaata ctttccctaa tctctttctt tcagggcaat     1200

aatgatacaa tgtatcatgc ctctttgcac cattctaaag aataacagtg ataatttctg     1260

ggttaaggca atagcaatat ttctgcatat aaatatttct gcatataaat tgtaactgat     1320

gtaagaggtt tcatattgct aatagcagct acaatccagc taccattctg cttttatttt     1380

atggttggga taaggctgga ttattctgag tccaagctag gcccttttgc taatcatgtt     1440

catacctctt atcttcctcc cacagctcct gggcaacgtg ctggtctgtg tgctggccca     1500

tcactttggc aaagaattgg gattcgaaca ccggtcgacg aattcgttaa cggatccgaa     1560

cgccaccatg ggcaagccta tccctaaccc tctgctgggc ctggactcca caggcagcgg     1620

caccggtatg gccgccgctg ccgccaccgc cgccgccgcc gccgcgccga gcggaggagg     1680

aggaggaggc gaggaggaga gactggagga aaagtcagaa gaccaggatc tccagggcct     1740

cagagacaag ccactgaagt ttaagaaggc gaagaaagac aagaaggagg acaaagaagg     1800

caagcatgag ccactacaac cttcagccca ccattctgca gagccagcag aggcaggcaa     1860

agcagaaaca tcagaaagct caggctctgc cccagcagtg ccagaagcct cggcttcccc     1920

caaacagcgg cgctccatta tccgtgaccg gggacctatg tatgatgacc ccaccttgcc     1980

tgaaggttgg acacgaaagc ttaaacaaag gaagtctggc cgatctgctg gaaagtatga     2040

tgtatatttg atcaatcccc agggaaaagc ttttcgctct aaagtagaat tgattgcata     2100

ctttgaaaag gtgggagaca cctccttgga ccctaatgat tttgacttca cggtaactgg     2160

gagagggagc ccctccagga gagagcagaa accacctaag aagcccaaat ctcccaaagc     2220

tccaggaact ggcaggggtc ggggacgccc caaagggagc ggcactggga gaccaaaggc     2280

agcagcatca gaaggtgttc aggtgaaaag ggtcctggag aagagccctg ggaaacttgt     2340

tgtcaagatg cctttccaag catcgcctgg gggtaagggt gagggaggtg gggctaccac     2400

atctgcccag gtcatggtga tcaaacgccc tggcagaaag cgaaaagctg aagctgaccc     2460

ccaggccatt cctaagaaac ggggtagaaa gcctgggagt gtggtggcag ctgctgcagc     2520

tgaggccaaa aagaaagccg tgaaggagtc ttccatacgg tctgtgcatg agactgtgct     2580

ccccatcaag aagcgcaaga cccgggagac ggtcagcatc gaggtcaagg aagtggtgaa     2640

gcccctgctg gtgtccaccc ttggtgagaa aagcgggaag ggactgaaga cctgcaagag     2700

ccctgggcgt aaaagcaagg agagcagccc caaggggcgc agcagcagtg cctcctcccc     2760

acctaagaag gagcaccatc atcaccacca tcactcagag tccacaaagg cccccatgcc     2820

actgctccca tccccacccc cacctgagcc tgagagctct gaggacccca tcagcccccc     2880

tgagcctcag gacttgagca gcagcatctg caaagaagag aagatgcccc gaggaggctc     2940

actggaaagc gatggctgcc ccaaggagcc agctaagact cagcctatgg tcgccaccac     3000

taccacagtt gcagaaaagt acaaacaccg aggggaggga gagcgcaaag acattgtttc     3060

atcttccatg ccaaggccaa acagagagga gcctgtggac agccggacgc ccgtgaccga     3120

gagagttagc taatctagaa gctcgctgat cagcctcaca agaataaagg cagctgttgt     3180

ctcttcagaa gtagctttgc acttttctaa actaggaata tcaccaggac tgttactcaa     3240

tgtgtgctgc aggaaagcac tgatatattt aaaaacaaaa ggtgtaacct atttattata     3300

taaagagttt gccttataaa tttacataaa aatgtccgtt tgtgtctttt gttgtaaaaa     3360

tcctcgagag atctacgggt ggcatccctg tgacccctcc ccagtgcctc tcctggccct     3420

ggaagttgcc actccagtgc ccaccagcct tgtcctaata aaattaagtt gcatcatttt     3480

gtctgactag gtgtccttct ataatattat ggggtggagg ggggtggtat ggagcaaggg     3540

gcaagttggg aagacaacct gtagggcctg cggggtctat tgggaaccaa gctggagtgc     3600

agtggcacaa tcttggctca ctgcaatctc cgcctcctgg gttcaagcga ttctcctgcc     3660

tcagcctccc gagttgttgg gattccaggc atgcatgacc aggctcagct aatttttgtt     3720

tttttggtag agacggggtt tcaccatatt ggccaggctg gtctccaact cctaatctca     3780

ggtgatctac ccaccttggc ctcccaaatt gctgggatta caggcgtgaa ccactgctcc     3840

cttccctgtc cttctgattt tgtaggtaac cacgtgcgga ccgagcggcc gcaggaaccc     3900

ctagtgatgg agttggccac tccctctctg cgcgctcgct cgctcactga ggccgggcga     3960

ccaaaggtcg cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc     4020

agctgcctgc agg                                                        4033


<210>  20
<211>  4143
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-CBA-5`UTR-V5-iMecp2-3`pUTR-hGHpA (M2d)

<400>  20
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgca acgcgctagt tattaatagt aatcaattac      180

ggggtcatta gttcatagcc catatatgga gttccgcgtt acataactta cggtaaatgg      240

cccgcctggc tgaccgccca acgacccccg cccattgacg tcaataatga cgtatgttcc      300

catagtaacg ccaataggga ctttccattg acgtcaatgg gtggagtatt tacggtaaac      360

tgcccacttg gcagtacatc aagtgtatca tatgccaagt acgcccccta ttgacgtcaa      420

tgacggtaaa tggcccgcct ggcattatgc ccagtacatg accttatggg actttcctac      480

ttggcagtac atctacgtat tagtcatcgc tattaccatg gtcgaggtga gccccacgtt      540

ctgcttcact ctccccatct cccccccctc cccaccccca attttgtatt tatttatttt      600

ttaattattt tgtgcagcga tgggggcggg gggggggggg gggcgcgcgc caggcggggc      660

ggggcggggc gaggggcggg gcggggcgag gcggagaggt gcggcggcag ccaatcagag      720

cggcgcgctc cgaaagtttc cttttatggc gaggcggcgg cggcggcggc cctataaaaa      780

gcgaagcgcg cggcgggcgg ggagtcgctg cgacgctgcc ttcgccccgt gccccgctcc      840

gccgccgcct cgcgccgccc gccccggctc tgactgaccg cgttactccc acaggtgagc      900

gggcgggacg gcccttctcc tccgggctgt aattagcccg tttagtgaac cgtcagatcg      960

cctggagacg ccatccacgc tgttttgacc tccatagaag acaccgggac cgatccagcc     1020

tccgcggatt cgaatcccgg ccgggaacgg tgcattggaa cgcggattcc ccgtgccaag     1080

agtgacgtaa gtaccgccta tagagtctat aggcccacaa aaaatgcttt cttcttttaa     1140

tatacttttt tgtttatctt atttctaata ctttccctaa tctctttctt tcagggcaat     1200

aatgatacaa tgtatcatgc ctctttgcac cattctaaag aataacagtg ataatttctg     1260

ggttaaggca atagcaatat ttctgcatat aaatatttct gcatataaat tgtaactgat     1320

gtaagaggtt tcatattgct aatagcagct acaatccagc taccattctg cttttatttt     1380

atggttggga taaggctgga ttattctgag tccaagctag gcccttttgc taatcatgtt     1440

catacctctt atcttcctcc cacagctcct gggcaacgtg ctggtctgtg tgctggccca     1500

tcactttggc aaagaattgg gattcgaaca ccggtcgacg tcgggagggc ggggcgcgac     1560

gtctgccgtg cggggtcccg gcatcggttg cgcgcgcgct ccctcctctc ggagagaggg     1620

ctgtggtaaa acccgtccgg aaagctagcg gatccgaacg ccaccatggg caagcctatc     1680

cctaaccctc tgctgggcct ggactccaca ggcagcggca ccggtatggc cgccgctgcc     1740

gccaccgccg ccgccgccgc cgcgccgagc ggaggaggag gaggaggcga ggaggagaga     1800

ctggaggaaa agtcagaaga ccaggatctc cagggcctca gagacaagcc actgaagttt     1860

aagaaggcga agaaagacaa gaaggaggac aaagaaggca agcatgagcc actacaacct     1920

tcagcccacc attctgcaga gccagcagag gcaggcaaag cagaaacatc agaaagctca     1980

ggctctgccc cagcagtgcc agaagcctcg gcttccccca aacagcggcg ctccattatc     2040

cgtgaccggg gacctatgta tgatgacccc accttgcctg aaggttggac acgaaagctt     2100

aaacaaagga agtctggccg atctgctgga aagtatgatg tatatttgat caatccccag     2160

ggaaaagctt ttcgctctaa agtagaattg attgcatact ttgaaaaggt gggagacacc     2220

tccttggacc ctaatgattt tgacttcacg gtaactggga gagggagccc ctccaggaga     2280

gagcagaaac cacctaagaa gcccaaatct cccaaagctc caggaactgg caggggtcgg     2340

ggacgcccca aagggagcgg cactgggaga ccaaaggcag cagcatcaga aggtgttcag     2400

gtgaaaaggg tcctggagaa gagccctggg aaacttgttg tcaagatgcc tttccaagca     2460

tcgcctgggg gtaagggtga gggaggtggg gctaccacat ctgcccaggt catggtgatc     2520

aaacgccctg gcagaaagcg aaaagctgaa gctgaccccc aggccattcc taagaaacgg     2580

ggtagaaagc ctgggagtgt ggtggcagct gctgcagctg aggccaaaaa gaaagccgtg     2640

aaggagtctt ccatacggtc tgtgcatgag actgtgctcc ccatcaagaa gcgcaagacc     2700

cgggagacgg tcagcatcga ggtcaaggaa gtggtgaagc ccctgctggt gtccaccctt     2760

ggtgagaaaa gcgggaaggg actgaagacc tgcaagagcc ctgggcgtaa aagcaaggag     2820

agcagcccca aggggcgcag cagcagtgcc tcctccccac ctaagaagga gcaccatcat     2880

caccaccatc actcagagtc cacaaaggcc cccatgccac tgctcccatc cccaccccca     2940

cctgagcctg agagctctga ggaccccatc agcccccctg agcctcagga cttgagcagc     3000

agcatctgca aagaagagaa gatgccccga ggaggctcac tggaaagcga tggctgcccc     3060

aaggagccag ctaagactca gcctatggtc gccaccacta ccacagttgc agaaaagtac     3120

aaacaccgag gggagggaga gcgcaaagac attgtttcat cttccatgcc aaggccaaac     3180

agagaggagc ctgtggacag ccggacgccc gtgaccgaga gagttagctg actttacata     3240

gagcggattg caaagcaaac caacaagaat aaaggcagct gttgtctctt ctccttatgg     3300

gtagggctct gacaaagctt cccgattaac tgaaataaaa aatatttttt tttctttcag     3360

taaacttaga gtttcgtggc ttcggggtgg gagtagttgg agcattggga tgtttttctt     3420

accgacaagc acagtcaggt tgaagaccta accagatatc tctagagata tcctcgagag     3480

atctacgggt ggcatccctg tgacccctcc ccagtgcctc tcctggccct ggaagttgcc     3540

actccagtgc ccaccagcct tgtcctaata aaattaagtt gcatcatttt gtctgactag     3600

gtgtccttct ataatattat ggggtggagg ggggtggtat ggagcaaggg gcaagttggg     3660

aagacaacct gtagggcctg cggggtctat tgggaaccaa gctggagtgc agtggcacaa     3720

tcttggctca ctgcaatctc cgcctcctgg gttcaagcga ttctcctgcc tcagcctccc     3780

gagttgttgg gattccaggc atgcatgacc aggctcagct aatttttgtt tttttggtag     3840

agacggggtt tcaccatatt ggccaggctg gtctccaact cctaatctca ggtgatctac     3900

ccaccttggc ctcccaaatt gctgggatta caggcgtgaa ccactgctcc cttccctgtc     3960

cttctgattt tgtaggtaac cacgtgcgga ccgagcggcc gcaggaaccc ctagtgatgg     4020

agttggccac tccctctctg cgcgctcgct cgctcactga ggccgggcga ccaaaggtcg     4080

cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc agctgcctgc     4140

agg                                                                   4143


<210>  21
<211>  3804
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-CBA-V5-iMecp2-hGHpA (M2e)

<400>  21
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgca acgcgctagt tattaatagt aatcaattac      180

ggggtcatta gttcatagcc catatatgga gttccgcgtt acataactta cggtaaatgg      240

cccgcctggc tgaccgccca acgacccccg cccattgacg tcaataatga cgtatgttcc      300

catagtaacg ccaataggga ctttccattg acgtcaatgg gtggagtatt tacggtaaac      360

tgcccacttg gcagtacatc aagtgtatca tatgccaagt acgcccccta ttgacgtcaa      420

tgacggtaaa tggcccgcct ggcattatgc ccagtacatg accttatggg actttcctac      480

ttggcagtac atctacgtat tagtcatcgc tattaccatg gtcgaggtga gccccacgtt      540

ctgcttcact ctccccatct cccccccctc cccaccccca attttgtatt tatttatttt      600

ttaattattt tgtgcagcga tgggggcggg gggggggggg gggcgcgcgc caggcggggc      660

ggggcggggc gaggggcggg gcggggcgag gcggagaggt gcggcggcag ccaatcagag      720

cggcgcgctc cgaaagtttc cttttatggc gaggcggcgg cggcggcggc cctataaaaa      780

gcgaagcgcg cggcgggcgg ggagtcgctg cgacgctgcc ttcgccccgt gccccgctcc      840

gccgccgcct cgcgccgccc gccccggctc tgactgaccg cgttactccc acaggtgagc      900

gggcgggacg gcccttctcc tccgggctgt aattagcccg tttagtgaac cgtcagatcg      960

cctggagacg ccatccacgc tgttttgacc tccatagaag acaccgggac cgatccagcc     1020

tccgcggatt cgaatcccgg ccgggaacgg tgcattggaa cgcggattcc ccgtgccaag     1080

agtgacgtaa gtaccgccta tagagtctat aggcccacaa aaaatgcttt cttcttttaa     1140

tatacttttt tgtttatctt atttctaata ctttccctaa tctctttctt tcagggcaat     1200

aatgatacaa tgtatcatgc ctctttgcac cattctaaag aataacagtg ataatttctg     1260

ggttaaggca atagcaatat ttctgcatat aaatatttct gcatataaat tgtaactgat     1320

gtaagaggtt tcatattgct aatagcagct acaatccagc taccattctg cttttatttt     1380

atggttggga taaggctgga ttattctgag tccaagctag gcccttttgc taatcatgtt     1440

catacctctt atcttcctcc cacagctcct gggcaacgtg ctggtctgtg tgctggccca     1500

tcactttggc aaagaattgg gattcgaaca ccggtcgacg aattcgttaa cggatccgaa     1560

cgccaccatg ggcaagccta tccctaaccc tctgctgggc ctggactcca caggcagcgg     1620

caccggtatg gccgccgctg ccgccaccgc cgccgccgcc gccgcgccga gcggaggagg     1680

aggaggaggc gaggaggaga gactggagga aaagtcagaa gaccaggatc tccagggcct     1740

cagagacaag ccactgaagt ttaagaaggc gaagaaagac aagaaggagg acaaagaagg     1800

caagcatgag ccactacaac cttcagccca ccattctgca gagccagcag aggcaggcaa     1860

agcagaaaca tcagaaagct caggctctgc cccagcagtg ccagaagcct cggcttcccc     1920

caaacagcgg cgctccatta tccgtgaccg gggacctatg tatgatgacc ccaccttgcc     1980

tgaaggttgg acacgaaagc ttaaacaaag gaagtctggc cgatctgctg gaaagtatga     2040

tgtatatttg atcaatcccc agggaaaagc ttttcgctct aaagtagaat tgattgcata     2100

ctttgaaaag gtgggagaca cctccttgga ccctaatgat tttgacttca cggtaactgg     2160

gagagggagc ccctccagga gagagcagaa accacctaag aagcccaaat ctcccaaagc     2220

tccaggaact ggcaggggtc ggggacgccc caaagggagc ggcactggga gaccaaaggc     2280

agcagcatca gaaggtgttc aggtgaaaag ggtcctggag aagagccctg ggaaacttgt     2340

tgtcaagatg cctttccaag catcgcctgg gggtaagggt gagggaggtg gggctaccac     2400

atctgcccag gtcatggtga tcaaacgccc tggcagaaag cgaaaagctg aagctgaccc     2460

ccaggccatt cctaagaaac ggggtagaaa gcctgggagt gtggtggcag ctgctgcagc     2520

tgaggccaaa aagaaagccg tgaaggagtc ttccatacgg tctgtgcatg agactgtgct     2580

ccccatcaag aagcgcaaga cccgggagac ggtcagcatc gaggtcaagg aagtggtgaa     2640

gcccctgctg gtgtccaccc ttggtgagaa aagcgggaag ggactgaaga cctgcaagag     2700

ccctgggcgt aaaagcaagg agagcagccc caaggggcgc agcagcagtg cctcctcccc     2760

acctaagaag gagcaccatc atcaccacca tcactcagag tccacaaagg cccccatgcc     2820

actgctccca tccccacccc cacctgagcc tgagagctct gaggacccca tcagcccccc     2880

tgagcctcag gacttgagca gcagcatctg caaagaagag aagatgcccc gaggaggctc     2940

actggaaagc gatggctgcc ccaaggagcc agctaagact cagcctatgg tcgccaccac     3000

taccacagtt gcagaaaagt acaaacaccg aggggaggga gagcgcaaag acattgtttc     3060

atcttccatg ccaaggccaa acagagagga gcctgtggac agccggacgc ccgtgaccga     3120

gagagttagc tgagtcgaga gatctacggg tggcatccct gtgacccctc cccagtgcct     3180

ctcctggccc tggaagttgc cactccagtg cccaccagcc ttgtcctaat aaaattaagt     3240

tgcatcattt tgtctgacta ggtgtccttc tataatatta tggggtggag gggggtggta     3300

tggagcaagg ggcaagttgg gaagacaacc tgtagggcct gcggggtcta ttgggaacca     3360

agctggagtg cagtggcaca atcttggctc actgcaatct ccgcctcctg ggttcaagcg     3420

attctcctgc ctcagcctcc cgagttgttg ggattccagg catgcatgac caggctcagc     3480

taatttttgt ttttttggta gagacggggt ttcaccatat tggccaggct ggtctccaac     3540

tcctaatctc aggtgatcta cccaccttgg cctcccaaat tgctgggatt acaggcgtga     3600

accactgctc ccttccctgt ccttctgatt ttgtaggtaa ccacgtgcgg accgagcggc     3660

cgcaggaacc cctagtgatg gagttggcca ctccctctct gcgcgctcgc tcgctcactg     3720

aggccgggcg accaaaggtc gcccgacgcc cgggctttgc ccgggcggcc tcagtgagcg     3780

agcgagcgcg cagctgcctg cagg                                            3804


<210>  22
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  sequence encoding an shRNA that inhibits expression of MeCP2, 
       shMecp2

<400>  22
gattgtagat tcaggttaa                                                    19


<210>  23
<211>  3340
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-shMecp2-CBA-GFP

<400>  23
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggcctct agagaacgct gacgtcatca acccgctcca      180

aggaatcgcg ggcccagtgt cactaggcgg gaacacccag cgcgcgtgcg ccctggcagg      240

aagatggctg tgagggacag gggagtggcg ccctgcaata tttgcatgtc gctatgtgtt      300

ctgggaaatc accataaacg tgaaatgtct ttggatttgg gaatcttata agttctgtat      360

gagaccacag atccccgatt gtagattcag gttaattcaa gagattaacc tgaatctaca      420

atctttttgg aaaagcttat cgataacgcg ctagttatta atagtaatca attacggggt      480

cattagttca tagcccatat atggagttcc gcgttacata acttacggta aatggcccgc      540

ctggctgacc gcccaacgac ccccgcccat tgacgtcaat aatgacgtat gttcccatag      600

taacgccaat agggactttc cattgacgtc aatgggtgga gtatttacgg taaactgccc      660

acttggcagt acatcaagtg tatcatatgc caagtacgcc ccctattgac gtcaatgacg      720

gtaaatggcc cgcctggcat tatgcccagt acatgacctt atgggacttt cctacttggc      780

agtacatcta cgtattagtc atcgctatta ccatggtcga ggtgagcccc acgttctgct      840

tcactctccc catctccccc ccctccccac ccccaatttt gtatttattt attttttaat      900

tattttgtgc agcgatgggg gcgggggggg ggggggggcg cgcgccaggc ggggcggggc      960

ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc ggcagccaat cagagcggcg     1020

cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg gcggccctat aaaaagcgaa     1080

gcgcgcggcg ggcggggagt cgctgcgacg ctgccttcgc cccgtgcccc gctccgccgc     1140

cgcctcgcgc cgcccgcccc ggctctgact gaccgcgtta ctcccacagg tgagcgggcg     1200

ggacggccct tctcctccgg gctgtaatta gcccgtttag tgaaccgtca gatcgcctgg     1260

agacgccatc cacgctgttt tgacctccat agaagacacc gggaccgatc cagcctccgc     1320

ggattcgaat cccggccggg aacggtgcat tggaacgcgg attccccgtg ccaagagtga     1380

cgtaagtacc gcctatagag tctataggcc cacaaaaaat gctttcttct tttaatatac     1440

ttttttgttt atcttatttc taatactttc cctaatctct ttctttcagg gcaataatga     1500

tacaatgtat catgcctctt tgcaccattc taaagaataa cagtgataat ttctgggtta     1560

aggcaatagc aatatttctg catataaata tttctgcata taaattgtaa ctgatgtaag     1620

aggtttcata ttgctaatag cagctacaat ccagctacca ttctgctttt attttatggt     1680

tgggataagg ctggattatt ctgagtccaa gctaggccct tttgctaatc atgttcatac     1740

ctcttatctt cctcccacag ctcctgggca acgtgctggt ctgtgtgctg gcccatcact     1800

ttggcaaaga attgggattc gaacacggtc gacgaattcg ttaacggatc cgaacgccac     1860

catgggcaag cctatcccta accctctgct gggcctggac tccacaggca gcggcaccgg     1920

tggatcctct agaatggtga gcaagggcga ggagctgttc accggggtgg tgcccatcct     1980

ggtcgagctg gacggcgacg taaacggcca caagttcagc gtgtccggcg agggcgaggg     2040

cgatgccacc tacggcaagc tgaccctgaa gttcatctgc accaccggca agctgcccgt     2100

gccctggccc accctcgtga ccaccctgac ctacggcgtg cagtgcttca gccgctaccc     2160

cgaccacatg aagcagcacg acttcttcaa gtccgccatg cccgaaggct acgtccagga     2220

gcgcaccatc ttcttcaagg acgacggcaa ctacaagacc cgcgccgagg tgaagttcga     2280

gggcgacacc ctggtgaacc gcatcgagct gaagggcatc gacttcaagg aggacggcaa     2340

catcctgggg cacaagctgg agtacaacta caacagccac aacgtctata tcatggccga     2400

caagcagaag aacggcatca aggtgaactt caagatccgc cacaacatcg aggacggcag     2460

cgtgcagctc gccgaccact accagcagaa cacccccatc ggcgacggcc ccgtgctgct     2520

gcccgacaac cactacctga gcacccagtc cgccctgagc aaagacccca acgagaagcg     2580

cgatcacatg gtcctgctgg agttcgtgac cgccgccggg atcactctcg gcatggacga     2640

gctgtacaag taagctagag atatcctcga gagatcgatc tacgggtggc atccctgtga     2700

cccctcccca gtgcctctcc tggccctgga agttgccact ccagtgccca ccagccttgt     2760

cctaataaaa ttaagttgca tcattttgtc tgactaggtg tccttctata atattatggg     2820

gtggaggggg gtggtatgga gcaaggggca agttgggaag acaacctgta gggcctgcgg     2880

ggtctattgg gaaccaagct ggagtgcagt ggcacaatct tggctcactg caatctccgc     2940

ctcctgggtt caagcgattc tcctgcctca gcctcccgag ttgttgggat tccaggcatg     3000

catgaccagg ctcagctaat ttttgttttt ttggtagaga cggggtttca ccatattggc     3060

caggctggtc tccaactcct aatctcaggt gatctaccca ccttggcctc ccaaattgct     3120

gggattacag gcgtgaacca ctgctccctt ccctgtcctt ctgattttgt aggtaaccac     3180

gtgcggaccg agcggccgca ggaaccccta gtgatggagt tggccactcc ctctctgcgc     3240

gctcgctcgc tcactgaggc cgggcgacca aaggtcgccc gacgcccggg ctttgcccgg     3300

gcggcctcag tgagcgagcg agcgcgcagc tgcctgcagg                           3340


<210>  24
<211>  4339
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-shMecp2-iMecp2

<400>  24
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggcctct agagaacgct gacgtcatca acccgctcca      180

aggaatcgcg ggcccagtgt cactaggcgg gaacacccag cgcgcgtgcg ccctggcagg      240

aagatggctg tgagggacag gggagtggcg ccctgcaata tttgcatgtc gctatgtgtt      300

ctgggaaatc accataaacg tgaaatgtct ttggatttgg gaatcttata agttctgtat      360

gagaccacag atccccgatt gtagattcag gttaattcaa gagattaacc tgaatctaca      420

atctttttgg aaaagcttat cgataacgcg ctagttatta atagtaatca attacggggt      480

cattagttca tagcccatat atggagttcc gcgttacata acttacggta aatggcccgc      540

ctggctgacc gcccaacgac ccccgcccat tgacgtcaat aatgacgtat gttcccatag      600

taacgccaat agggactttc cattgacgtc aatgggtgga gtatttacgg taaactgccc      660

acttggcagt acatcaagtg tatcatatgc caagtacgcc ccctattgac gtcaatgacg      720

gtaaatggcc cgcctggcat tatgcccagt acatgacctt atgggacttt cctacttggc      780

agtacatcta cgtattagtc atcgctatta ccatggtcga ggtgagcccc acgttctgct      840

tcactctccc catctccccc ccctccccac ccccaatttt gtatttattt attttttaat      900

tattttgtgc agcgatgggg gcgggggggg ggggggggcg cgcgccaggc ggggcggggc      960

ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc ggcagccaat cagagcggcg     1020

cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg gcggccctat aaaaagcgaa     1080

gcgcgcggcg ggcggggagt cgctgcgacg ctgccttcgc cccgtgcccc gctccgccgc     1140

cgcctcgcgc cgcccgcccc ggctctgact gaccgcgtta ctcccacagg tgagcgggcg     1200

ggacggccct tctcctccgg gctgtaatta gcccgtttag tgaaccgtca gatcgcctgg     1260

agacgccatc cacgctgttt tgacctccat agaagacacc gggaccgatc cagcctccgc     1320

ggattcgaat cccggccggg aacggtgcat tggaacgcgg attccccgtg ccaagagtga     1380

cgtaagtacc gcctatagag tctataggcc cacaaaaaat gctttcttct tttaatatac     1440

ttttttgttt atcttatttc taatactttc cctaatctct ttctttcagg gcaataatga     1500

tacaatgtat catgcctctt tgcaccattc taaagaataa cagtgataat ttctgggtta     1560

aggcaatagc aatatttctg catataaata tttctgcata taaattgtaa ctgatgtaag     1620

aggtttcata ttgctaatag cagctacaat ccagctacca ttctgctttt attttatggt     1680

tgggataagg ctggattatt ctgagtccaa gctaggccct tttgctaatc atgttcatac     1740

ctcttatctt cctcccacag ctcctgggca acgtgctggt ctgtgtgctg gcccatcact     1800

ttggcaaaga attgggattc gaacacggtc gacgaattcg ttaacggatc cgaacgccac     1860

catgggcaag cctatcccta accctctgct gggcctggac tccacaggca gcggcaccgg     1920

tatggccgcc gctgccgcca ccgccgccgc cgccgccgcg ccgagcggag gaggaggagg     1980

aggcgaggag gagagactgg aggaaaagtc agaagaccag gatctccagg gcctcagaga     2040

caagccactg aagtttaaga aggcgaagaa agacaagaag gaggacaaag aaggcaagca     2100

tgagccacta caaccttcag cccaccattc tgcagagcca gcagaggcag gcaaagcaga     2160

aacatcagaa agctcaggct ctgccccagc agtgccagaa gcctcggctt cccccaaaca     2220

gcggcgctcc attatccgtg accggggacc tatgtatgat gaccccacct tgcctgaagg     2280

ttggacacga aagcttaaac aaaggaagtc tggccgatct gctggaaagt atgatgtata     2340

tttgatcaat ccccagggaa aagcttttcg ctctaaagta gaattgattg catactttga     2400

aaaggtggga gacacctcct tggaccctaa tgattttgac ttcacggtaa ctgggagagg     2460

gagcccctcc aggagagagc agaaaccacc taagaagccc aaatctccca aagctccagg     2520

aactggcagg ggtcggggac gccccaaagg gagcggcact gggagaccaa aggcagcagc     2580

atcagaaggt gttcaggtga aaagggtcct ggagaagagc cctgggaaac ttgttgtcaa     2640

gatgcctttc caagcatcgc ctgggggtaa gggtgaggga ggtggggcta ccacatctgc     2700

ccaggtcatg gtgatcaaac gccctggcag aaagcgaaaa gctgaagctg acccccaggc     2760

cattcctaag aaacggggta gaaagcctgg gagtgtggtg gcagctgctg cagctgaggc     2820

caaaaagaaa gccgtgaagg agtcttccat acggtctgtg catgagactg tgctccccat     2880

caagaagcgc aagacccggg agacggtcag catcgaggtc aaggaagtgg tgaagcccct     2940

gctggtgtcc acccttggtg agaaaagcgg gaagggactg aagacctgca agagccctgg     3000

gcgtaaaagc aaggagagca gccccaaggg gcgcagcagc agtgcctcct ccccacctaa     3060

gaaggagcac catcatcacc accatcactc agagtccaca aaggccccca tgccactgct     3120

cccatcccca cccccacctg agcctgagag ctctgaggac cccatcagcc cccctgagcc     3180

tcaggacttg agcagcagca tctgcaaaga agagaagatg ccccgaggag gctcactgga     3240

aagcgatggc tgccccaagg agccagctaa gactcagcct atggtcgcca ccactaccac     3300

agttgcagaa aagtacaaac accgagggga gggagagcgc aaagacattg tttcatcttc     3360

catgccaagg ccaaacagag aggagcctgt ggacagccgg acgcccgtga ccgagagagt     3420

tagctgactt tacatagagc ggattgcaaa gcaaaccaac aagaataaag gcagctgttg     3480

tctcttctcc ttatgggtag ggctctgaca aagcttcccg attaactgaa ataaaaaata     3540

tttttttttc tttcagtaaa cttagagttt cgtggcttcg gggtgggagt agttggagca     3600

ttgggatgtt tttcttaccg acaagcacag tcaggttgaa gacctaacca gatatctcta     3660

gagatatcct cgagagatct acgggtggca tccctgtgac ccctccccag tgcctctcct     3720

ggccctggaa gttgccactc cagtgcccac cagccttgtc ctaataaaat taagttgcat     3780

cattttgtct gactaggtgt ccttctataa tattatgggg tggagggggg tggtatggag     3840

caaggggcaa gttgggaaga caacctgtag ggcctgcggg gtctattggg aaccaagctg     3900

gagtgcagtg gcacaatctt ggctcactgc aatctccgcc tcctgggttc aagcgattct     3960

cctgcctcag cctcccgagt tgttgggatt ccaggcatgc atgaccaggc tcagctaatt     4020

tttgtttttt tggtagagac ggggtttcac catattggcc aggctggtct ccaactccta     4080

atctcaggtg atctacccac cttggcctcc caaattgctg ggattacagg cgtgaaccac     4140

tgctcccttc cctgtccttc tgattttgta ggtaaccacg tgcggaccga gcggccgcag     4200

gaacccctag tgatggagtt ggccactccc tctctgcgcg ctcgctcgct cactgaggcc     4260

gggcgaccaa aggtcgcccg acgcccgggc tttgcccggg cggcctcagt gagcgagcga     4320

gcgcgcagct gcctgcagg                                                  4339


<210>  25
<211>  3981
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector AAV-CBA-iMecp2-3`pUTR-hGHpA (M2f)

<400>  25
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgca acgcgctagt tattaatagt aatcaattac      180

ggggtcatta gttcatagcc catatatgga gttccgcgtt acataactta cggtaaatgg      240

cccgcctggc tgaccgccca acgacccccg cccattgacg tcaataatga cgtatgttcc      300

catagtaacg ccaataggga ctttccattg acgtcaatgg gtggagtatt tacggtaaac      360

tgcccacttg gcagtacatc aagtgtatca tatgccaagt acgcccccta ttgacgtcaa      420

tgacggtaaa tggcccgcct ggcattatgc ccagtacatg accttatggg actttcctac      480

ttggcagtac atctacgtat tagtcatcgc tattaccatg gtcgaggtga gccccacgtt      540

ctgcttcact ctccccatct cccccccctc cccaccccca attttgtatt tatttatttt      600

ttaattattt tgtgcagcga tgggggcggg gggggggggg gggcgcgcgc caggcggggc      660

ggggcggggc gaggggcggg gcggggcgag gcggagaggt gcggcggcag ccaatcagag      720

cggcgcgctc cgaaagtttc cttttatggc gaggcggcgg cggcggcggc cctataaaaa      780

gcgaagcgcg cggcgggcgg ggagtcgctg cgacgctgcc ttcgccccgt gccccgctcc      840

gccgccgcct cgcgccgccc gccccggctc tgactgaccg cgttactccc acaggtgagc      900

gggcgggacg gcccttctcc tccgggctgt aattagcccg tttagtgaac cgtcagatcg      960

cctggagacg ccatccacgc tgttttgacc tccatagaag acaccgggac cgatccagcc     1020

tccgcggatt cgaatcccgg ccgggaacgg tgcattggaa cgcggattcc ccgtgccaag     1080

agtgacgtaa gtaccgccta tagagtctat aggcccacaa aaaatgcttt cttcttttaa     1140

tatacttttt tgtttatctt atttctaata ctttccctaa tctctttctt tcagggcaat     1200

aatgatacaa tgtatcatgc ctctttgcac cattctaaag aataacagtg ataatttctg     1260

ggttaaggca atagcaatat ttctgcatat aaatatttct gcatataaat tgtaactgat     1320

gtaagaggtt tcatattgct aatagcagct acaatccagc taccattctg cttttatttt     1380

atggttggga taaggctgga ttattctgag tccaagctag gcccttttgc taatcatgtt     1440

catacctctt atcttcctcc cacagctcct gggcaacgtg ctggtctgtg tgctggccca     1500

tcactttggc aaagaattgg gattcgaaca ccggtcgacg aattcgttaa cggatccgcc     1560

accatggccg ccgctgccgc caccgccgcc gccgccgccg cgccgagcgg aggaggagga     1620

ggaggcgagg aggagagact ggaggaaaag tcagaagacc aggatctcca gggcctcaga     1680

gacaagccac tgaagtttaa gaaggcgaag aaagacaaga aggaggacaa agaaggcaag     1740

catgagccac tacaaccttc agcccaccat tctgcagagc cagcagaggc aggcaaagca     1800

gaaacatcag aaagctcagg ctctgcccca gcagtgccag aagcctcggc ttcccccaaa     1860

cagcggcgct ccattatccg tgaccgggga cctatgtatg atgaccccac cttgcctgaa     1920

ggttggacac gaaagcttaa acaaaggaag tctggccgat ctgctggaaa gtatgatgta     1980

tatttgatca atccccaggg aaaagctttt cgctctaaag tagaattgat tgcatacttt     2040

gaaaaggtgg gagacacctc cttggaccct aatgattttg acttcacggt aactgggaga     2100

gggagcccct ccaggagaga gcagaaacca cctaagaagc ccaaatctcc caaagctcca     2160

ggaactggca ggggtcgggg acgccccaaa gggagcggca ctgggagacc aaaggcagca     2220

gcatcagaag gtgttcaggt gaaaagggtc ctggagaaga gccctgggaa acttgttgtc     2280

aagatgcctt tccaagcatc gcctgggggt aagggtgagg gaggtggggc taccacatct     2340

gcccaggtca tggtgatcaa acgccctggc agaaagcgaa aagctgaagc tgacccccag     2400

gccattccta agaaacgggg tagaaagcct gggagtgtgg tggcagctgc tgcagctgag     2460

gccaaaaaga aagccgtgaa ggagtcttcc atacggtctg tgcatgagac tgtgctcccc     2520

atcaagaagc gcaagacccg ggagacggtc agcatcgagg tcaaggaagt ggtgaagccc     2580

ctgctggtgt ccacccttgg tgagaaaagc gggaagggac tgaagacctg caagagccct     2640

gggcgtaaaa gcaaggagag cagccccaag gggcgcagca gcagtgcctc ctccccacct     2700

aagaaggagc accatcatca ccaccatcac tcagagtcca caaaggcccc catgccactg     2760

ctcccatccc cacccccacc tgagcctgag agctctgagg accccatcag cccccctgag     2820

cctcaggact tgagcagcag catctgcaaa gaagagaaga tgccccgagg aggctcactg     2880

gaaagcgatg gctgccccaa ggagccagct aagactcagc ctatggtcgc caccactacc     2940

acagttgcag aaaagtacaa acaccgaggg gagggagagc gcaaagacat tgtttcatct     3000

tccatgccaa ggccaaacag agaggagcct gtggacagcc ggacgcccgt gaccgagaga     3060

gttagctgac tttacataga gcggattgca aagcaaacca acaagaataa aggcagctgt     3120

tgtctcttct ccttatgggt agggctctga caaagcttcc cgattaactg aaataaaaaa     3180

tatttttttt tctttcagta aacttagagt ttcgtggctt cggggtggga gtagttggag     3240

cattgggatg tttttcttac cgacaagcac agtcaggttg aagacctaac cagatatctc     3300

tagagatatc ctcgagagat ctacgggtgg catccctgtg acccctcccc agtgcctctc     3360

ctggccctgg aagttgccac tccagtgccc accagccttg tcctaataaa attaagttgc     3420

atcattttgt ctgactaggt gtccttctat aatattatgg ggtggagggg ggtggtatgg     3480

agcaaggggc aagttgggaa gacaacctgt agggcctgcg gggtctattg ggaaccaagc     3540

tggagtgcag tggcacaatc ttggctcact gcaatctccg cctcctgggt tcaagcgatt     3600

ctcctgcctc agcctcccga gttgttggga ttccaggcat gcatgaccag gctcagctaa     3660

tttttgtttt tttggtagag acggggtttc accatattgg ccaggctggt ctccaactcc     3720

taatctcagg tgatctaccc accttggcct cccaaattgc tgggattaca ggcgtgaacc     3780

actgctccct tccctgtcct tctgattttg taggtaacca cgtgcggacc gagcggccgc     3840

aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg     3900

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc     3960

gagcgcgcag ctgcctgcag g                                               3981


<210>  26
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  sgRNA sequence designed on the third exon of MECP2

<400>  26
aagcttaagc aaaggaaatc                                                   20


<210>  27
<211>  61
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  wild-type MECP2 gene exon 3 sequence

<400>  27
acacggaagc ttaagcaaag gaaatctggc cgctctgctg ggaagtatga tgtgtatttg       60

a                                                                       61


<210>  28
<211>  20
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  amino acid sequence encoded by wild-type MECP2 gene exon 3

<400>  28

Thr Arg Lys Leu Lys Gln Arg Lys Ser Gly Arg Ser Ala Gly Lys Tyr 
1               5                   10                  15      


Asp Val Tyr Leu 
            20  


<210>  29
<211>  19
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  MECP2 knock-out (KO) exon 3 amino acid sequence

<400>  29

Thr Arg Lys Leu Lys Gln Arg Asn Leu Ala Ala Leu Leu Gly Ser Met 
1               5                   10                  15      


Met Cys Ile 
            


<210>  30
<211>  60
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  MECP2 KO exon 3 sequence (single nucleotide deletion)

<400>  30
acacggaagc ttaagcaaag gaatctggcc gctctgctgg gaagtatgat gtgtatttga       60


