                         SEQUENCE LISTING

<110>  REGENXBIO INC.
 
<120>  TREATMENT OF MUCOPOLYSACCHARIDOSIS IVA

<130>  12656-130-228

<140>  PCT/US2021/015436
<141>  2021-01-28

<150>  62/967,499
<151>  2020-01-29

<160>  32     

<170>  PatentIn version 3.5

<210>  1
<211>  738
<212>  PRT
<213>  Adeno-associated Virus (AAV)

<220>
<223>  AAV8 capsid protein

<400>  1

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 Lys Pro 
            20                  25                  30          


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


Thr Phe Glu Asp Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser 
            420                 425                 430         


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


Ser Arg Thr Gln Thr Thr Gly Gly Thr Ala Asn Thr Gln Thr Leu Gly 
    450                 455                 460                 


Phe Ser Gln Gly Gly Pro Asn Thr Met Ala Asn Gln Ala Lys Asn Trp 
465                 470                 475                 480 


Leu Pro Gly Pro Cys Tyr Arg Gln Gln Arg Val Ser Thr Thr Thr Gly 
                485                 490                 495     


Gln Asn Asn Asn Ser Asn Phe Ala Trp Thr Ala Gly Thr Lys Tyr His 
            500                 505                 510         


Leu Asn Gly Arg Asn Ser Leu Ala Asn Pro Gly Ile Ala Met Ala Thr 
        515                 520                 525             


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


Phe Gly Lys Gln Asn Ala Ala Arg Asp Asn Ala Asp Tyr Ser Asp Val 
545                 550                 555                 560 


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


Glu Glu Tyr Gly Ile Val Ala Asp Asn Leu Gln Gln Gln Asn Thr Ala 
            580                 585                 590         


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


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


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


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


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


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


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


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


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


Asn Leu 
        


<210>  2
<211>  1569
<212>  DNA
<213>  homo sapiens

<220>
<223>  Human GALNS (hGALNS)

<400>  2
atggcggcgg ttgtcgcggc gacgaggtgg tggcagctgt tgctggtgct cagcgccgcg       60

gggatggggg cctcgggcgc cccgcagccc cccaacatcc tgctcctgct catggacgac      120

atgggatggg gtgacctcgg ggtgtatgga gagccctcca gagagacccc gaatttggac      180

cggatggctg cagaagggct gcttttccca aacttctatt ctgccaaccc tctgtgctcg      240

ccatcgaggg cggcactgct cacaggacgg ctacccatcc gcaatggctt ctacaccacc      300

aacgcccatg ccagaaacgc ctacacaccg caggagattg tgggcggcat cccagactcg      360

gagcagctcc tgccggagct tctgaagaag gccggctacg tcagcaagat tgtcggcaag      420

tggcatctgg gtcacaggcc ccagttccac cccctgaagc acggatttga tgagtggttt      480

ggatccccca actgccactt tggaccttat gacaacaagg ccaggcccaa catccctgtg      540

tacagggact gggagatggt tggcagatat tatgaagaat ttcctattaa tctgaagacg      600

ggggaagcca acctcaccca gatctacctg caggaagccc tggacttcat taagagacag      660

gcacggcacc accccttttt cctctactgg gctgtcgacg ccacgcacgc acccgtctat      720

gcctccaaac ccttcttggg caccagtcag cgagggcggt atggagacgc cgtccgggag      780

attgatgaca gcattgggaa gatactggag ctcctccaag acctgcacgt cgcggacaac      840

accttcgtct tcttcacgtc ggacaacggc gctgccctca tttccgcccc cgaacaaggt      900

ggcagcaacg gcccctttct gtgtgggaag cagaccacgt ttgaaggagg gatgagggag      960

cctgccctcg catggtggcc agggcacgtc actgcaggcc aggtgagcca ccagctgggc     1020

agcatcatgg acctcttcac caccagcctg gcccttgcgg gcctgacgcc gcccagcgac     1080

agggccattg atggcctcaa cctcctcccc accctcctgc agggccggct gatggacagg     1140

cctatcttct attaccgtgg cgacacgctg atggcggcca ccctcgggca gcacaaggct     1200

cacttctgga cctggaccaa ctcctgggag aacttcagac agggcattga tttctgccct     1260

gggcagaacg tttcaggggt cacaactcac aatctggaag accacacgaa gctgcccctg     1320

atcttccacc tgggacggga cccaggggag aggttccccc tcagctttgc cagcgccgag     1380

taccaggagg ccctcagcag gatcacctcg gtcgtccagc agcaccagga ggccttggtc     1440

cccgcgcagc cccagctcaa cgtgtgcaac tgggcggtca tgaactgggc acctccgggc     1500

tgtgaaaagt tagggaagtg tctgacacct ccagaatcca ttcccaagaa gtgcctctgg     1560

tcccactag                                                             1569


<210>  3
<211>  1569
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hGLANSco (Codon Optimized)

<400>  3
atggctgctg ttgttgccgc taccagatgg tggcagctgc tgctggttct gtctgccgct       60

ggaatgggag cttctggtgc tccccagcct cctaacattc tgctgctgct catggacgac      120

atgggctggg gcgatctggg agtgtatggc gagcctagca gagagacacc caacctggat      180

agaatggccg ccgagggcct gctgttcccc aatttctaca gcgccaatcc tctgtgcagc      240

ccctctagag ctgctctgct gacaggcaga ctgcccatca gaaacggctt ctacaccacc      300

aacgctcacg cccggaatgc ctacacaccc caagagatcg ttggcggcat ccccgattct      360

gagcagctcc tgcctgagct gctgaagaag gccggctacg tcagcaagat cgtcggcaaa      420

tggcacctgg gccacagacc tcagtttcac cctctgaagc acggcttcga cgagtggttc      480

ggcagcccca attgtcactt cggcccctac gacaacaagg ccagacctaa catccccgtg      540

tacagagact gggagatggt cggacggtac tacgaggaat tccccatcaa cctgaaaacc      600

ggcgaggcca atctgaccca gatctacctg caagaggccc tggacttcat caagcggcag      660

gccagacacc atcctttctt tctgtactgg gccgtcgacg ccacacacgc ccctgtgtat      720

gccagcaagc cttttctggg caccagccag cgtggcagat atggcgacgc cgtgcgggaa      780

atcgatgaca gcatcggcaa gatcctggaa ctgctgcagg atctgcacgt ggccgacaac      840

accttcgtgt tcttcaccag cgacaacggc gctgccctga tttctgctcc tgagcaaggc      900

ggcagcaacg gcccatttct gtgtggcaag cagaccacct ttgaaggcgg catgagagag      960

cctgctctgg cttggtggcc tggacatgtg acagccggac aagtgtctca ccagctgggc     1020

agcatcatgg acctgtttac cacctctctg gccctggccg gactgacacc tccatctgat     1080

agagccatcg acggcctgaa cctgctgcct acactgcttc agggcagact gatggacaga     1140

cccatcttct actaccgggg cgacaccctg atggccgcta cactgggaca gcacaaggcc     1200

cacttttgga cctggaccaa cagctgggag aacttccggc agggcatcga cttttgccct     1260

ggccagaatg tgtccggcgt gaccacacac aatctggaag atcacaccaa gctgcccctg     1320

atctttcacc tgggcagaga tcccggcgag agattccctc tgtcttttgc cagcgccgag     1380

taccaagaag ccctgagcag aatcacctcc gtggtgcagc agcaccaaga ggctctggtt     1440

ccagctcagc cccagctgaa cgtgtgtaat tgggccgtga tgaactgggc ccctcctgga     1500

tgtgaaaagc tgggcaagtg tctgacccct cctgagagca tccccaagaa atgcctgtgg     1560

tcccactga                                                             1569


<210>  4
<211>  1604
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  D8-hGALNS

<400>  4
accgccatgc ggggtccgag cggggctctg tggctgctcc tggctctgcg caccgtgctc       60

ggatcagatg atgatgatga tgatgatgat gccgaggcag aaaccggtgc cccgcagccc      120

cccaacatcc tgctcctgct catggacgac atgggatggg gtgacctcgg ggtgtatgga      180

gagccctcca gagagacccc gaatttggac cggatggctg cagaagggct gcttttccca      240

aacttctatt ctgccaaccc tctgtgctcg ccatcgaggg cggcactgct cacaggacgg      300

ctacccatcc gcaatggctt ctacaccacc aacgcccatg ccagaaacgc ctacacaccg      360

caggagattg tgggcggcat cccagactcg gagcagctcc tgccggagct tctgaagaag      420

gccggctacg tcagcaagat tgtcggcaag tggcatctgg gtcacaggcc ccagttccac      480

cccctgaagc acggatttga tgagtggttt ggatccccca actgccactt tggaccttat      540

gacaacaagg ccaggcccaa catccctgtg tacagggact gggagatggt tggcagatat      600

tatgaagaat ttcctattaa tctgaagacg ggggaagcca acctcaccca gatctacctg      660

caggaagccc tggacttcat taagagacag gcacggcacc accccttttt cctctactgg      720

gctgtcgacg ccacgcacgc acccgtctat gcctccaaac ccttcttggg caccagtcag      780

cgagggcggt atggagacgc cgtccgggag attgatgaca gcattgggaa gatactggag      840

ctcctccaag acctgcacgt cgcggacaac accttcgtct tcttcacgtc ggacaacggc      900

gctgccctca tttccgcccc cgaacaaggt ggcagcaacg gcccctttct gtgtgggaag      960

cagaccacgt ttgaaggagg gatgagggag cctgccctcg catggtggcc agggcacgtc     1020

actgcaggcc aggtgagcca ccagctgggc agcatcatgg acctcttcac caccagcctg     1080

gcccttgcgg gcctgacgcc gcccagcgac agggccattg atggcctcaa cctcctcccc     1140

accctcctgc agggccggct gatggacagg cctatcttct attaccgtgg cgacacgctg     1200

atggcggcca ccctcgggca gcacaaggct cacttctgga cctggaccaa ctcctgggag     1260

aacttcagac agggcattga tttctgccct gggcagaacg tttcaggggt cacaactcac     1320

aatctggaag accacacgaa gctgcccctg atcttccacc tgggacggga cccaggggag     1380

aggttccccc tcagctttgc cagcgccgag taccaggagg ccctcagcag gatcacctcg     1440

gtcgtccagc agcaccagga ggccttggtc cccgcgcagc cccagctcaa cgtgtgcaac     1500

tgggcggtca tgaactgggc acctccgggc tgtgaaaagt tagggaagtg tctgacacct     1560

ccagaatcca ttcccaagaa gtgcctctgg tcccactagc tcga                      1604


<210>  5
<211>  1593
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  D8-GALNSco (codon optimized)

<400>  5
atgagaggac catctggtgc tctgtggctg ctgctggctc tgagaacagt gctgggcagc       60

gacgacgatg atgacgatga cgacgccgag gctgaaacag gtgctcccca gcctcctaac      120

atcctgctgc tgctcatgga cgatatgggc tggggcgatc tgggagtgta tggcgagcct      180

agcagagaga cacccaacct ggatagaatg gccgccgagg gcctgctgtt ccccaatttc      240

tacagcgcca atcctctgtg cagcccctct agagctgctc tgctgacagg cagactgccc      300

atcagaaacg gcttctacac caccaacgct cacgcccgga atgcctacac accccaagag      360

atcgttggcg gcatccccga ttctgaacag ctgctgcctg agctgctgaa gaaggccggc      420

tacgtcagca agatcgtcgg caaatggcac ctgggccaca gacctcagtt tcaccctctg      480

aagcacggct tcgacgagtg gttcggcagc cccaattgtc acttcggccc ctacgacaac      540

aaggccagac caaacatccc cgtgtacaga gactgggaga tggtcggacg gtactacgag      600

gaattcccca tcaacctgaa aaccggcgag gccaatctga cccagatcta cctgcaagag      660

gccctggact tcatcaagcg gcaggccaga caccatcctt tctttctgta ctgggccgtc      720

gacgccacac acgcccctgt gtatgccagc aagccttttc tgggcaccag ccagcgtggc      780

agatatggcg acgccgtgcg ggaaatcgat gacagcatcg gcaagatcct ggaactgctg      840

caggatctgc acgtggccga caacaccttc gtgttcttca ccagcgacaa cggcgctgcc      900

ctgatttctg ctcctgagca aggcggcagc aacggcccat ttctgtgtgg caagcagacc      960

acctttgaag gcggcatgag agagcctgct ctggcttggt ggcctggaca tgtgacagcc     1020

ggacaagtgt ctcaccagct gggctccatc atggacctgt ttaccacctc tctggccctg     1080

gccggactga cacctccatc tgatagagcc atcgacggcc tgaacctgct gcctacactg     1140

cttcagggca gactgatgga cagacccatc ttctactacc ggggcgacac cctgatggcc     1200

gctacactgg gacagcacaa ggcccacttt tggacctgga ccaacagctg ggagaacttc     1260

cggcagggca tcgacttttg ccctggccag aatgtgtccg gcgtgaccac acacaatctg     1320

gaagatcaca ccaagctgcc cctgatcttt cacctgggca gagatcccgg cgagagattc     1380

cctctgtctt ttgccagcgc cgagtaccaa gaagccctga gcagaatcac cagcgtggtg     1440

cagcagcacc aagaggctct ggttccagct cagccccagc tgaacgtgtg taattgggcc     1500

gtgatgaact gggcccctcc tggatgtgaa aagctgggca agtgtctgac ccctcctgag     1560

agcatcccca agaaatgcct gtggtcccac tga                                  1593


<210>  6
<211>  460
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TBG promoter

<400>  6
gggctggaag ctacctttga catcatttcc tctgcgaatg catgtataat ttctacagaa       60

cctattagaa aggatcaccc agcctctgct tttgtacaac tttcccttaa aaaactgcca      120

attccactgc tgtttggccc aatagtgaga actttttcct gctgcctctt ggtgcttttg      180

cctatggccc ctattctgcc tgctgaagac actcttgcca gcatggactt aaacccctcc      240

agctctgaca atcctctttc tcttttgttt tacatgaagg gtctggcagc caaagcaatc      300

actcaaagtt caaaccttat cattttttgc tttgttcctc ttggccttgg ttttgtacat      360

cagctttgaa aataccatcc cagggttaat gctggggtta atttataact aagagtgctc      420

tagttttgca atacaggaca tgctataaaa atggaaagat                            460


<210>  7
<211>  130
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5-prime ITR

<400>  7
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct                                                             130


<210>  8
<211>  130
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  3-prime ITR

<400>  8
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag                                                             130


<210>  9
<211>  127
<212>  DNA
<213>  Rabbit

<220>
<223>  Rabbit globin poly A

<400>  9
gatctttttc cctctgccaa aaattatggg gacatcatga agccccttga gcatctgact       60

tctggctaat aaaggaaatt tattttcatt gcaatagtgt gttggaattt tttgtgtctc      120

tcactcg                                                                127


<210>  10
<211>  133
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  beta-globin/Ig-Intron (chimeric intron)

<400>  10
gtaagtatca aggttacaag acaggtttaa ggagaccaat agaaactggg cttgtcgaga       60

cagagaagac tcttgcgttt ctgataggca cctattggtc ttactgacat ccactttgcc      120

tttctctcca cag                                                         133


<210>  11
<211>  100
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Alpha mic/bik enhancer  

<400>  11
aggttaattt ttaaaaagca gtcaaaagtc caagtggccc ttggcagcat ttactctctc       60

tgtttgctct ggttaataat ctcaggagca caaacattcc                            100


<210>  12
<211>  1569
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  GALNS (codon optimized and CpG depleted)

<400>  12
atggctgctg tggtggctgc tacaagatgg tggcaactgc tgctggtgct gtctgcagct       60

ggaatgggag cttctggtgc ccctcagcct cctaatatcc tgctgctgct gatggatgac      120

atgggctggg gagatctggg agtgtatggg gagcctagca gagagacacc caacctggat      180

agaatggctg cagagggcct gctgttcccc aacttctact ctgccaatcc tctgtgcagc      240

ccctctagag ctgcactgct tacaggcaga ctgcccatca gaaatggctt ctacaccaca      300

aatgcccatg ccagaaatgc ctacacaccc caagagatag ttggaggcat ccctgactct      360

gaacagctgc tgcctgagct gctgaagaaa gctggctatg tgtccaagat agttggcaag      420

tggcacctgg gccacagacc tcagtttcac cctctgaaac atggctttga tgagtggttt      480

ggcagcccca actgccactt tggcccctat gataacaagg ccagacctaa catccctgtg      540

tacagagact gggagatggt tggaaggtac tatgaagagt tccccatcaa cctgaaaaca      600

ggggaagcca atctgaccca gatctacctg caagaggccc tggacttcat caagagacag      660

gccagacacc atcctttctt tctgtactgg gctgttgatg ccacacatgc ccctgtgtat      720

gccagcaagc cttttctggg caccagccag aggggcagat atggggatgc tgtcagagaa      780

attgatgaca gcattggcaa gatcctggaa ctgctgcagg acctgcatgt ggctgacaac      840

acctttgtgt tcttcacctc tgacaatggg gcagccctga tctctgcccc tgagcaaggt      900

ggcagcaatg gcccatttct gtgtggcaag cagaccacct ttgaaggtgg catgagagag      960

cctgctctgg cctggtggcc tggacatgtt acagctggac aagtgtctca ccagctgggc     1020

agcatcatgg acctgtttac cacatctctg gccctggctg gactgacccc tccatctgat     1080

agagccattg atggcctgaa cctgctgcct acacttctgc agggcagact gatggacaga     1140

cccatcttct actacagagg tgacaccctg atggctgcca cactgggaca gcacaaggcc     1200

cacttttgga cctggaccaa cagctgggag aacttcagac agggcattga tttctgccct     1260

ggccagaatg tgtctggggt caccactcac aacctggaag atcacaccaa gctgcccctc     1320

atcttccacc tgggaagaga tcctggggag agattccctc tgagctttgc ctctgctgag     1380

taccaagaag ccctgagcag aatcacatct gtggtgcagc agcatcaaga ggctctggtt     1440

ccagctcagc cccagctgaa tgtgtgcaac tgggcagtga tgaattgggc cccacctggc     1500

tgtgaaaagc tgggcaaatg tctgacccca cctgagagca tccctaaaaa gtgcctgtgg     1560

tcccactga                                                             1569


<210>  13
<211>  969
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LSPX1 Promoter

<400>  13
aggttaattt ttaaaaagca gtcaaaagtc caagtggccc ttggcagcat ttactctctc       60

tgtttgctct ggttaataat ctcaggagca caaacattcc agatccaggt taatttttaa      120

aaagcagtca aaagtccaag tggcccttgg cagcatttac tctctctgtt tgctctggtt      180

aataatctca ggagcacaaa cattccagat ccggcgcgcc agggctggaa gctacctttg      240

tctagaaggc tcagaggcac acaggagttt ctgggctcac cctgccccct tccaacccct      300

cagttcccat cctccagcag ctgtttgtgt gctgcctctg aagtccacac tgaacaaact      360

tcagcctact catgtcccta aaatgggcaa acattgcaag cagcaaacag caaacacaca      420

gccctccctg cctgctgacc ttggagctgg ggcagaggtc agagacctct ctgggcccat      480

gccacctcca acatccactc gaccccttgg aatttcggtg gagaggagca gaggttgtcc      540

tggcgtggtt taggtagtgt gagaggggta cccggggatc ttgctaccag tggaacagcc      600

actaaggatt ctgcagtgag agcagagggc cagctaagtg gtactctccc agagactgtc      660

tgactcacgc caccccctcc accttggaca caggacgctg tggtttctga gccaggtaca      720

atgactcctt tcggtaagtg cagtggaagc tgtacactgc ccaggcaaag cgtccgggca      780

gcgtaggcgg gcgactcaga tcccagccag tggacttagc ccctgtttgc tcctccgata      840

actggggtga ccttggttaa tattcaccag cagcctcccc cgttgcccct ctggatccac      900

tgcttaaata cggacgagga cagggccctg tctcctcagc ttcaggcacc accactgacc      960

tgggacagt                                                              969


<210>  14
<211>  1050
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LSPX2 Promoter

<400>  14
aggctcagag gcacacagga gtttctgggc tcaccctgcc cccttccaac ccctcagttc       60

ccatcctcca gcagctgttt gtgtgctgcc tctgaagtcc acactgaaca aacttcagcc      120

tactcatgtc cctaaaatgg gcaaacattg caagcagcaa acagcaaaca cacagccctc      180

cctgcctgct gaccttggag ctggggcaga ggtcagagac ctctctgggc ccatgccacc      240

tccaacatcc actcgacccc ttggaatttc ggtggagagg agcagaggtt gtcctggcgt      300

ggtttaggta gtgtgagagg gtctagaagg ctcagaggca cacaggagtt tctgggctca      360

ccctgccccc ttccaacccc tcagttccca tcctccagca gctgtttgtg tgctgcctct      420

gaagtccaca ctgaacaaac ttcagcctac tcatgtccct aaaatgggca aacattgcaa      480

gcagcaaaca gcaaacacac agccctccct gcctgctgac cttggagctg gggcagaggt      540

cagagacctc tctgggccca tgccacctcc aacatccact cgaccccttg gaatttcggt      600

ggagaggagc agaggttgtc ctggcgtggt ttaggtagtg tgagaggggt acccggggat      660

cttgctacca gtggaacagc cactaaggat tctgcagtga gagcagaggg ccagctaagt      720

ggtactctcc cagagactgt ctgactcacg ccaccccctc caccttggac acaggacgct      780

gtggtttctg agccaggtac aatgactcct ttcggtaagt gcagtggaag ctgtacactg      840

cccaggcaaa gcgtccgggc agcgtaggcg ggcgactcag atcccagcca gtggacttag      900

cccctgtttg ctcctccgat aactggggtg accttggtta atattcacca gcagcctccc      960

ccgttgcccc tctggatcca ctgcttaaat acggacgagg acagggccct gtctcctcag     1020

cttcaggcac caccactgac ctgggacagt                                      1050


<210>  15
<211>  1089
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LTP1 Promoter

<400>  15
aggttaattt ttaaaaagca gtcaaaagtc caagtggccc ttggcagcat ttactctctc       60

tgtttgctct ggttaataat ctcaggagca caaacattcc agatccaggt taatttttaa      120

aaagcagtca aaagtccaag tggcccttgg cagcatttac tctctctgtt tgctctggtt      180

aataatctca ggagcacaaa cattccagat ccggcgcgcc agggctggaa gctacctttg      240

acatcatttc ctctgcgaat gcatgtataa tttctacaga acctattaga aaggatcacc      300

cagcctctgc ttttgtacaa ctttccctta aaaaactgcc aattccactg ctgtttggcc      360

caatagtgag aactttttcc tgctgcctct tggtgctttt gcctatggcc cctattctgc      420

ctgctgaaga cactcttgcc agcatggact taaacccctc cagctctgac aatcctcttt      480

ctcttttgtt ttacatgaag ggtctggcag ccaaagcaat cactcaaagt tcaaacctta      540

tcattttttg ctttgttcct cttggccttg gttttgtaca tcagctttga aaataccatc      600

ccagggttaa tgctggggtt aatttataac taagagtgct ctagttttgc aatacaggac      660

atgctataaa aatggaaaga tgttgctttc tgagaggatc ttgctaccag tggaacagcc      720

actaaggatt ctgcagtgag agcagagggc cagctaagtg gtactctccc agagactgtc      780

tgactcacgc caccccctcc accttggaca caggacgctg tggtttctga gccaggtaca      840

gtgactcctt tcggtaagtg cagtggaagc tgtacactgc ccaggcaaag cgtccgggca      900

gcgtaggcgg gcgactcaga tcccagccag tggacttagc ccctgtttgc tcctccgata      960

actggggtga ccttggttaa tattcaccag cagcctcccc cgttgcccct ctggatccac     1020

tgcttaaata cggacgagga cagggccctg tctcctcagc ttcaggcacc accactgacc     1080

tgggacagt                                                             1089


<210>  16
<211>  1579
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LMTP6 Promoter

<400>  16
aggctcagag gcacacagga gtttctgggc tcaccctgcc cccttccaac ccctcagttc       60

ccatcctcca gcagctgttt gtgtgctgcc tctgaagtcc acactgaaca aacttcagcc      120

tactcatgtc cctaaaatgg gcaaacattg caagcagcaa acagcaaaca cacagccctc      180

cctgcctgct gaccttggag ctggggcaga ggtcagagac ctctctgggc ccatgccacc      240

tccaacatcc actcgacccc ttggaatttc ggtggagagg agcagaggtt gtcctggcgt      300

ggtttaggta gtgtgagagg gccactacgg gtttaggctg cccatgtaag gaggcaaggc      360

ctggggacac ccgagatgcc tggttataat taacccagac atgtggctgc cccccccccc      420

cccaacacct gctgcctcta aaaataaccc tgtccctggt ggatcccact acgggtttag      480

gctgcccatg taaggaggca aggcctgggg acacccgaga tgcctggtta taattaaccc      540

agacatgtgg ctgccccccc cccccccaac acctgctgcc tctaaaaata accctgtccc      600

tggtggatcc cactacgggt ttaggctgcc catgtaagga ggcaaggcct ggggacaccc      660

gagatgcctg gttataatta acccagacat gtggctgccc cccccccccc caacacctgc      720

tgcctctaaa aataaccctg tccctggtgg atcccctgca tgcgaagatc ttcgaacaag      780

gctgtggggg actgagggca ggctgtaaca ggcttggggg ccagggctta tacgtgcctg      840

ggactcccaa agtattactg ttccatgttc ccggcgaagg gccagctgtc ccccgccagc      900

tagactcagc acttagttta ggaaccagtg agcaagtcag cccttggggc agcccataca      960

aggccatggg gctgggcaag ctgcacgcct gggtccgggg tgggcacggt gcccgggcaa     1020

cgagctgaaa gctcatctgc tctcaggggc ccctccctgg ggacagcccc tcctggctag     1080

tcacaccctg taggctcctc tatataaccc aggggcacag gggctgccct cattctacca     1140

ccacctccac agcacagaca gacactcagg agccagccag cgtcgagatc ttgctaccag     1200

tggaacagcc actaaggatt ctgcagtgag agcagagggc cagctaagtg gtactctccc     1260

agagactgtc tgactcacgc caccccctcc accttggaca caggacgctg tggtttctga     1320

gccaggtaca gtgactcctt tcggtaagtg cagtggaagc tgtacactgc ccaggcaaag     1380

cgtccgggca gcgtaggcgg gcgactcaga tcccagccag tggacttagc ccctgtttgc     1440

tcctccgata actggggtga ccttggttaa tattcaccag cagcctcccc cgttgcccct     1500

ctggatccac tgcttaaata cggacgagga cagggccctg tctcctcagc ttcaggcacc     1560

accactgacc tgggacagt                                                  1579


<210>  17
<211>  969
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LBTP1 Promoter

<400>  17
aggttaattt ttaaaaagca gtcaaaagtc caagtggccc ttggcagcat ttactctctc       60

tgtttgctct ggttaataat ctcaggagca caaacattcc agatccaggt taatttttaa      120

aaagcagtca aaagtccaag tggcccttgg cagcatttac tctctctgtt tgctctggtt      180

aataatctca ggagcacaaa cattccagat ccggcgcgcc agggctggaa gctacctttg      240

tctagaaggc tcagaggcac acaggagttt ctgggctcac cctgccccct tccaacccct      300

cagttcccat cctccagcag ctgtttgtgt gctgcctctg aagtccacac tgaacaaact      360

tcagcctact catgtcccta aaatgggcaa acattgcaag cagcaaacag caaacacaca      420

gccctccctg cctgctgacc ttggagctgg ggcagaggtc agagacctct ctgggcccat      480

gccacctcca acatccactc gaccccttgg aatttcggtg gagaggagca gaggttgtcc      540

tggcgtggtt taggtagtgt gagaggggta cccggggatc ttgctaccag tggaacagcc      600

actaaggatt ctgcagtgag agcagagggc cagctaagtg gtactctccc agagactgtc      660

tgactcacgc caccccctcc accttggaca caggacgctg tggtttctga gccaggtaca      720

atgactcctt tcggtaagtg cagtggaagc tgtacactgc ccaggcaaag cgtccgggca      780

gcgtaggcgg gcgactcaga tcccagccag tggacttagc ccctgtttgc tcctccgata      840

actggggtga ccttggttaa tattcaccag cagcctcccc cgttgcccct ctggatccac      900

tgcttaaata cggacgagga cagggccctg tctcctcagc ttcaggcacc accactgacc      960

tgggacagt                                                              969


<210>  18
<211>  2074
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LBTP2 Promoter

<400>  18
aggctcagag gcacacagga gtttctgggc tcaccctgcc cccttccaac ccctcagttc       60

ccatcctcca gcagctgttt gtgtgctgcc tctgaagtcc acactgaaca aacttcagcc      120

tactcatgtc cctaaaatgg gcaaacattg caagcagcaa acagcaaaca cacagccctc      180

cctgcctgct gaccttggag ctggggcaga ggtcagagac ctctctgggc ccatgccacc      240

tccaacatcc actcgacccc ttggaatttc ggtggagagg agcagaggtt gtcctggcgt      300

ggtttaggta gtgtgagagg gcacacatac acgaacacac atacatatac atacattcac      360

atatatgcac acacatacac atatacacgc atacacgtac acacaaatgc acactcacac      420

atgcacacac atacataata tacacactct cacacatgca catacacaca tacatacaca      480

tacatgtgca tgcacacaca caaatacaca tgcatacatc cacattcaca cagatgcaga      540

cacaaatgca cacacacaca cacacacaca cacacacaca cacacacaca cgcacactgc      600

caccctgaac tagtggtggc taaatgaaca ataagtctcc atcaccagct tggggggagg      660

taggtggtag tgtaggtgcc cccattgtgt gatcatgttc attgtatgag tttgtctgtg      720

ttcattcatc atagtgacag tccccatgtg ggtagcagag agtacgtgtg catgcatcat      780

ctccgtgttt gctcatgagt gtgtatgtca gtgtgttcca gtctttctgt gtgagtgtcg      840

tccccaatcc cccatccccc cccccagatc tctaattagt ggtttggggt ttgttccttt      900

tccctcctgt tcctttcctc agcagcgcgg cagcagcggc ggcagcctcg gtggtagcag      960

cagcagcagc agcagcagca gcagcagcag cagcagcagc agcaacagaa gctgccgcgc     1020

cgctgagtag cagcaaggac tccgagtcaa gagtaggatt gtaggattgg atctgagtgg     1080

gaacaagagt gagctggcct gagagaggag cagatgcctc ccagcgccct caggccaccc     1140

attgccagta atcttcaagc cagacctctt gagaggagac gggacagcca accctagcct     1200

acccaggtac agacactggg cagttctggg ggactgccca cagatgccta ttggattcct     1260

ggggtatgta ggactcccgg gtctaccagc ccttttcacc tttccccata gcacccccaa     1320

ggaagctctg acaacttgcc catattcctg tttcccaccc gtcccctggg cacccccttt     1380

tcttctctcc ctcccagatc ccttctttgg ggagctcagc aaatggagca ggaaatttgg     1440

accctctgcc tccctctctc gccttcctca ttggatccgg agtcttctcc gctgggaaag     1500

ctgtaattag agggtggatc cctacagaca gagagcagcc cccccacccc caccccccag     1560

tcccttctaa ctttagatct cttctctccc attctcccat tctccctccc tctcccttct     1620

ccctctccca ctggctcctc ggttctctcc atctgcctga ctccttggga cccggtcccc     1680

agatcttgct accagtggaa cagccactaa ggattctgca gtgagagcag agggccagct     1740

aagtggtact ctcccagaga ctgtctgact cacgccaccc cctccacctt ggacacagga     1800

cgctgtggtt tctgagccag gtacagtgac tcctttcggt aagtgcagtg gaagctgtac     1860

actgcccagg caaagcgtcc gggcagcgta ggcgggcgac tcagatccca gccagtggac     1920

ttagcccctg tttgctcctc cgataactgg ggtgaccttg gttaatattc accagcagcc     1980

tcccccgttg cccctctgga tccactgctt aaatacggac gaggacaggg ccctgtctcc     2040

tcagcttcag gcaccaccac tgacctggga cagt                                 2074


<210>  19
<211>  321
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ApoE Hepatic Control Region containing ApoE Enhancer

<400>  19
aggctcagag gcacacagga gtttctgggc tcaccctgcc cccttccaac ccctcagttc       60

ccatcctcca gcagctgttt gtgtgctgcc tctgaagtcc acactgaaca aacttcagcc      120

tactcatgtc cctaaaatgg gcaaacattg caagcagcaa acagcaaaca cacagccctc      180

cctgcctgct gaccttggag ctggggcaga ggtcagagac ctctctgggc ccatgccacc      240

tccaacatcc actcgacccc ttggaatttc ggtggagagg agcagaggtt gtcctggcgt      300

ggtttaggta gtgtgagagg g                                                321


<210>  20
<211>  154
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ApoE Enhancer

<400>  20
gctgtttgtg tgctgcctct gaagtccaca ctgaacaaac ttcagcctac tcatgtccct       60

aaaatgggca aacattgcaa gcagcaaaca gcaaacacac agccctccct gcctgctgac      120

cttggagctg gggcagaggt cagagacctc tctg                                  154


<210>  21
<211>  393
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hAAT Promoter

<400>  21
gatcttgcta ccagtggaac agccactaag gattctgcag tgagagcaga gggccagcta       60

agtggtactc tcccagagac tgtctgactc acgccacccc ctccaccttg gacacaggac      120

gctgtggttt ctgagccagg tacaatgact cctttcggta agtgcagtgg aagctgtaca      180

ctgcccaggc aaagcgtccg ggcagcgtag gcgggcgact cagatcccag ccagtggact      240

tagcccctgt ttgctcctcc gataactggg gtgaccttgg ttaatattca ccagcagcct      300

cccccgttgc ccctctggat ccactgctta aatacggacg aggacagggc cctgtctcct      360

cagcttcagg caccaccact gacctgggac agt                                   393


<210>  22
<211>  393
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hAAT(delta-ATG) Promoter

<400>  22
gatcttgcta ccagtggaac agccactaag gattctgcag tgagagcaga gggccagcta       60

agtggtactc tcccagagac tgtctgactc acgccacccc ctccaccttg gacacaggac      120

gctgtggttt ctgagccagg tacagtgact cctttcggta agtgcagtgg aagctgtaca      180

ctgcccaggc aaagcgtccg ggcagcgtag gcgggcgact cagatcccag ccagtggact      240

tagcccctgt ttgctcctcc gataactggg gtgaccttgg ttaatattca ccagcagcct      300

cccccgttgc ccctctggat ccactgctta aatacggacg aggacagggc cctgtctcct      360

cagcttcagg caccaccact gacctgggac agt                                   393


<210>  23
<211>  1360
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Sp7/Osx 

<400>  23
cacacataca cgaacacaca tacatataca tacattcaca tatatgcaca cacatacaca       60

tatacacgca tacacgtaca cacaaatgca cactcacaca tgcacacaca tacataatat      120

acacactctc acacatgcac atacacacat acatacacat acatgtgcat gcacacacac      180

aaatacacat gcatacatcc acattcacac agatgcagac acaaatgcac acacacacac      240

acacacacac acacacacac acacacacac gcacactgcc accctgaact agtggtggct      300

aaatgaacaa taagtctcca tcaccagctt ggggggaggt aggtggtagt gtaggtgccc      360

ccattgtgtg atcatgttca ttgtatgagt ttgtctgtgt tcattcatca tagtgacagt      420

ccccatgtgg gtagcagaga gtacgtgtgc atgcatcatc tccgtgtttg ctcatgagtg      480

tgtatgtcag tgtgttccag tctttctgtg tgagtgtcgt ccccaatccc ccatcccccc      540

ccccagatct ctaattagtg gtttggggtt tgttcctttt ccctcctgtt cctttcctca      600

gcagcgcggc agcagcggcg gcagcctcgg tggtagcagc agcagcagca gcagcagcag      660

cagcagcagc agcagcagca gcaacagaag ctgccgcgcc gctgagtagc agcaaggact      720

ccgagtcaag agtaggattg taggattgga tctgagtggg aacaagagtg agctggcctg      780

agagaggagc agatgcctcc cagcgccctc aggccaccca ttgccagtaa tcttcaagcc      840

agacctcttg agaggagacg ggacagccaa ccctagccta cccaggtaca gacactgggc      900

agttctgggg gactgcccac agatgcctat tggattcctg gggtatgtag gactcccggg      960

tctaccagcc cttttcacct ttccccatag cacccccaag gaagctctga caacttgccc     1020

atattcctgt ttcccacccg tcccctgggc accccctttt cttctctccc tcccagatcc     1080

cttctttggg gagctcagca aatggagcag gaaatttgga ccctctgcct ccctctctcg     1140

ccttcctcat tggatccgga gtcttctccg ctgggaaagc tgtaattaga gggtggatcc     1200

ctacagacag agagcagccc ccccaccccc accccccagt cccttctaac tttagatctc     1260

ttctctccca ttctcccatt ctccctccct ctcccttctc cctctcccac tggctcctcg     1320

gttctctcca tctgcctgac tccttgggac ccggtcccca                           1360


<210>  24
<211>  756
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  minimal Sp7/Osx 

<400>  24
cgcggcagca gcggcggcag cctcggtggt agcagcagca gcagcagcag cagcagcagc       60

agcagcagca gcagcagcaa cagaagctgc cgcgccgctg agtagcagca aggactccga      120

gtcaagagta ggattgtagg attggatctg agtgggaaca agagtgagct ggcctgagag      180

aggagcagat gcctcccagc gccctcaggc cacccattgc cagtaatctt caagccagac      240

ctcttgagag gagacgggac agccaaccct agcctaccca ggtacagaca ctgggcagtt      300

ctgggggact gcccacagat gcctattgga ttcctggggt atgtaggact cccgggtcta      360

ccagcccttt tcacctttcc ccatagcacc cccaaggaag ctctgacaac ttgcccatat      420

tcctgtttcc cacccgtccc ctgggcaccc ccttttcttc tctccctccc agatcccttc      480

tttggggagc tcagcaaatg gagcaggaaa tttggaccct ctgcctccct ctctcgcctt      540

cctcattgga tccggagtct tctccgctgg gaaagctgta attagagggt ggatccctac      600

agacagagag cagccccccc acccccaccc cccagtccct tctaacttta gatctcttct      660

ctcccattct cccattctcc ctccctctcc cttctccctc tcccactggc tcctcggttc      720

tctccatctg cctgactcct tgggacccgg tcccca                                756


<210>  25
<211>  56
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  beta-globin polyadenylation signal

<400>  25
aataaaggaa atttattttc attgcaatag tgtgttggaa ttttttgtgt ctctca           56


<210>  26
<211>  736
<212>  PRT
<213>  AAV9

<220>
<223>  AAV9 capsid protein  

<400>  26

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>  27
<211>  1569
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hGALNScoV2

<400>  27
atggctgctg tggtggctgc tacaagatgg tggcaactgc tgctggtgct gtctgcagct       60

ggaatgggag cttctggtgc ccctcagcct cctaatatcc tgctgctgct gatggatgac      120

atgggctggg gagatctggg agtgtatggg gagcctagca gagagacacc caacctggat      180

agaatggctg cagagggcct gctgttcccc aacttctact ctgccaatcc tctgtgcagc      240

ccctctagag ctgcactgct tacaggcaga ctgcccatca gaaatggctt ctacaccaca      300

aatgcccatg ccagaaatgc ctacacaccc caagagatag ttggaggcat ccctgactct      360

gaacagctgc tgcctgagct gctgaagaaa gctggctatg tgtccaagat agttggcaag      420

tggcacctgg gccacagacc tcagtttcac cctctgaaac atggctttga tgagtggttt      480

ggcagcccca actgccactt tggcccctat gataacaagg ccagacctaa catccctgtg      540

tacagagact gggagatggt tggaaggtac tatgaagagt tccccatcaa cctgaaaaca      600

ggggaagcca atctgaccca gatctacctg caagaggccc tggacttcat caagagacag      660

gccagacacc atcctttctt tctgtactgg gctgttgatg ccacacatgc ccctgtgtat      720

gccagcaagc cttttctggg caccagccag aggggcagat atggggatgc tgtcagagaa      780

attgatgaca gcattggcaa gatcctggaa ctgctgcagg acctgcatgt ggctgacaac      840

acctttgtgt tcttcacctc tgacaatggg gcagccctga tctctgcccc tgagcaaggt      900

ggcagcaatg gcccatttct gtgtggcaag cagaccacct ttgaaggtgg catgagagag      960

cctgctctgg cctggtggcc tggacatgtt acagctggac aagtgtctca ccagctgggc     1020

agcatcatgg acctgtttac cacatctctg gccctggctg gactgacccc tccatctgat     1080

agagccattg atggcctgaa cctgctgcct acacttctgc agggcagact gatggacaga     1140

cccatcttct actacagagg tgacaccctg atggctgcca cactgggaca gcacaaggcc     1200

cacttttgga cctggaccaa cagctgggag aacttcagac agggcattga tttctgccct     1260

ggccagaatg tgtctggggt caccactcac aacctggaag atcacaccaa gctgcccctc     1320

atcttccacc tgggaagaga tcctggggag agattccctc tgagctttgc ctctgctgag     1380

taccaagaag ccctgagcag aatcacatct gtggtgcagc agcatcaaga ggctctggtt     1440

ccagctcagc cccagctgaa tgtgtgcaac tgggcagtga tgaattgggc cccacctggc     1500

tgtgaaaagc tgggcaaatg tctgacccca cctgagagca tccctaaaaa gtgcctgtgg     1560

tcccactga                                                             1569


<210>  28
<211>  1676
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG promoter

<400>  28
gacattgatt attgactagt tattaatagt aatcaattac ggggtcatta gttcatagcc       60

catatatgga gttccgcgtt acataactta cggtaaatgg cccgcctggc tgaccgccca      120

acgacccccg cccattgacg tcaataatga cgtatgttcc catagtaacg ccaataggga      180

ctttccattg acgtcaatgg gtggagtatt tacggtaaac tgcccacttg gcagtacatc      240

aagtgtatca tatgccaagt acgcccccta ttgacgtcaa tgacggtaaa tggcccgcct      300

ggcattatgc ccagtacatg accttatggg actttcctac ttggcagtac atctacgtat      360

tagtcatcgc tattaccatg gtcgaggtga gccccacgtt ctgcttcact ctccccatct      420

cccccccctc cccaccccca attttgtatt tatttatttt ttaattattt tgtgcagcga      480

tgggggcggg gggggggggg gggcgcgcgc caggcggggc ggggcggggc gaggggcggg      540

gcggggcgag gcggagaggt gcggcggcag ccaatcagag cggcgcgctc cgaaagtttc      600

cttttatggc gaggcggcgg cggcggcggc cctataaaaa gcgaagcgcg cggcgggcgg      660

gagtcgctgc gcgctgcctt cgccccgtgc cccgctccgc cgccgcctcg cgccgcccgc      720

cccggctctg actgaccgcg ttactcccac aggtgagcgg gcgggacggc ccttctcctc      780

cgggctgtaa ttagcgcttg gtttaatgac ggcttgtttc ttttctgtgg ctgcgtgaaa      840

gccttgaggg gctccgggag ggccctttgt gcggggggag cggctcgggg ggtgcgtgcg      900

tgtgtgtgtg cgtggggagc gccgcgtgcg gctccgcgct gcccggcggc tgtgagcgct      960

gcgggcgcgg cgcggggctt tgtgcgctcc gcagtgtgcg cgaggggagc gcggccgggg     1020

gcggtgcccc gcggtgcggg gggggctgcg aggggaacaa aggctgcgtg cggggtgtgt     1080

gcgtgggggg gtgagcaggg ggtgtgggcg cgtcggtcgg gctgcaaccc cccctgcacc     1140

cccctccccg agttgctgag cacggcccgg cttcgggtgc ggggctccgt acggggcgtg     1200

gcgcggggct cgccgtgccg ggcggggggt ggcggcaggt gggggtgccg ggcggggcgg     1260

ggccgcctcg ggccggggag ggctcggggg aggggcgcgg cggcccccgg agcgccggcg     1320

gctgtcgagg cgcggcgagc cgcagccatt gccttttatg gtaatcgtgc gagagggcgc     1380

agggacttcc tttgtcccaa atctgtgcgg agccgaaatc tgggaggcgc cgccgcaccc     1440

cctctagcgg gcgcggggcg aagcggtgcg gcgccggcag gaaggaaatg ggcggggagg     1500

gccttcgtgc gtcgccgcgc cgccgtcccc ttctccctct ccagcctcgg ggctgtccgc     1560

ggggggacgg ctgccttcgg gggggacggg gcagggcggg gttcggcttc tggcgtgtga     1620

ccggcggctc tagagcctct gctaaccatg ttcatgcctt cttctttttc ctacag         1676


<210>  29
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  forward primer

<400>  29
gccaaaaatt atggggacat                                                   20


<210>  30
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  reverse primer

<400>  30
attccaacac actattgcaa tg                                                22


<210>  31
<211>  27
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  probe

<400>  31
atgaagcccc ttgagcatct gacttct                                           27


<210>  32
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  D8 sequence

<400>  32
gacgacgatg atgacgatga cgac                                              24


