                         SEQUENCE LISTING

<110>  NATIONWIDE CHILDREN'S HOSPITAL, INC.
       KASPAR, Brian K.
 
<120>  INTRATHECAL DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9

<130>  28335/47099PCT

<150>  US-61/678,458
<151>  2012-08-01

<160>  1     

<170>  PatentIn version 3.5

<210>  1
<211>  6042
<212>  DNA
<213>  adeno-associated virus 9


<220>
<221>  misc_feature
<222>  (980)..(3336)
<223>  Vector genome insert

<220>
<221>  misc_feature
<222>  (980)..(1085)
<223>  Mutated ITR

<220>
<221>  misc_feature
<222>  (1132)..(1411)
<223>  CMV enhancer

<220>
<221>  misc_feature
<222>  (1418)..(1687)
<223>  Chicken beta-Actin promoter

<220>
<221>  misc_feature
<222>  (1753)..(1812)
<223>  Modified SV40 late 16s intron

<220>
<221>  misc_feature
<222>  (1753)..(1783)
<223>  SV40 late 19s intron

<220>
<221>  misc_feature
<222>  (1982)..(2866)
<223>  hSMN

<220>
<221>  misc_feature
<222>  (2970)..(3116)
<223>  BGHpA

<220>
<221>  misc_feature
<222>  (3196)..(3336)
<223>  ITR

<400>  1
gcccaatacg caaaccgcct ctccccgcgc gttggccgat tcattaatgc agctggcgta       60

atagcgaaga ggcccgcacc gatcgccctt cccaacagtt gcgcagcctg aatggcgaat      120

ggcgattccg ttgcaatggc tggcggtaat attgttctgg atattaccag caaggccgat      180

agtttgagtt cttctactca ggcaagtgat gttattacta atcaaagaag tattgcgaca      240

acggttaatt tgcgtgatgg acagactctt ttactcggtg gcctcactga ttataaaaac      300

acttctcagg attctggcgt accgttcctg tctaaaatcc ctttaatcgg cctcctgttt      360

agctcccgct ctgattctaa cgaggaaagc acgttatacg tgctcgtcaa agcaaccata      420

gtacgcgccc tgtagcggcg cattaagcgc ggcgggtgtg gtggttacgc gcagcgtgac      480

cgctacactt gccagcgccc tagcgcccgc tcctttcgct ttcttccctt cctttctcgc      540

cacgttcgcc ggctttcccc gtcaagctct aaatcggggg ctccctttag ggttccgatt      600

tagtgcttta cggcacctcg accccaaaaa acttgattag ggtgatggtt cacgtagtgg      660

gccatcgccc tgatagacgg tttttcgccc tttgacgttg gagtccacgt tctttaatag      720

tggactcttg ttccaaactg gaacaacact caaccctatc tcggtctatt cttttgattt      780

ataagggatt ttgccgattt cggcctattg gttaaaaaat gagctgattt aacaaaaatt      840

taacgcgaat tttaacaaaa tattaacgct tacaatttaa atatttgctt atacaatctt      900

cctgtttttg gggcttttct gattatcaac cggggtacat atgattgaca tgctagtttt      960

acgattaccg ttcatcgccc tgcgcgctcg ctcgctcact gaggccgccc gggcaaagcc     1020

cgggcgtcgg gcgacctttg gtcgcccggc ctcagtgagc gagcgagcgc gcagagaggg     1080

agtggaattc acgcgtggat ctgaattcaa ttcacgcgtg gtacctctgg tcgttacata     1140

acttacggta aatggcccgc ctggctgacc gcccaacgac ccccgcccat tgacgtcaat     1200

aatgacgtat gttcccatag taacgccaat agggactttc cattgacgtc aatgggtgga     1260

gtatttacgg taaactgccc acttggcagt acatcaagtg tatcatatgc caagtacgcc     1320

ccctattgac gtcaatgacg gtaaatggcc cgcctggcat tatgcccagt acatgacctt     1380

atgggacttt cctacttggc agtacatcta ctcgaggcca cgttctgctt cactctcccc     1440

atctcccccc cctccccacc cccaattttg tatttattta ttttttaatt attttgtgca     1500

gcgatggggg cggggggggg gggggggcgc gcgccaggcg gggcggggcg gggcgagggg     1560

cggggcgggg cgaggcggag aggtgcggcg gcagccaatc agagcggcgc gctccgaaag     1620

tttcctttta tggcgaggcg gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg     1680

gcgggagcgg gatcagccac cgcggtggcg gcctagagtc gacgaggaac tgaaaaacca     1740

gaaagttaac tggtaagttt agtctttttg tcttttattt caggtcccgg atccggtggt     1800

ggtgcaaatc aaagaactgc tcctcagtgg atgttgcctt tacttctagg cctgtacgga     1860

agtgttactt ctgctctaaa agctgcggaa ttgtacccgc ggccgatcca ccggtccgga     1920

attcccggga tatcgtcgac ccacgcgtcc gggccccacg ctgcgcaccc gcgggtttgc     1980

tatggcgatg agcagcggcg gcagtggtgg cggcgtcccg gagcaggagg attccgtgct     2040

gttccggcgc ggcacaggcc agagcgatga ttctgacatt tgggatgata cagcactgat     2100

aaaagcatat gataaagctg tggcttcatt taagcatgct ctaaagaatg gtgacatttg     2160

tgaaacttcg ggtaaaccaa aaaccacacc taaaagaaaa cctgctaaga agaataaaag     2220

ccaaaagaag aatactgcag cttccttaca acagtggaaa gttggggaca aatgttctgc     2280

catttggtca gaagacggtt gcatttaccc agctaccatt gcttcaattg attttaagag     2340

agaaacctgt gttgtggttt acactggata tggaaataga gaggagcaaa atctgtccga     2400

tctactttcc ccaatctgtg aagtagctaa taatatagaa cagaatgctc aagagaatga     2460

aaatgaaagc caagtttcaa cagatgaaag tgagaactcc aggtctcctg gaaataaatc     2520

agataacatc aagcccaaat ctgctccatg gaactctttt ctccctccac caccccccat     2580

gccagggcca agactgggac caggaaagcc aggtctaaaa ttcaatggcc caccaccgcc     2640

accgccacca ccaccacccc acttactatc atgctggctg cctccatttc cttctggacc     2700

accaataatt cccccaccac ctcccatatg tccagattct cttgatgatg ctgatgcttt     2760

gggaagtatg ttaatttcat ggtacatgag tggctatcat actggctatt atatgggttt     2820

tagacaaaat caaaaagaag gaaggtgctc acattcctta aattaaggag aaatgctggc     2880

atagagcagc actaaatgac accactaaag aaacgatcag acagatctag aaagcttatc     2940

gataccgtcg actagagctc gctgatcagc ctcgactgtg ccttctagtt gccagccatc     3000

tgttgtttgc ccctcccccg tgccttcctt gaccctggaa ggtgccactc ccactgtcct     3060

ttcctaataa aatgaggaaa ttgcatcgca ttgtctgagt aggtgtcatt ctattctggg     3120

gggtggggtg gggcaggaca gcaaggggga ggattgggaa gacaatagca ggcatgctgg     3180

ggagagatcg atctgaggaa cccctagtga tggagttggc cactccctct ctgcgcgctc     3240

gctcgctcac tgaggccggg cgaccaaagg tcgcccgacg cccgggcttt gcccgggcgg     3300

cctcagtgag cgagcgagcg cgcagagagg gagtggcccc cccccccccc cccccggcga     3360

ttctcttgtt tgctccagac tctcaggcaa tgacctgata gcctttgtag agacctctca     3420

aaaatagcta ccctctccgg catgaattta tcagctagaa cggttgaata tcatattgat     3480

ggtgatttga ctgtctccgg cctttctcac ccgtttgaat ctttacctac acattactca     3540

ggcattgcat ttaaaatata tgagggttct aaaaattttt atccttgcgt tgaaataaag     3600

gcttctcccg caaaagtatt acagggtcat aatgtttttg gtacaaccga tttagcttta     3660

tgctctgagg ctttattgct taattttgct aattctttgc cttgcctgta tgatttattg     3720

gatgttggaa tcgcctgatg cggtattttc tccttacgca tctgtgcggt atttcacacc     3780

gcatatggtg cactctcagt acaatctgct ctgatgccgc atagttaagc cagccccgac     3840

acccgccaac actatggtgc actctcagta caatctgctc tgatgccgca tagttaagcc     3900

agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg ctcccggcat     3960

ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg ttttcaccgt     4020

catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta taggttaatg     4080

tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat gtgcgcggaa     4140

cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg agacaataac     4200

cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa catttccgtg     4260

tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac ccagaaacgc     4320

tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac atcgaactgg     4380

atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt ccaatgatga     4440

gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc gggcaagagc     4500

aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca ccagtcacag     4560

aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc ataaccatga     4620

gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag gagctaaccg     4680

cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa ccggagctga     4740

atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg gcaacaacgt     4800

tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa ttaatagact     4860

ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg gctggctggt     4920

ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt gcagcactgg     4980

ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt caggcaacta     5040

tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag cattggtaac     5100

tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat ttttaattta     5160

aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct taacgtgagt     5220

tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct tgagatcctt     5280

tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca gcggtggttt     5340

gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc agcagagcgc     5400

agataccaaa tactgttctt ctagtgtagc cgtagttagg ccaccacttc aagaactctg     5460

tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct gccagtggcg     5520

ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag gcgcagcggt     5580

cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc tacaccgaac     5640

tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg agaaaggcgg     5700

acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag cttccagggg     5760

gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt gagcgtcgat     5820

ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac gcggcctttt     5880

tacggttcct ggccttttgc tggccttttg ctcacatgtt ctttcctgcg ttatcccctg     5940

attctgtgga taaccgtatt accgcctttg agtgagctga taccgctcgc cgcagccgaa     6000

cgaccgagcg cagcgagtca gtgagcgagg aagcggaaga gc                        6042


