﻿               SEQUENCE LISTING

<110> Centro de Investigaciones Energéticas, Medioambientales
      y Tecnológicas (CIEMAT) et al.

<120> Pyruvate kinase deficiency (PKD) gene editing treatment method

<130> 906 345

<160> 40

<170> BiSSAP 1.3.6

<210> 1
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG1

<400> 1
ctgcgggacc atggaatgag                                                20


<210> 2
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG2

<400> 2
tggggacagg gtggcctact                                                20


<210> 3
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG3

<400> 3
aaaactgctg gtcttatcta                                                20


<210> 4
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG4

<400> 4
agaaaagggg cacacccagt                                                20


<210> 5
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG5

<400> 5
tggtcccgca gccccaggcc                                                20


<210> 6
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG6

<400> 6
ctccctctca ttccatggtc                                                20


<210> 7
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG7

<400> 7
cagccccagg cccacactga                                                20


<210> 8
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG8

<400> 8
ttccatggtc ccgcagcccc                                                20


<210> 9
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG9

<400> 9
cactgaaagc atgtcgatcc                                                20


<210> 10
<211> 20
<212> DNA
<213> Artificial Sequence


<220> 
<223> SG10

<400> 10
aaactgctgg tcttatctaa                                                20


<210> 11
<211> 20
<212> RNA
<213> Artificial Sequence


<220> 
<223> crRNA SG1 sequence (as RNA)

<400> 11
cugcgggacc auggaaugag                                                20


<210> 12
<211> 61
<212> RNA
<213> Artificial Sequence


<220> 
<223> sgRNA SG1 sequence (as RNA)

<400> 12
cugcgggacc auggaaugag guuuuagagc uagaaauagc aaguuaaaua aggcuagucc     60

g                                                                     61


<210> 13
<211> 425
<212> DNA
<213> Artificial Sequence


<220> 
<223> LHA (left homologous arm)

<400> 13
cagagtggtg aaggcactct gcatttcttg gttgagacag agaaaaaaag tggtcagaac      60

tgggtaaccc tccccccacc atattatcac agtgatccct tttgtctttc ttcaggctcc     120

agccccaccc tacagcccct gctccctgga ttcactagag ctaacttcag taaagtacaa     180

agaaaatggg gccatatgac tggccaaaaa aaaaatatct attcacgtgg atgaccagat     240

agtatgaatg gattgaaaat ttatcaggaa aaaaggatga gaggaaatgc caggagatga     300

gggcagagag caggccgttc tgggggaggg attctgtggg gacagggtgg cctactgggt     360

gtgccccttt tctcttctct gtctccctta gataagacca gcagttttgt catcctctcc     420

ctctc                                                                 425


<210> 14
<211> 425
<212> DNA
<213> Artificial Sequence


<220> 
<223> RHA (right homology arm)

<400> 14
gtcccgcagc cccaggccca cactgaaagc atgtcgatcc aggagaacat atcatccctg      60

cagcttcggt catgggtctc taagtcccaa agagacttag caaagtccat cctgattggg     120

gctccaggag gtaagaaggg gagacagaag ccatggaaca taggaggaaa atgagggtga     180

aaactaggag ccagggtgga gggcataaat gatccacatc agccactggc taggtgggtt     240

ttggagagga acgtacgttc ttcagagcct cccgtgtgtt aaattatgga ccctggcctg     300

ggtcttttcc aggccctata ggcaggccag agccacagca tgtaagccac ggggcactcc     360

cgtggttcct ggactctggc ccctggcata cagggcttcc aatggaacag gagacagtgg     420

tgaca                                                                 425


<210> 15
<211> 40
<212> DNA
<213> Artificial Sequence


<220> 
<223> 5'UTR sequence

<400> 15
ttccatggtc ccgcagcccc aggcccacac tgaaagcatg                           40


<210> 16
<211> 1719
<212> DNA
<213> Artificial Sequence


<220> 
<223> coRPK cDNA

<400> 16
agcatccagg aaaatatcag ctctctgcag ctgcggtcct gggtgtccaa gagccagaga      60

gacctggcca agagcatcct gatcggagcc cctggcggac cagccggata cctgagaagg     120

gctagcgtgg cccagctgac ccaggaactg ggcaccgcct ttttccagca gcagcagctg     180

ccagccgcca tggccgacac ctttctggaa cacctgtgcc tgctggacat cgactctgag     240

cccgtggccg ccagaagcac cagcatcatt gccaccatcg gccctgccag cagaagcgtg     300

gagcggctga aagagatgat caaggccggc atgaatatcg cccggctgaa cttctcccac     360

ggcagccacg agtaccacgc agagagcatt gccaacgtcc gggaggccgt ggagagcttt     420

gccggcagcc ccctgagcta cagacccgtg gccattgccc tggacaccaa gggccccgag     480

atcagaacag gaattctgca gggagggcct gagagcgagg tggagctggt gaagggcagc     540

caagtgctgg tgaccgtgga ccccgccttc agaaccagag gcaacgccaa cacagtgtgg     600

gtggactacc ccaacatcgt gcgggtggtg cctgtgggcg gcagaatcta catcgacgac     660

ggcctgatca gcctggtggt gcagaagatc ggacctgagg gcctggtgac ccaggtcgag     720

aatggcggcg tgctgggcag cagaaagggc gtgaatctgc caggcgccca ggtggacctg     780

cctggcctgt ctgagcagga cgtgagagac ctgagatttg gcgtggagca cggcgtggac     840

atcgtgttcg ccagcttcgt gcggaaggcc tctgatgtgg ccgccgtgag agccgctctg     900

ggccctgaag gccacggcat caagatcatc agcaagatcg agaaccacga gggcgtgaag     960

cggttcgacg agatcctgga agtgtccgac ggcatcatgg tggccagagg cgacctgggc    1020

atcgagatcc ccgccgagaa ggtgttcctg gcccagaaaa tgatgatcgg acggtgcaac    1080

ctggccggca aacctgtggt gtgcgccacc cagatgctgg aaagcatgat caccaagccc    1140

agacccacca gagccgagac aagcgacgtg gccaacgccg tgctggatgg cgctgactgc    1200

atcatgctgt ccggcgagac agccaagggc aacttccccg tggaggccgt gaagatgcag    1260

cacgccattg ccagagaagc cgaggccgcc gtgtaccacc ggcagctgtt cgaggaactg    1320

cggagagccg cccctctgag cagagatccc accgaagtga ccgccatcgg agccgtggaa    1380

gccgccttca agtgctgcgc cgctgcaatc atcgtgctga ccaccacagg cagaagcgcc    1440

cagctgctgt ccagatacag acccagagcc gccgtgatcg ccgtgacaag atccgcccag    1500

gccgctagac aggtccacct gtgcagaggc gtgttccccc tgctgtaccg ggagcctccc    1560

gaggccatct gggccgacga cgtggacaga cgggtgcagt tcggcatcga gagcggcaag    1620

ctgcggggct tcctgagagt gggcgacctg gtgatcgtgg tgacaggctg gcggcctggc    1680

agcggctaca ccaacatcat gagggtgctg tccatcagc                           1719


<210> 17
<211> 27
<212> DNA
<213> Artificial Sequence


<220> 
<223> FLAG-Tag

<400> 17
gactacaaag acgatgacga taaatga                                        27


<210> 18
<211> 225
<212> DNA
<213> Artificial Sequence


<220> 
<223> bGH poly(A) signal

<400> 18
ctgtgccttc tagttgccag ccatctgttg tttgcccctc ccccgtgcct tccttgaccc     60

tggaaggtgc cactcccact gtcctttcct aataaaatga ggaaattgca tcgcattgtc    120

tgagtaggtg tcattctatt ctggggggtg gggtggggca ggacagcaag ggggaggatt    180

gggaagacaa tagcaggcat gctggggatg cggtgggctc tatgg                    225


<210> 19
<211> 2884
<212> DNA
<213> Artificial Sequence


<220> 
<223> LHA-5'UTR-coRPK-FLAG-bGH poly(A)-RHA

<400> 19
cagagtggtg aaggcactct gcatttcttg gttgagacag agaaaaaaag tggtcagaac      60

tgggtaaccc tccccccacc atattatcac agtgatccct tttgtctttc ttcaggctcc     120

agccccaccc tacagcccct gctccctgga ttcactagag ctaacttcag taaagtacaa     180

agaaaatggg gccatatgac tggccaaaaa aaaaatatct attcacgtgg atgaccagat     240

agtatgaatg gattgaaaat ttatcaggaa aaaaggatga gaggaaatgc caggagatga     300

gggcagagag caggccgttc tgggggaggg attctgtggg gacagggtgg cctactgggt     360

gtgccccttt tctcttctct gtctccctta gataagacca gcagttttgt catcctctcc     420

ctctcttcca tggtcccgca gccccaggcc cacactgaaa gcatgagcat ccaggaaaat     480

atcagctctc tgcagctgcg gtcctgggtg tccaagagcc agagagacct ggccaagagc     540

atcctgatcg gagcccctgg cggaccagcc ggatacctga gaagggctag cgtggcccag     600

ctgacccagg aactgggcac cgcctttttc cagcagcagc agctgccagc cgccatggcc     660

gacacctttc tggaacacct gtgcctgctg gacatcgact ctgagcccgt ggccgccaga     720

agcaccagca tcattgccac catcggccct gccagcagaa gcgtggagcg gctgaaagag     780

atgatcaagg ccggcatgaa tatcgcccgg ctgaacttct cccacggcag ccacgagtac     840

cacgcagaga gcattgccaa cgtccgggag gccgtggaga gctttgccgg cagccccctg     900

agctacagac ccgtggccat tgccctggac accaagggcc ccgagatcag aacaggaatt     960

ctgcagggag ggcctgagag cgaggtggag ctggtgaagg gcagccaagt gctggtgacc    1020

gtggaccccg ccttcagaac cagaggcaac gccaacacag tgtgggtgga ctaccccaac    1080

atcgtgcggg tggtgcctgt gggcggcaga atctacatcg acgacggcct gatcagcctg    1140

gtggtgcaga agatcggacc tgagggcctg gtgacccagg tcgagaatgg cggcgtgctg    1200

ggcagcagaa agggcgtgaa tctgccaggc gcccaggtgg acctgcctgg cctgtctgag    1260

caggacgtga gagacctgag atttggcgtg gagcacggcg tggacatcgt gttcgccagc    1320

ttcgtgcgga aggcctctga tgtggccgcc gtgagagccg ctctgggccc tgaaggccac    1380

ggcatcaaga tcatcagcaa gatcgagaac cacgagggcg tgaagcggtt cgacgagatc    1440

ctggaagtgt ccgacggcat catggtggcc agaggcgacc tgggcatcga gatccccgcc    1500

gagaaggtgt tcctggccca gaaaatgatg atcggacggt gcaacctggc cggcaaacct    1560

gtggtgtgcg ccacccagat gctggaaagc atgatcacca agcccagacc caccagagcc    1620

gagacaagcg acgtggccaa cgccgtgctg gatggcgctg actgcatcat gctgtccggc    1680

gagacagcca agggcaactt ccccgtggag gccgtgaaga tgcagcacgc cattgccaga    1740

gaagccgagg ccgccgtgta ccaccggcag ctgttcgagg aactgcggag agccgcccct    1800

ctgagcagag atcccaccga agtgaccgcc atcggagccg tggaagccgc cttcaagtgc    1860

tgcgccgctg caatcatcgt gctgaccacc acaggcagaa gcgcccagct gctgtccaga    1920

tacagaccca gagccgccgt gatcgccgtg acaagatccg cccaggccgc tagacaggtc    1980

cacctgtgca gaggcgtgtt ccccctgctg taccgggagc ctcccgaggc catctgggcc    2040

gacgacgtgg acagacgggt gcagttcggc atcgagagcg gcaagctgcg gggcttcctg    2100

agagtgggcg acctggtgat cgtggtgaca ggctggcggc ctggcagcgg ctacaccaac    2160

atcatgaggg tgctgtccat cagcgactac aaagacgatg acgataaatg aacgcgtgag    2220

ttacaaataa agcactgtgc cttctagttg ccagccatct gttgtttgcc cctcccccgt    2280

gccttccttg accctggaag gtgccactcc cactgtcctt tcctaataaa atgaggaaat    2340

tgcatcgcat tgtctgagta ggtgtcattc tattctgggg ggtggggtgg ggcaggacag    2400

caagggggag gattgggaag acaatagcag gcatgctggg gatgcggtgg gctctatggg    2460

tcccgcagcc ccaggcccac actgaaagca tgtcgatcca ggagaacata tcatccctgc    2520

agcttcggtc atgggtctct aagtcccaaa gagacttagc aaagtccatc ctgattgggg    2580

ctccaggagg taagaagggg agacagaagc catggaacat aggaggaaaa tgagggtgaa    2640

aactaggagc cagggtggag ggcataaatg atccacatca gccactggct aggtgggttt    2700

tggagaggaa cgtacgttct tcagagcctc ccgtgtgtta aattatggac cctggcctgg    2760

gtcttttcca ggccctatag gcaggccaga gccacagcat gtaagccacg gggcactccc    2820

gtggttcctg gactctggcc cctggcatac agggcttcca atggaacagg agacagtggt    2880

gaca                                                                 2884


<210> 20
<211> 2860
<212> DNA
<213> Artificial Sequence


<220> 
<223> LHA-5'UTR-coRPK-bGH poly(A)-RHA

<400> 20
cagagtggtg aaggcactct gcatttcttg gttgagacag agaaaaaaag tggtcagaac      60

tgggtaaccc tccccccacc atattatcac agtgatccct tttgtctttc ttcaggctcc     120

agccccaccc tacagcccct gctccctgga ttcactagag ctaacttcag taaagtacaa     180

agaaaatggg gccatatgac tggccaaaaa aaaaatatct attcacgtgg atgaccagat     240

agtatgaatg gattgaaaat ttatcaggaa aaaaggatga gaggaaatgc caggagatga     300

gggcagagag caggccgttc tgggggaggg attctgtggg gacagggtgg cctactgggt     360

gtgccccttt tctcttctct gtctccctta gataagacca gcagttttgt catcctctcc     420

ctctcttcca tggtcccgca gccccaggcc cacactgaaa gcatgagcat ccaggaaaat     480

atcagctctc tgcagctgcg gtcctgggtg tccaagagcc agagagacct ggccaagagc     540

atcctgatcg gagcccctgg cggaccagcc ggatacctga gaagggctag cgtggcccag     600

ctgacccagg aactgggcac cgcctttttc cagcagcagc agctgccagc cgccatggcc     660

gacacctttc tggaacacct gtgcctgctg gacatcgact ctgagcccgt ggccgccaga     720

agcaccagca tcattgccac catcggccct gccagcagaa gcgtggagcg gctgaaagag     780

atgatcaagg ccggcatgaa tatcgcccgg ctgaacttct cccacggcag ccacgagtac     840

cacgcagaga gcattgccaa cgtccgggag gccgtggaga gctttgccgg cagccccctg     900

agctacagac ccgtggccat tgccctggac accaagggcc ccgagatcag aacaggaatt     960

ctgcagggag ggcctgagag cgaggtggag ctggtgaagg gcagccaagt gctggtgacc    1020

gtggaccccg ccttcagaac cagaggcaac gccaacacag tgtgggtgga ctaccccaac    1080

atcgtgcggg tggtgcctgt gggcggcaga atctacatcg acgacggcct gatcagcctg    1140

gtggtgcaga agatcggacc tgagggcctg gtgacccagg tcgagaatgg cggcgtgctg    1200

ggcagcagaa agggcgtgaa tctgccaggc gcccaggtgg acctgcctgg cctgtctgag    1260

caggacgtga gagacctgag atttggcgtg gagcacggcg tggacatcgt gttcgccagc    1320

ttcgtgcgga aggcctctga tgtggccgcc gtgagagccg ctctgggccc tgaaggccac    1380

ggcatcaaga tcatcagcaa gatcgagaac cacgagggcg tgaagcggtt cgacgagatc    1440

ctggaagtgt ccgacggcat catggtggcc agaggcgacc tgggcatcga gatccccgcc    1500

gagaaggtgt tcctggccca gaaaatgatg atcggacggt gcaacctggc cggcaaacct    1560

gtggtgtgcg ccacccagat gctggaaagc atgatcacca agcccagacc caccagagcc    1620

gagacaagcg acgtggccaa cgccgtgctg gatggcgctg actgcatcat gctgtccggc    1680

gagacagcca agggcaactt ccccgtggag gccgtgaaga tgcagcacgc cattgccaga    1740

gaagccgagg ccgccgtgta ccaccggcag ctgttcgagg aactgcggag agccgcccct    1800

ctgagcagag atcccaccga agtgaccgcc atcggagccg tggaagccgc cttcaagtgc    1860

tgcgccgctg caatcatcgt gctgaccacc acaggcagaa gcgcccagct gctgtccaga    1920

tacagaccca gagccgccgt gatcgccgtg acaagatccg cccaggccgc tagacaggtc    1980

cacctgtgca gaggcgtgtt ccccctgctg taccgggagc ctcccgaggc catctgggcc    2040

gacgacgtgg acagacgggt gcagttcggc atcgagagcg gcaagctgcg gggcttcctg    2100

agagtgggcg acctggtgat cgtggtgaca ggctggcggc ctggcagcgg ctacaccaac    2160

atcatgaggg tgctgtccat cagctgaacg cgtgagttac aaataaagca ctgtgccttc    2220

tagttgccag ccatctgttg tttgcccctc ccccgtgcct tccttgaccc tggaaggtgc    2280

cactcccact gtcctttcct aataaaatga ggaaattgca tcgcattgtc tgagtaggtg    2340

tcattctatt ctggggggtg gggtggggca ggacagcaag ggggaggatt gggaagacaa    2400

tagcaggcat gctggggatg cggtgggctc tatgggtccc gcagccccag gcccacactg    2460

aaagcatgtc gatccaggag aacatatcat ccctgcagct tcggtcatgg gtctctaagt    2520

cccaaagaga cttagcaaag tccatcctga ttggggctcc aggaggtaag aaggggagac    2580

agaagccatg gaacatagga ggaaaatgag ggtgaaaact aggagccagg gtggagggca    2640

taaatgatcc acatcagcca ctggctaggt gggttttgga gaggaacgta cgttcttcag    2700

agcctcccgt gtgttaaatt atggaccctg gcctgggtct tttccaggcc ctataggcag    2760

gccagagcca cagcatgtaa gccacggggc actcccgtgg ttcctggact ctggcccctg    2820

gcatacaggg cttccaatgg aacaggagac agtggtgaca                          2860


<210> 21
<211> 2884
<212> DNA
<213> Artificial Sequence


<220> 
<223> LHA-5'UTR-coRPK-bGH poly(A)-RHA

<400> 21
tgtcaccact gtctcctgtt ccattggaag ccctgtatgc caggggccag agtccaggaa      60

ccacgggagt gccccgtggc ttacatgctg tggctctggc ctgcctatag ggcctggaaa     120

agacccaggc cagggtccat aatttaacac acgggaggct ctgaagaacg tacgttcctc     180

tccaaaaccc acctagccag tggctgatgt ggatcattta tgccctccac cctggctcct     240

agttttcacc ctcattttcc tcctatgttc catggcttct gtctcccctt cttacctcct     300

ggagccccaa tcaggatgga ctttgctaag tctctttggg acttagagac ccatgaccga     360

agctgcaggg atgatatgtt ctcctggatc gacatgcttt cagtgtgggc ctggggctgc     420

gggacccata gagcccaccg catccccagc atgcctgcta ttgtcttccc aatcctcccc     480

cttgctgtcc tgccccaccc caccccccag aatagaatga cacctactca gacaatgcga     540

tgcaatttcc tcattttatt aggaaaggac agtgggagtg gcaccttcca gggtcaagga     600

aggcacgggg gaggggcaaa caacagatgg ctggcaacta gaaggcacag tgctttattt     660

gtaactcacg cgttcattta tcgtcatcgt ctttgtagtc gctgatggac agcaccctca     720

tgatgttggt gtagccgctg ccaggccgcc agcctgtcac cacgatcacc aggtcgccca     780

ctctcaggaa gccccgcagc ttgccgctct cgatgccgaa ctgcacccgt ctgtccacgt     840

cgtcggccca gatggcctcg ggaggctccc ggtacagcag ggggaacacg cctctgcaca     900

ggtggacctg tctagcggcc tgggcggatc ttgtcacggc gatcacggcg gctctgggtc     960

tgtatctgga cagcagctgg gcgcttctgc ctgtggtggt cagcacgatg attgcagcgg    1020

cgcagcactt gaaggcggct tccacggctc cgatggcggt cacttcggtg ggatctctgc    1080

tcagaggggc ggctctccgc agttcctcga acagctgccg gtggtacacg gcggcctcgg    1140

cttctctggc aatggcgtgc tgcatcttca cggcctccac ggggaagttg cccttggctg    1200

tctcgccgga cagcatgatg cagtcagcgc catccagcac ggcgttggcc acgtcgcttg    1260

tctcggctct ggtgggtctg ggcttggtga tcatgctttc cagcatctgg gtggcgcaca    1320

ccacaggttt gccggccagg ttgcaccgtc cgatcatcat tttctgggcc aggaacacct    1380

tctcggcggg gatctcgatg cccaggtcgc ctctggccac catgatgccg tcggacactt    1440

ccaggatctc gtcgaaccgc ttcacgccct cgtggttctc gatcttgctg atgatcttga    1500

tgccgtggcc ttcagggccc agagcggctc tcacggcggc cacatcagag gccttccgca    1560

cgaagctggc gaacacgatg tccacgccgt gctccacgcc aaatctcagg tctctcacgt    1620

cctgctcaga caggccaggc aggtccacct gggcgcctgg cagattcacg ccctttctgc    1680

tgcccagcac gccgccattc tcgacctggg tcaccaggcc ctcaggtccg atcttctgca    1740

ccaccaggct gatcaggccg tcgtcgatgt agattctgcc gcccacaggc accacccgca    1800

cgatgttggg gtagtccacc cacactgtgt tggcgttgcc tctggttctg aaggcggggt    1860

ccacggtcac cagcacttgg ctgcccttca ccagctccac ctcgctctca ggccctccct    1920

gcagaattcc tgttctgatc tcggggccct tggtgtccag ggcaatggcc acgggtctgt    1980

agctcagggg gctgccggca aagctctcca cggcctcccg gacgttggca atgctctctg    2040

cgtggtactc gtggctgccg tgggagaagt tcagccgggc gatattcatg ccggccttga    2100

tcatctcttt cagccgctcc acgcttctgc tggcagggcc gatggtggca atgatgctgg    2160

tgcttctggc ggccacgggc tcagagtcga tgtccagcag gcacaggtgt tccagaaagg    2220

tgtcggccat ggcggctggc agctgctgct gctggaaaaa ggcggtgccc agttcctggg    2280

tcagctgggc cacgctagcc cttctcaggt atccggctgg tccgccaggg gctccgatca    2340

ggatgctctt ggccaggtct ctctggctct tggacaccca ggaccgcagc tgcagagagc    2400

tgatattttc ctggatgctc atgctttcag tgtgggcctg gggctgcggg accatggaag    2460

agagggagag gatgacaaaa ctgctggtct tatctaaggg agacagagaa gagaaaaggg    2520

gcacacccag taggccaccc tgtccccaca gaatccctcc cccagaacgg cctgctctct    2580

gccctcatct cctggcattt cctctcatcc ttttttcctg ataaattttc aatccattca    2640

tactatctgg tcatccacgt gaatagatat tttttttttg gccagtcata tggccccatt    2700

ttctttgtac tttactgaag ttagctctag tgaatccagg gagcaggggc tgtagggtgg    2760

ggctggagcc tgaagaaaga caaaagggat cactgtgata atatggtggg gggagggtta    2820

cccagttctg accacttttt ttctctgtct caaccaagaa atgcagagtg ccttcaccac    2880

tctg                                                                 2884


<210> 22
<211> 2860
<212> DNA
<213> Artificial Sequence


<220> 
<223> LHA-5'UTR-coRPK-bGH poly(A)-RHA

<400> 22
tgtcaccact gtctcctgtt ccattggaag ccctgtatgc caggggccag agtccaggaa      60

ccacgggagt gccccgtggc ttacatgctg tggctctggc ctgcctatag ggcctggaaa     120

agacccaggc cagggtccat aatttaacac acgggaggct ctgaagaacg tacgttcctc     180

tccaaaaccc acctagccag tggctgatgt ggatcattta tgccctccac cctggctcct     240

agttttcacc ctcattttcc tcctatgttc catggcttct gtctcccctt cttacctcct     300

ggagccccaa tcaggatgga ctttgctaag tctctttggg acttagagac ccatgaccga     360

agctgcaggg atgatatgtt ctcctggatc gacatgcttt cagtgtgggc ctggggctgc     420

gggacccata gagcccaccg catccccagc atgcctgcta ttgtcttccc aatcctcccc     480

cttgctgtcc tgccccaccc caccccccag aatagaatga cacctactca gacaatgcga     540

tgcaatttcc tcattttatt aggaaaggac agtgggagtg gcaccttcca gggtcaagga     600

aggcacgggg gaggggcaaa caacagatgg ctggcaacta gaaggcacag tgctttattt     660

gtaactcacg cgttcagctg atggacagca ccctcatgat gttggtgtag ccgctgccag     720

gccgccagcc tgtcaccacg atcaccaggt cgcccactct caggaagccc cgcagcttgc     780

cgctctcgat gccgaactgc acccgtctgt ccacgtcgtc ggcccagatg gcctcgggag     840

gctcccggta cagcaggggg aacacgcctc tgcacaggtg gacctgtcta gcggcctggg     900

cggatcttgt cacggcgatc acggcggctc tgggtctgta tctggacagc agctgggcgc     960

ttctgcctgt ggtggtcagc acgatgattg cagcggcgca gcacttgaag gcggcttcca    1020

cggctccgat ggcggtcact tcggtgggat ctctgctcag aggggcggct ctccgcagtt    1080

cctcgaacag ctgccggtgg tacacggcgg cctcggcttc tctggcaatg gcgtgctgca    1140

tcttcacggc ctccacgggg aagttgccct tggctgtctc gccggacagc atgatgcagt    1200

cagcgccatc cagcacggcg ttggccacgt cgcttgtctc ggctctggtg ggtctgggct    1260

tggtgatcat gctttccagc atctgggtgg cgcacaccac aggtttgccg gccaggttgc    1320

accgtccgat catcattttc tgggccagga acaccttctc ggcggggatc tcgatgccca    1380

ggtcgcctct ggccaccatg atgccgtcgg acacttccag gatctcgtcg aaccgcttca    1440

cgccctcgtg gttctcgatc ttgctgatga tcttgatgcc gtggccttca gggcccagag    1500

cggctctcac ggcggccaca tcagaggcct tccgcacgaa gctggcgaac acgatgtcca    1560

cgccgtgctc cacgccaaat ctcaggtctc tcacgtcctg ctcagacagg ccaggcaggt    1620

ccacctgggc gcctggcaga ttcacgccct ttctgctgcc cagcacgccg ccattctcga    1680

cctgggtcac caggccctca ggtccgatct tctgcaccac caggctgatc aggccgtcgt    1740

cgatgtagat tctgccgccc acaggcacca cccgcacgat gttggggtag tccacccaca    1800

ctgtgttggc gttgcctctg gttctgaagg cggggtccac ggtcaccagc acttggctgc    1860

ccttcaccag ctccacctcg ctctcaggcc ctccctgcag aattcctgtt ctgatctcgg    1920

ggcccttggt gtccagggca atggccacgg gtctgtagct cagggggctg ccggcaaagc    1980

tctccacggc ctcccggacg ttggcaatgc tctctgcgtg gtactcgtgg ctgccgtggg    2040

agaagttcag ccgggcgata ttcatgccgg ccttgatcat ctctttcagc cgctccacgc    2100

ttctgctggc agggccgatg gtggcaatga tgctggtgct tctggcggcc acgggctcag    2160

agtcgatgtc cagcaggcac aggtgttcca gaaaggtgtc ggccatggcg gctggcagct    2220

gctgctgctg gaaaaaggcg gtgcccagtt cctgggtcag ctgggccacg ctagcccttc    2280

tcaggtatcc ggctggtccg ccaggggctc cgatcaggat gctcttggcc aggtctctct    2340

ggctcttgga cacccaggac cgcagctgca gagagctgat attttcctgg atgctcatgc    2400

tttcagtgtg ggcctggggc tgcgggacca tggaagagag ggagaggatg acaaaactgc    2460

tggtcttatc taagggagac agagaagaga aaaggggcac acccagtagg ccaccctgtc    2520

cccacagaat ccctccccca gaacggcctg ctctctgccc tcatctcctg gcatttcctc    2580

tcatcctttt ttcctgataa attttcaatc cattcatact atctggtcat ccacgtgaat    2640

agatattttt tttttggcca gtcatatggc cccattttct ttgtacttta ctgaagttag    2700

ctctagtgaa tccagggagc aggggctgta gggtggggct ggagcctgaa gaaagacaaa    2760

agggatcact gtgataatat ggtgggggga gggttaccca gttctgacca ctttttttct    2820

ctgtctcaac caagaaatgc agagtgcctt caccactctg                          2860


<210> 23
<211> 2876
<212> DNA
<213> Artificial Sequence


<220> 
<223> AAV backbone

<400> 23
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt      60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     120

aggggttcct gcggccgcag gaacccctag tgatggagtt ggccactccc tctctgcgcg     180

ctcgctcgct cactgaggcc gggcgaccaa aggtcgcccg acgcccgggc tttgcccggg     240

cggcctcagt gagcgagcga gcgcgcagct gcctgcaggg gcgcctgatg cggtattttc     300

tccttacgca tctgtgcggt atttcacacc gcatacgtca aagcaaccat agtacgcgcc     360

ctgtagcggc gcattaagcg cggcgggtgt ggtggttacg cgcagcgtga ccgctacact     420

tgccagcgcc ctagcgcccg ctcctttcgc tttcttccct tcctttctcg ccacgttcgc     480

cggctttccc cgtcaagctc taaatcgggg gctcccttta gggttccgat ttagtgcttt     540

acggcacctc gaccccaaaa aacttgattt gggtgatggt tcacgtagtg ggccatcgcc     600

ctgatagacg gtttttcgcc ctttgacgtt ggagtccacg ttctttaata gtggactctt     660

gttccaaact ggaacaacac tcaaccctat ctcgggctat tcttttgatt tataagggat     720

tttgccgatt tcggcctatt ggttaaaaaa tgagctgatt taacaaaaat ttaacgcgaa     780

ttttaacaaa atattaacgt ttacaatttt atggtgcact ctcagtacaa tctgctctga     840

tgccgcatag ttaagccagc cccgacaccc gccaacaccc gctgacgcgc cctgacgggc     900

ttgtctgctc ccggcatccg cttacagaca agctgtgacc gtctccggga gctgcatgtg     960

tcagaggttt tcaccgtcat caccgaaacg cgcgagacga aagggcctcg tgatacgcct    1020

atttttatag gttaatgtca tgataataat ggtttcttag acgtcaggtg gcacttttcg    1080

gggaaatgtg cgcggaaccc ctatttgttt atttttctaa atacattcaa atatgtatcc    1140

gctcatgaga caataaccct gataaatgct tcaataatat tgaaaaagga agagtatgag    1200

tattcaacat ttccgtgtcg cccttattcc cttttttgcg gcattttgcc ttcctgtttt    1260

tgctcaccca gaaacgctgg tgaaagtaaa agatgctgaa gatcagttgg gtgcacgagt    1320

gggttacatc gaactggatc tcaacagcgg taagatcctt gagagttttc gccccgaaga    1380

acgttttcca atgatgagca cttttaaagt tctgctatgt ggcgcggtat tatcccgtat    1440

tgacgccggg caagagcaac tcggtcgccg catacactat tctcagaatg acttggttga    1500

gtactcacca gtcacagaaa agcatcttac ggatggcatg acagtaagag aattatgcag    1560

tgctgccata accatgagtg ataacactgc ggccaactta cttctgacaa cgatcggagg    1620

accgaaggag ctaaccgctt ttttgcacaa catgggggat catgtaactc gccttgatcg    1680

ttgggaaccg gagctgaatg aagccatacc aaacgacgag cgtgacacca cgatgcctgt    1740

agcaatggca acaacgttgc gcaaactatt aactggcgaa ctacttactc tagcttcccg    1800

gcaacaatta atagactgga tggaggcgga taaagttgca ggaccacttc tgcgctcggc    1860

ccttccggct ggctggttta ttgctgataa atctggagcc ggtgagcgtg ggtctcgcgg    1920

tatcattgca gcactggggc cagatggtaa gccctcccgt atcgtagtta tctacacgac    1980

ggggagtcag gcaactatgg atgaacgaaa tagacagatc gctgagatag gtgcctcact    2040

gattaagcat tggtaactgt cagaccaagt ttactcatat atactttaga ttgatttaaa    2100

acttcatttt taatttaaaa ggatctaggt gaagatcctt tttgataatc tcatgaccaa    2160

aatcccttaa cgtgagtttt cgttccactg agcgtcagac cccgtagaaa agatcaaagg    2220

atcttcttga gatccttttt ttctgcgcgt aatctgctgc ttgcaaacaa aaaaaccacc    2280

gctaccagcg gtggtttgtt tgccggatca agagctacca actctttttc cgaaggtaac    2340

tggcttcagc agagcgcaga taccaaatac tgtccttcta gtgtagccgt agttaggcca    2400

ccacttcaag aactctgtag caccgcctac atacctcgct ctgctaatcc tgttaccagt    2460

ggctgctgcc agtggcgata agtcgtgtct taccgggttg gactcaagac gatagttacc    2520

ggataaggcg cagcggtcgg gctgaacggg gggttcgtgc acacagccca gcttggagcg    2580

aacgacctac accgaactga gatacctaca gcgtgagcta tgagaaagcg ccacgcttcc    2640

cgaagggaga aaggcggaca ggtatccggt aagcggcagg gtcggaacag gagagcgcac    2700

gagggagctt ccagggggaa acgcctggta tctttatagt cctgtcgggt ttcgccacct    2760

ctgacttgag cgtcgatttt tgtgatgctc gtcagggggg cggagcctat ggaaaaacgc    2820

cagcaacgcg gcctttttac ggttcctggc cttttgctgg ccttttgctc acatgt        2876


<210> 24
<211> 5899
<212> DNA
<213> Artificial Sequence


<220> 
<223> coRPK AAV used for pre-clinical studies

<400> 24
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct gcggccgcca gagtggtgaa ggcactctgc atttcttggt tgagacagag      180

aaaaaaagtg gtcagaactg ggtaaccctc cccccaccat attatcacag tgatcccttt      240

tgtctttctt caggctccag ccccacccta cagcccctgc tccctggatt cactagagct      300

aacttcagta aagtacaaag aaaatggggc catatgactg gccaaaaaaa aaatatctat      360

tcacgtggat gaccagatag tatgaatgga ttgaaaattt atcaggaaaa aaggatgaga      420

ggaaatgcca ggagatgagg gcagagagca ggccgttctg ggggagggat tctgtgggga      480

cagggtggcc tactgggtgt gccccttttc tcttctctgt ctcccttaga taagaccagc      540

agttttgtca tcctctccct ctcttccatg gtcccgcagc cccaggccca cactgaaagc      600

atgagcatcc aggaaaatat cagctctctg cagctgcggt cctgggtgtc caagagccag      660

agagacctgg ccaagagcat cctgatcgga gcccctggcg gaccagccgg atacctgaga      720

agggctagcg tggcccagct gacccaggaa ctgggcaccg cctttttcca gcagcagcag      780

ctgccagccg ccatggccga cacctttctg gaacacctgt gcctgctgga catcgactct      840

gagcccgtgg ccgccagaag caccagcatc attgccacca tcggccctgc cagcagaagc      900

gtggagcggc tgaaagagat gatcaaggcc ggcatgaata tcgcccggct gaacttctcc      960

cacggcagcc acgagtacca cgcagagagc attgccaacg tccgggaggc cgtggagagc     1020

tttgccggca gccccctgag ctacagaccc gtggccattg ccctggacac caagggcccc     1080

gagatcagaa caggaattct gcagggaggg cctgagagcg aggtggagct ggtgaagggc     1140

agccaagtgc tggtgaccgt ggaccccgcc ttcagaacca gaggcaacgc caacacagtg     1200

tgggtggact accccaacat cgtgcgggtg gtgcctgtgg gcggcagaat ctacatcgac     1260

gacggcctga tcagcctggt ggtgcagaag atcggacctg agggcctggt gacccaggtc     1320

gagaatggcg gcgtgctggg cagcagaaag ggcgtgaatc tgccaggcgc ccaggtggac     1380

ctgcctggcc tgtctgagca ggacgtgaga gacctgagat ttggcgtgga gcacggcgtg     1440

gacatcgtgt tcgccagctt cgtgcggaag gcctctgatg tggccgccgt gagagccgct     1500

ctgggccctg aaggccacgg catcaagatc atcagcaaga tcgagaacca cgagggcgtg     1560

aagcggttcg acgagatcct ggaagtgtcc gacggcatca tggtggccag aggcgacctg     1620

ggcatcgaga tccccgccga gaaggtgttc ctggcccaga aaatgatgat cggacggtgc     1680

aacctggccg gcaaacctgt ggtgtgcgcc acccagatgc tggaaagcat gatcaccaag     1740

cccagaccca ccagagccga gacaagcgac gtggccaacg ccgtgctgga tggcgctgac     1800

tgcatcatgc tgtccggcga gacagccaag ggcaacttcc ccgtggaggc cgtgaagatg     1860

cagcacgcca ttgccagaga agccgaggcc gccgtgtacc accggcagct gttcgaggaa     1920

ctgcggagag ccgcccctct gagcagagat cccaccgaag tgaccgccat cggagccgtg     1980

gaagccgcct tcaagtgctg cgccgctgca atcatcgtgc tgaccaccac aggcagaagc     2040

gcccagctgc tgtccagata cagacccaga gccgccgtga tcgccgtgac aagatccgcc     2100

caggccgcta gacaggtcca cctgtgcaga ggcgtgttcc ccctgctgta ccgggagcct     2160

cccgaggcca tctgggccga cgacgtggac agacgggtgc agttcggcat cgagagcggc     2220

aagctgcggg gcttcctgag agtgggcgac ctggtgatcg tggtgacagg ctggcggcct     2280

ggcagcggct acaccaacat catgagggtg ctgtccatca gcgactacaa agacgatgac     2340

gataaatgaa cgcgtgagtt acaaataaag cactgtgcct tctagttgcc agccatctgt     2400

tgtttgcccc tcccccgtgc cttccttgac cctggaaggt gccactccca ctgtcctttc     2460

ctaataaaat gaggaaattg catcgcattg tctgagtagg tgtcattcta ttctgggggg     2520

tggggtgggg caggacagca agggggagga ttgggaagac aatagcaggc atgctgggga     2580

tgcggtgggc tctatgggtc ccgcagcccc aggcccacac tgaaagcatg tcgatccagg     2640

agaacatatc atccctgcag cttcggtcat gggtctctaa gtcccaaaga gacttagcaa     2700

agtccatcct gattggggct ccaggaggta agaaggggag acagaagcca tggaacatag     2760

gaggaaaatg agggtgaaaa ctaggagcca gggtggaggg cataaatgat ccacatcagc     2820

cactggctag gtgggttttg gagaggaacg tacgttcttc agagcctccc gtgtgttaaa     2880

ttatggaccc tggcctgggt cttttccagg ccctataggc aggccagagc cacagcatgt     2940

aagccacggg gcactcccgt ggttcctgga ctctggcccc tggcatacag ggcttccaat     3000

ggaacaggag acagtggtga cagcggccgc aggaacccct agtgatggag ttggccactc     3060

cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc cgacgcccgg     3120

gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag ctgcctgcag gggcgcctga     3180

tgcggtattt tctccttacg catctgtgcg gtatttcaca ccgcatacgt caaagcaacc     3240

atagtacgcg ccctgtagcg gcgcattaag cgcggcgggt gtggtggtta cgcgcagcgt     3300

gaccgctaca cttgccagcg ccctagcgcc cgctcctttc gctttcttcc cttcctttct     3360

cgccacgttc gccggctttc cccgtcaagc tctaaatcgg gggctccctt tagggttccg     3420

atttagtgct ttacggcacc tcgaccccaa aaaacttgat ttgggtgatg gttcacgtag     3480

tgggccatcg ccctgataga cggtttttcg ccctttgacg ttggagtcca cgttctttaa     3540

tagtggactc ttgttccaaa ctggaacaac actcaaccct atctcgggct attcttttga     3600

tttataaggg attttgccga tttcggccta ttggttaaaa aatgagctga tttaacaaaa     3660

atttaacgcg aattttaaca aaatattaac gtttacaatt ttatggtgca ctctcagtac     3720

aatctgctct gatgccgcat agttaagcca gccccgacac ccgccaacac ccgctgacgc     3780

gccctgacgg gcttgtctgc tcccggcatc cgcttacaga caagctgtga ccgtctccgg     3840

gagctgcatg tgtcagaggt tttcaccgtc atcaccgaaa cgcgcgagac gaaagggcct     3900

cgtgatacgc ctatttttat aggttaatgt catgataata atggtttctt agacaaacct     3960

agatattgat agtctgatcg gtcaacgtat aatcgagtcc tagcttttgc aaacatctat     4020

caagagacag gatcagcagg aggctttcgc atgattgaac aagatggatt gcacgcaggt     4080

tctccggcgg cttgggtgga gaggctattc ggctatgact gggcacaaca gacaatcggc     4140

tgctctgatg ccgccgtgtt ccggctgtca gcgcaggggc gtccggttct ttttgtcaag     4200

accgacctgt ccggtgccct gaatgaactg caagacgagg cagcgcggct atcgtggctg     4260

gcgacgacgg gcgttccttg cgcggctgtg ctcgacgttg tcactgaagc gggaagggac     4320

tggctgctat tgggcgaagt gccggggcag gatctcctgt catctcacct tgctcctgcc     4380

gagaaagtat ccatcatggc tgatgcaatg cggcggctgc atacgcttga tccggctacc     4440

tgcccattcg accaccaagc gaaacatcgc atcgagcgag cacgtactcg gatggaagcc     4500

ggtcttgtcg atcaggatga tctggacgaa gagcatcagg ggctcgcgcc agccgaactg     4560

ttcgccaggc tcaaggcgtc tatgcccgac ggcgaggatc tcgtcgtgac ccacggcgat     4620

gcctgcttgc cgaatatcat ggtggaaaat ggccgctttt ctggattcat cgactgtggc     4680

cgtctgggtg tggcggaccg ctatcaggac atagcgttgg ctacccgtga tattgctgaa     4740

gagcttggcg gcgaatgggc tgaccgcttc cttgtgcttt acggtatcgc cgcgcccgat     4800

tcgcagcgca tcgccttcta tcgccttctt gacgagttct tctgaccgat tctaggtgca     4860

ttggcgcaga aaaaaatgcc tgatgcgacg ctgcgcgtct tatactccca catatgccag     4920

attcagcaac ggatacggct tccccaactt gcccacttcc atacgtgtcc tccttaccag     4980

aaatttatcc ttaacgatcg gacggggagt caggcaacta tggatgaacg aaatagacag     5040

atcgctgaga taggtgcctc actgattaag cattggtaac tgtcagacca agtttactca     5100

tatatacttt agattgattt aaaacttcat ttttaattta aaaggatcta ggtgaagatc     5160

ctttttgata atctcatgac caaaatccct taacgtgagt tttcgttcca ctgagcgtca     5220

gaccccgtag aaaagatcaa aggatcttct tgagatcctt tttttctgcg cgtaatctgc     5280

tgcttgcaaa caaaaaaacc accgctacca gcggtggttt gtttgccgga tcaagagcta     5340

ccaactcttt ttccgaaggt aactggcttc agcagagcgc agataccaaa tactgttctt     5400

ctagtgtagc cgtagttagg ccaccacttc aagaactctg tagcaccgcc tacatacctc     5460

gctctgctaa tcctgttacc agtggctgct gccagtggcg ataagtcgtg tcttaccggg     5520

ttggactcaa gacgatagtt accggataag gcgcagcggt cgggctgaac ggggggttcg     5580

tgcacacagc ccagcttgga gcgaacgacc tacaccgaac tgagatacct acagcgtgag     5640

ctatgagaaa gcgccacgct tcccgaaggg agaaaggcgg acaggtatcc ggtaagcggc     5700

agggtcggaa caggagagcg cacgagggag cttccagggg gaaacgcctg gtatctttat     5760

agtcctgtcg ggtttcgcca cctctgactt gagcgtcgat ttttgtgatg ctcgtcaggg     5820

gggcggagcc tatggaaaaa cgccagcaac gcggcctttt tacggttcct ggccttttgc     5880

tggccttttg ctcacatgt                                                  5899


<210> 25
<211> 5875
<212> DNA
<213> Artificial Sequence


<220> 
<223> coRPK AAV used for pre-clinical studies

<400> 25
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct gcggccgcca gagtggtgaa ggcactctgc atttcttggt tgagacagag      180

aaaaaaagtg gtcagaactg ggtaaccctc cccccaccat attatcacag tgatcccttt      240

tgtctttctt caggctccag ccccacccta cagcccctgc tccctggatt cactagagct      300

aacttcagta aagtacaaag aaaatggggc catatgactg gccaaaaaaa aaatatctat      360

tcacgtggat gaccagatag tatgaatgga ttgaaaattt atcaggaaaa aaggatgaga      420

ggaaatgcca ggagatgagg gcagagagca ggccgttctg ggggagggat tctgtgggga      480

cagggtggcc tactgggtgt gccccttttc tcttctctgt ctcccttaga taagaccagc      540

agttttgtca tcctctccct ctcttccatg gtcccgcagc cccaggccca cactgaaagc      600

atgagcatcc aggaaaatat cagctctctg cagctgcggt cctgggtgtc caagagccag      660

agagacctgg ccaagagcat cctgatcgga gcccctggcg gaccagccgg atacctgaga      720

agggctagcg tggcccagct gacccaggaa ctgggcaccg cctttttcca gcagcagcag      780

ctgccagccg ccatggccga cacctttctg gaacacctgt gcctgctgga catcgactct      840

gagcccgtgg ccgccagaag caccagcatc attgccacca tcggccctgc cagcagaagc      900

gtggagcggc tgaaagagat gatcaaggcc ggcatgaata tcgcccggct gaacttctcc      960

cacggcagcc acgagtacca cgcagagagc attgccaacg tccgggaggc cgtggagagc     1020

tttgccggca gccccctgag ctacagaccc gtggccattg ccctggacac caagggcccc     1080

gagatcagaa caggaattct gcagggaggg cctgagagcg aggtggagct ggtgaagggc     1140

agccaagtgc tggtgaccgt ggaccccgcc ttcagaacca gaggcaacgc caacacagtg     1200

tgggtggact accccaacat cgtgcgggtg gtgcctgtgg gcggcagaat ctacatcgac     1260

gacggcctga tcagcctggt ggtgcagaag atcggacctg agggcctggt gacccaggtc     1320

gagaatggcg gcgtgctggg cagcagaaag ggcgtgaatc tgccaggcgc ccaggtggac     1380

ctgcctggcc tgtctgagca ggacgtgaga gacctgagat ttggcgtgga gcacggcgtg     1440

gacatcgtgt tcgccagctt cgtgcggaag gcctctgatg tggccgccgt gagagccgct     1500

ctgggccctg aaggccacgg catcaagatc atcagcaaga tcgagaacca cgagggcgtg     1560

aagcggttcg acgagatcct ggaagtgtcc gacggcatca tggtggccag aggcgacctg     1620

ggcatcgaga tccccgccga gaaggtgttc ctggcccaga aaatgatgat cggacggtgc     1680

aacctggccg gcaaacctgt ggtgtgcgcc acccagatgc tggaaagcat gatcaccaag     1740

cccagaccca ccagagccga gacaagcgac gtggccaacg ccgtgctgga tggcgctgac     1800

tgcatcatgc tgtccggcga gacagccaag ggcaacttcc ccgtggaggc cgtgaagatg     1860

cagcacgcca ttgccagaga agccgaggcc gccgtgtacc accggcagct gttcgaggaa     1920

ctgcggagag ccgcccctct gagcagagat cccaccgaag tgaccgccat cggagccgtg     1980

gaagccgcct tcaagtgctg cgccgctgca atcatcgtgc tgaccaccac aggcagaagc     2040

gcccagctgc tgtccagata cagacccaga gccgccgtga tcgccgtgac aagatccgcc     2100

caggccgcta gacaggtcca cctgtgcaga ggcgtgttcc ccctgctgta ccgggagcct     2160

cccgaggcca tctgggccga cgacgtggac agacgggtgc agttcggcat cgagagcggc     2220

aagctgcggg gcttcctgag agtgggcgac ctggtgatcg tggtgacagg ctggcggcct     2280

ggcagcggct acaccaacat catgagggtg ctgtccatca gctgaacgcg tgagttacaa     2340

ataaagcact gtgccttcta gttgccagcc atctgttgtt tgcccctccc ccgtgccttc     2400

cttgaccctg gaaggtgcca ctcccactgt cctttcctaa taaaatgagg aaattgcatc     2460

gcattgtctg agtaggtgtc attctattct ggggggtggg gtggggcagg acagcaaggg     2520

ggaggattgg gaagacaata gcaggcatgc tggggatgcg gtgggctcta tgggtcccgc     2580

agccccaggc ccacactgaa agcatgtcga tccaggagaa catatcatcc ctgcagcttc     2640

ggtcatgggt ctctaagtcc caaagagact tagcaaagtc catcctgatt ggggctccag     2700

gaggtaagaa ggggagacag aagccatgga acataggagg aaaatgaggg tgaaaactag     2760

gagccagggt ggagggcata aatgatccac atcagccact ggctaggtgg gttttggaga     2820

ggaacgtacg ttcttcagag cctcccgtgt gttaaattat ggaccctggc ctgggtcttt     2880

tccaggccct ataggcaggc cagagccaca gcatgtaagc cacggggcac tcccgtggtt     2940

cctggactct ggcccctggc atacagggct tccaatggaa caggagacag tggtgacagc     3000

ggccgcagga acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca     3060

ctgaggccgg gcgaccaaag gtcgcccgac gcccgggctt tgcccgggcg gcctcagtga     3120

gcgagcgagc gcgcagctgc ctgcaggggc gcctgatgcg gtattttctc cttacgcatc     3180

tgtgcggtat ttcacaccgc atacgtcaaa gcaaccatag tacgcgccct gtagcggcgc     3240

attaagcgcg gcgggtgtgg tggttacgcg cagcgtgacc gctacacttg ccagcgccct     3300

agcgcccgct cctttcgctt tcttcccttc ctttctcgcc acgttcgccg gctttccccg     3360

tcaagctcta aatcgggggc tccctttagg gttccgattt agtgctttac ggcacctcga     3420

ccccaaaaaa cttgatttgg gtgatggttc acgtagtggg ccatcgccct gatagacggt     3480

ttttcgccct ttgacgttgg agtccacgtt ctttaatagt ggactcttgt tccaaactgg     3540

aacaacactc aaccctatct cgggctattc ttttgattta taagggattt tgccgatttc     3600

ggcctattgg ttaaaaaatg agctgattta acaaaaattt aacgcgaatt ttaacaaaat     3660

attaacgttt acaattttat ggtgcactct cagtacaatc tgctctgatg ccgcatagtt     3720

aagccagccc cgacacccgc caacacccgc tgacgcgccc tgacgggctt gtctgctccc     3780

ggcatccgct tacagacaag ctgtgaccgt ctccgggagc tgcatgtgtc agaggttttc     3840

accgtcatca ccgaaacgcg cgagacgaaa gggcctcgtg atacgcctat ttttataggt     3900

taatgtcatg ataataatgg tttcttagac aaacctagat attgatagtc tgatcggtca     3960

acgtataatc gagtcctagc ttttgcaaac atctatcaag agacaggatc agcaggaggc     4020

tttcgcatga ttgaacaaga tggattgcac gcaggttctc cggcggcttg ggtggagagg     4080

ctattcggct atgactgggc acaacagaca atcggctgct ctgatgccgc cgtgttccgg     4140

ctgtcagcgc aggggcgtcc ggttcttttt gtcaagaccg acctgtccgg tgccctgaat     4200

gaactgcaag acgaggcagc gcggctatcg tggctggcga cgacgggcgt tccttgcgcg     4260

gctgtgctcg acgttgtcac tgaagcggga agggactggc tgctattggg cgaagtgccg     4320

gggcaggatc tcctgtcatc tcaccttgct cctgccgaga aagtatccat catggctgat     4380

gcaatgcggc ggctgcatac gcttgatccg gctacctgcc cattcgacca ccaagcgaaa     4440

catcgcatcg agcgagcacg tactcggatg gaagccggtc ttgtcgatca ggatgatctg     4500

gacgaagagc atcaggggct cgcgccagcc gaactgttcg ccaggctcaa ggcgtctatg     4560

cccgacggcg aggatctcgt cgtgacccac ggcgatgcct gcttgccgaa tatcatggtg     4620

gaaaatggcc gcttttctgg attcatcgac tgtggccgtc tgggtgtggc ggaccgctat     4680

caggacatag cgttggctac ccgtgatatt gctgaagagc ttggcggcga atgggctgac     4740

cgcttccttg tgctttacgg tatcgccgcg cccgattcgc agcgcatcgc cttctatcgc     4800

cttcttgacg agttcttctg accgattcta ggtgcattgg cgcagaaaaa aatgcctgat     4860

gcgacgctgc gcgtcttata ctcccacata tgccagattc agcaacggat acggcttccc     4920

caacttgccc acttccatac gtgtcctcct taccagaaat ttatccttaa cgatcggacg     4980

gggagtcagg caactatgga tgaacgaaat agacagatcg ctgagatagg tgcctcactg     5040

attaagcatt ggtaactgtc agaccaagtt tactcatata tactttagat tgatttaaaa     5100

cttcattttt aatttaaaag gatctaggtg aagatccttt ttgataatct catgaccaaa     5160

atcccttaac gtgagttttc gttccactga gcgtcagacc ccgtagaaaa gatcaaagga     5220

tcttcttgag atcctttttt tctgcgcgta atctgctgct tgcaaacaaa aaaaccaccg     5280

ctaccagcgg tggtttgttt gccggatcaa gagctaccaa ctctttttcc gaaggtaact     5340

ggcttcagca gagcgcagat accaaatact gttcttctag tgtagccgta gttaggccac     5400

cacttcaaga actctgtagc accgcctaca tacctcgctc tgctaatcct gttaccagtg     5460

gctgctgcca gtggcgataa gtcgtgtctt accgggttgg actcaagacg atagttaccg     5520

gataaggcgc agcggtcggg ctgaacgggg ggttcgtgca cacagcccag cttggagcga     5580

acgacctaca ccgaactgag atacctacag cgtgagctat gagaaagcgc cacgcttccc     5640

gaagggagaa aggcggacag gtatccggta agcggcaggg tcggaacagg agagcgcacg     5700

agggagcttc cagggggaaa cgcctggtat ctttatagtc ctgtcgggtt tcgccacctc     5760

tgacttgagc gtcgattttt gtgatgctcg tcaggggggc ggagcctatg gaaaaacgcc     5820

agcaacgcgg cctttttacg gttcctggcc ttttgctggc cttttgctca catgt          5875


<210> 26
<211> 5899
<212> DNA
<213> Artificial Sequence


<220> 
<223> coRPK AAV used for pre-clinical studies

<400> 26
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct gcggccgctg tcaccactgt ctcctgttcc attggaagcc ctgtatgcca      180

ggggccagag tccaggaacc acgggagtgc cccgtggctt acatgctgtg gctctggcct      240

gcctataggg cctggaaaag acccaggcca gggtccataa tttaacacac gggaggctct      300

gaagaacgta cgttcctctc caaaacccac ctagccagtg gctgatgtgg atcatttatg      360

ccctccaccc tggctcctag ttttcaccct cattttcctc ctatgttcca tggcttctgt      420

ctccccttct tacctcctgg agccccaatc aggatggact ttgctaagtc tctttgggac      480

ttagagaccc atgaccgaag ctgcagggat gatatgttct cctggatcga catgctttca      540

gtgtgggcct ggggctgcgg gacccataga gcccaccgca tccccagcat gcctgctatt      600

gtcttcccaa tcctccccct tgctgtcctg ccccacccca ccccccagaa tagaatgaca      660

cctactcaga caatgcgatg caatttcctc attttattag gaaaggacag tgggagtggc      720

accttccagg gtcaaggaag gcacggggga ggggcaaaca acagatggct ggcaactaga      780

aggcacagtg ctttatttgt aactcacgcg ttcatttatc gtcatcgtct ttgtagtcgc      840

tgatggacag caccctcatg atgttggtgt agccgctgcc aggccgccag cctgtcacca      900

cgatcaccag gtcgcccact ctcaggaagc cccgcagctt gccgctctcg atgccgaact      960

gcacccgtct gtccacgtcg tcggcccaga tggcctcggg aggctcccgg tacagcaggg     1020

ggaacacgcc tctgcacagg tggacctgtc tagcggcctg ggcggatctt gtcacggcga     1080

tcacggcggc tctgggtctg tatctggaca gcagctgggc gcttctgcct gtggtggtca     1140

gcacgatgat tgcagcggcg cagcacttga aggcggcttc cacggctccg atggcggtca     1200

cttcggtggg atctctgctc agaggggcgg ctctccgcag ttcctcgaac agctgccggt     1260

ggtacacggc ggcctcggct tctctggcaa tggcgtgctg catcttcacg gcctccacgg     1320

ggaagttgcc cttggctgtc tcgccggaca gcatgatgca gtcagcgcca tccagcacgg     1380

cgttggccac gtcgcttgtc tcggctctgg tgggtctggg cttggtgatc atgctttcca     1440

gcatctgggt ggcgcacacc acaggtttgc cggccaggtt gcaccgtccg atcatcattt     1500

tctgggccag gaacaccttc tcggcgggga tctcgatgcc caggtcgcct ctggccacca     1560

tgatgccgtc ggacacttcc aggatctcgt cgaaccgctt cacgccctcg tggttctcga     1620

tcttgctgat gatcttgatg ccgtggcctt cagggcccag agcggctctc acggcggcca     1680

catcagaggc cttccgcacg aagctggcga acacgatgtc cacgccgtgc tccacgccaa     1740

atctcaggtc tctcacgtcc tgctcagaca ggccaggcag gtccacctgg gcgcctggca     1800

gattcacgcc ctttctgctg cccagcacgc cgccattctc gacctgggtc accaggccct     1860

caggtccgat cttctgcacc accaggctga tcaggccgtc gtcgatgtag attctgccgc     1920

ccacaggcac cacccgcacg atgttggggt agtccaccca cactgtgttg gcgttgcctc     1980

tggttctgaa ggcggggtcc acggtcacca gcacttggct gcccttcacc agctccacct     2040

cgctctcagg ccctccctgc agaattcctg ttctgatctc ggggcccttg gtgtccaggg     2100

caatggccac gggtctgtag ctcagggggc tgccggcaaa gctctccacg gcctcccgga     2160

cgttggcaat gctctctgcg tggtactcgt ggctgccgtg ggagaagttc agccgggcga     2220

tattcatgcc ggccttgatc atctctttca gccgctccac gcttctgctg gcagggccga     2280

tggtggcaat gatgctggtg cttctggcgg ccacgggctc agagtcgatg tccagcaggc     2340

acaggtgttc cagaaaggtg tcggccatgg cggctggcag ctgctgctgc tggaaaaagg     2400

cggtgcccag ttcctgggtc agctgggcca cgctagccct tctcaggtat ccggctggtc     2460

cgccaggggc tccgatcagg atgctcttgg ccaggtctct ctggctcttg gacacccagg     2520

accgcagctg cagagagctg atattttcct ggatgctcat gctttcagtg tgggcctggg     2580

gctgcgggac catggaagag agggagagga tgacaaaact gctggtctta tctaagggag     2640

acagagaaga gaaaaggggc acacccagta ggccaccctg tccccacaga atccctcccc     2700

cagaacggcc tgctctctgc cctcatctcc tggcatttcc tctcatcctt ttttcctgat     2760

aaattttcaa tccattcata ctatctggtc atccacgtga atagatattt tttttttggc     2820

cagtcatatg gccccatttt ctttgtactt tactgaagtt agctctagtg aatccaggga     2880

gcaggggctg tagggtgggg ctggagcctg aagaaagaca aaagggatca ctgtgataat     2940

atggtggggg gagggttacc cagttctgac cacttttttt ctctgtctca accaagaaat     3000

gcagagtgcc ttcaccactc tggcggccgc aggaacccct agtgatggag ttggccactc     3060

cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc cgacgcccgg     3120

gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag ctgcctgcag gggcgcctga     3180

tgcggtattt tctccttacg catctgtgcg gtatttcaca ccgcatacgt caaagcaacc     3240

atagtacgcg ccctgtagcg gcgcattaag cgcggcgggt gtggtggtta cgcgcagcgt     3300

gaccgctaca cttgccagcg ccctagcgcc cgctcctttc gctttcttcc cttcctttct     3360

cgccacgttc gccggctttc cccgtcaagc tctaaatcgg gggctccctt tagggttccg     3420

atttagtgct ttacggcacc tcgaccccaa aaaacttgat ttgggtgatg gttcacgtag     3480

tgggccatcg ccctgataga cggtttttcg ccctttgacg ttggagtcca cgttctttaa     3540

tagtggactc ttgttccaaa ctggaacaac actcaaccct atctcgggct attcttttga     3600

tttataaggg attttgccga tttcggccta ttggttaaaa aatgagctga tttaacaaaa     3660

atttaacgcg aattttaaca aaatattaac gtttacaatt ttatggtgca ctctcagtac     3720

aatctgctct gatgccgcat agttaagcca gccccgacac ccgccaacac ccgctgacgc     3780

gccctgacgg gcttgtctgc tcccggcatc cgcttacaga caagctgtga ccgtctccgg     3840

gagctgcatg tgtcagaggt tttcaccgtc atcaccgaaa cgcgcgagac gaaagggcct     3900

cgtgatacgc ctatttttat aggttaatgt catgataata atggtttctt agacaaacct     3960

agatattgat agtctgatcg gtcaacgtat aatcgagtcc tagcttttgc aaacatctat     4020

caagagacag gatcagcagg aggctttcgc atgattgaac aagatggatt gcacgcaggt     4080

tctccggcgg cttgggtgga gaggctattc ggctatgact gggcacaaca gacaatcggc     4140

tgctctgatg ccgccgtgtt ccggctgtca gcgcaggggc gtccggttct ttttgtcaag     4200

accgacctgt ccggtgccct gaatgaactg caagacgagg cagcgcggct atcgtggctg     4260

gcgacgacgg gcgttccttg cgcggctgtg ctcgacgttg tcactgaagc gggaagggac     4320

tggctgctat tgggcgaagt gccggggcag gatctcctgt catctcacct tgctcctgcc     4380

gagaaagtat ccatcatggc tgatgcaatg cggcggctgc atacgcttga tccggctacc     4440

tgcccattcg accaccaagc gaaacatcgc atcgagcgag cacgtactcg gatggaagcc     4500

ggtcttgtcg atcaggatga tctggacgaa gagcatcagg ggctcgcgcc agccgaactg     4560

ttcgccaggc tcaaggcgtc tatgcccgac ggcgaggatc tcgtcgtgac ccacggcgat     4620

gcctgcttgc cgaatatcat ggtggaaaat ggccgctttt ctggattcat cgactgtggc     4680

cgtctgggtg tggcggaccg ctatcaggac atagcgttgg ctacccgtga tattgctgaa     4740

gagcttggcg gcgaatgggc tgaccgcttc cttgtgcttt acggtatcgc cgcgcccgat     4800

tcgcagcgca tcgccttcta tcgccttctt gacgagttct tctgaccgat tctaggtgca     4860

ttggcgcaga aaaaaatgcc tgatgcgacg ctgcgcgtct tatactccca catatgccag     4920

attcagcaac ggatacggct tccccaactt gcccacttcc atacgtgtcc tccttaccag     4980

aaatttatcc ttaacgatcg gacggggagt caggcaacta tggatgaacg aaatagacag     5040

atcgctgaga taggtgcctc actgattaag cattggtaac tgtcagacca agtttactca     5100

tatatacttt agattgattt aaaacttcat ttttaattta aaaggatcta ggtgaagatc     5160

ctttttgata atctcatgac caaaatccct taacgtgagt tttcgttcca ctgagcgtca     5220

gaccccgtag aaaagatcaa aggatcttct tgagatcctt tttttctgcg cgtaatctgc     5280

tgcttgcaaa caaaaaaacc accgctacca gcggtggttt gtttgccgga tcaagagcta     5340

ccaactcttt ttccgaaggt aactggcttc agcagagcgc agataccaaa tactgttctt     5400

ctagtgtagc cgtagttagg ccaccacttc aagaactctg tagcaccgcc tacatacctc     5460

gctctgctaa tcctgttacc agtggctgct gccagtggcg ataagtcgtg tcttaccggg     5520

ttggactcaa gacgatagtt accggataag gcgcagcggt cgggctgaac ggggggttcg     5580

tgcacacagc ccagcttgga gcgaacgacc tacaccgaac tgagatacct acagcgtgag     5640

ctatgagaaa gcgccacgct tcccgaaggg agaaaggcgg acaggtatcc ggtaagcggc     5700

agggtcggaa caggagagcg cacgagggag cttccagggg gaaacgcctg gtatctttat     5760

agtcctgtcg ggtttcgcca cctctgactt gagcgtcgat ttttgtgatg ctcgtcaggg     5820

gggcggagcc tatggaaaaa cgccagcaac gcggcctttt tacggttcct ggccttttgc     5880

tggccttttg ctcacatgt                                                  5899


<210> 27
<211> 5875
<212> DNA
<213> Artificial Sequence


<220> 
<223> coRPK AAV for clinical use

<400> 27
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct gcggccgctg tcaccactgt ctcctgttcc attggaagcc ctgtatgcca      180

ggggccagag tccaggaacc acgggagtgc cccgtggctt acatgctgtg gctctggcct      240

gcctataggg cctggaaaag acccaggcca gggtccataa tttaacacac gggaggctct      300

gaagaacgta cgttcctctc caaaacccac ctagccagtg gctgatgtgg atcatttatg      360

ccctccaccc tggctcctag ttttcaccct cattttcctc ctatgttcca tggcttctgt      420

ctccccttct tacctcctgg agccccaatc aggatggact ttgctaagtc tctttgggac      480

ttagagaccc atgaccgaag ctgcagggat gatatgttct cctggatcga catgctttca      540

gtgtgggcct ggggctgcgg gacccataga gcccaccgca tccccagcat gcctgctatt      600

gtcttcccaa tcctccccct tgctgtcctg ccccacccca ccccccagaa tagaatgaca      660

cctactcaga caatgcgatg caatttcctc attttattag gaaaggacag tgggagtggc      720

accttccagg gtcaaggaag gcacggggga ggggcaaaca acagatggct ggcaactaga      780

aggcacagtg ctttatttgt aactcacgcg ttcagctgat ggacagcacc ctcatgatgt      840

tggtgtagcc gctgccaggc cgccagcctg tcaccacgat caccaggtcg cccactctca      900

ggaagccccg cagcttgccg ctctcgatgc cgaactgcac ccgtctgtcc acgtcgtcgg      960

cccagatggc ctcgggaggc tcccggtaca gcagggggaa cacgcctctg cacaggtgga     1020

cctgtctagc ggcctgggcg gatcttgtca cggcgatcac ggcggctctg ggtctgtatc     1080

tggacagcag ctgggcgctt ctgcctgtgg tggtcagcac gatgattgca gcggcgcagc     1140

acttgaaggc ggcttccacg gctccgatgg cggtcacttc ggtgggatct ctgctcagag     1200

gggcggctct ccgcagttcc tcgaacagct gccggtggta cacggcggcc tcggcttctc     1260

tggcaatggc gtgctgcatc ttcacggcct ccacggggaa gttgcccttg gctgtctcgc     1320

cggacagcat gatgcagtca gcgccatcca gcacggcgtt ggccacgtcg cttgtctcgg     1380

ctctggtggg tctgggcttg gtgatcatgc tttccagcat ctgggtggcg cacaccacag     1440

gtttgccggc caggttgcac cgtccgatca tcattttctg ggccaggaac accttctcgg     1500

cggggatctc gatgcccagg tcgcctctgg ccaccatgat gccgtcggac acttccagga     1560

tctcgtcgaa ccgcttcacg ccctcgtggt tctcgatctt gctgatgatc ttgatgccgt     1620

ggccttcagg gcccagagcg gctctcacgg cggccacatc agaggccttc cgcacgaagc     1680

tggcgaacac gatgtccacg ccgtgctcca cgccaaatct caggtctctc acgtcctgct     1740

cagacaggcc aggcaggtcc acctgggcgc ctggcagatt cacgcccttt ctgctgccca     1800

gcacgccgcc attctcgacc tgggtcacca ggccctcagg tccgatcttc tgcaccacca     1860

ggctgatcag gccgtcgtcg atgtagattc tgccgcccac aggcaccacc cgcacgatgt     1920

tggggtagtc cacccacact gtgttggcgt tgcctctggt tctgaaggcg gggtccacgg     1980

tcaccagcac ttggctgccc ttcaccagct ccacctcgct ctcaggccct ccctgcagaa     2040

ttcctgttct gatctcgggg cccttggtgt ccagggcaat ggccacgggt ctgtagctca     2100

gggggctgcc ggcaaagctc tccacggcct cccggacgtt ggcaatgctc tctgcgtggt     2160

actcgtggct gccgtgggag aagttcagcc gggcgatatt catgccggcc ttgatcatct     2220

ctttcagccg ctccacgctt ctgctggcag ggccgatggt ggcaatgatg ctggtgcttc     2280

tggcggccac gggctcagag tcgatgtcca gcaggcacag gtgttccaga aaggtgtcgg     2340

ccatggcggc tggcagctgc tgctgctgga aaaaggcggt gcccagttcc tgggtcagct     2400

gggccacgct agcccttctc aggtatccgg ctggtccgcc aggggctccg atcaggatgc     2460

tcttggccag gtctctctgg ctcttggaca cccaggaccg cagctgcaga gagctgatat     2520

tttcctggat gctcatgctt tcagtgtggg cctggggctg cgggaccatg gaagagaggg     2580

agaggatgac aaaactgctg gtcttatcta agggagacag agaagagaaa aggggcacac     2640

ccagtaggcc accctgtccc cacagaatcc ctcccccaga acggcctgct ctctgccctc     2700

atctcctggc atttcctctc atcctttttt cctgataaat tttcaatcca ttcatactat     2760

ctggtcatcc acgtgaatag atattttttt tttggccagt catatggccc cattttcttt     2820

gtactttact gaagttagct ctagtgaatc cagggagcag gggctgtagg gtggggctgg     2880

agcctgaaga aagacaaaag ggatcactgt gataatatgg tggggggagg gttacccagt     2940

tctgaccact ttttttctct gtctcaacca agaaatgcag agtgccttca ccactctggc     3000

ggccgcagga acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca     3060

ctgaggccgg gcgaccaaag gtcgcccgac gcccgggctt tgcccgggcg gcctcagtga     3120

gcgagcgagc gcgcagctgc ctgcaggggc gcctgatgcg gtattttctc cttacgcatc     3180

tgtgcggtat ttcacaccgc atacgtcaaa gcaaccatag tacgcgccct gtagcggcgc     3240

attaagcgcg gcgggtgtgg tggttacgcg cagcgtgacc gctacacttg ccagcgccct     3300

agcgcccgct cctttcgctt tcttcccttc ctttctcgcc acgttcgccg gctttccccg     3360

tcaagctcta aatcgggggc tccctttagg gttccgattt agtgctttac ggcacctcga     3420

ccccaaaaaa cttgatttgg gtgatggttc acgtagtggg ccatcgccct gatagacggt     3480

ttttcgccct ttgacgttgg agtccacgtt ctttaatagt ggactcttgt tccaaactgg     3540

aacaacactc aaccctatct cgggctattc ttttgattta taagggattt tgccgatttc     3600

ggcctattgg ttaaaaaatg agctgattta acaaaaattt aacgcgaatt ttaacaaaat     3660

attaacgttt acaattttat ggtgcactct cagtacaatc tgctctgatg ccgcatagtt     3720

aagccagccc cgacacccgc caacacccgc tgacgcgccc tgacgggctt gtctgctccc     3780

ggcatccgct tacagacaag ctgtgaccgt ctccgggagc tgcatgtgtc agaggttttc     3840

accgtcatca ccgaaacgcg cgagacgaaa gggcctcgtg atacgcctat ttttataggt     3900

taatgtcatg ataataatgg tttcttagac aaacctagat attgatagtc tgatcggtca     3960

acgtataatc gagtcctagc ttttgcaaac atctatcaag agacaggatc agcaggaggc     4020

tttcgcatga ttgaacaaga tggattgcac gcaggttctc cggcggcttg ggtggagagg     4080

ctattcggct atgactgggc acaacagaca atcggctgct ctgatgccgc cgtgttccgg     4140

ctgtcagcgc aggggcgtcc ggttcttttt gtcaagaccg acctgtccgg tgccctgaat     4200

gaactgcaag acgaggcagc gcggctatcg tggctggcga cgacgggcgt tccttgcgcg     4260

gctgtgctcg acgttgtcac tgaagcggga agggactggc tgctattggg cgaagtgccg     4320

gggcaggatc tcctgtcatc tcaccttgct cctgccgaga aagtatccat catggctgat     4380

gcaatgcggc ggctgcatac gcttgatccg gctacctgcc cattcgacca ccaagcgaaa     4440

catcgcatcg agcgagcacg tactcggatg gaagccggtc ttgtcgatca ggatgatctg     4500

gacgaagagc atcaggggct cgcgccagcc gaactgttcg ccaggctcaa ggcgtctatg     4560

cccgacggcg aggatctcgt cgtgacccac ggcgatgcct gcttgccgaa tatcatggtg     4620

gaaaatggcc gcttttctgg attcatcgac tgtggccgtc tgggtgtggc ggaccgctat     4680

caggacatag cgttggctac ccgtgatatt gctgaagagc ttggcggcga atgggctgac     4740

cgcttccttg tgctttacgg tatcgccgcg cccgattcgc agcgcatcgc cttctatcgc     4800

cttcttgacg agttcttctg accgattcta ggtgcattgg cgcagaaaaa aatgcctgat     4860

gcgacgctgc gcgtcttata ctcccacata tgccagattc agcaacggat acggcttccc     4920

caacttgccc acttccatac gtgtcctcct taccagaaat ttatccttaa cgatcggacg     4980

gggagtcagg caactatgga tgaacgaaat agacagatcg ctgagatagg tgcctcactg     5040

attaagcatt ggtaactgtc agaccaagtt tactcatata tactttagat tgatttaaaa     5100

cttcattttt aatttaaaag gatctaggtg aagatccttt ttgataatct catgaccaaa     5160

atcccttaac gtgagttttc gttccactga gcgtcagacc ccgtagaaaa gatcaaagga     5220

tcttcttgag atcctttttt tctgcgcgta atctgctgct tgcaaacaaa aaaaccaccg     5280

ctaccagcgg tggtttgttt gccggatcaa gagctaccaa ctctttttcc gaaggtaact     5340

ggcttcagca gagcgcagat accaaatact gttcttctag tgtagccgta gttaggccac     5400

cacttcaaga actctgtagc accgcctaca tacctcgctc tgctaatcct gttaccagtg     5460

gctgctgcca gtggcgataa gtcgtgtctt accgggttgg actcaagacg atagttaccg     5520

gataaggcgc agcggtcggg ctgaacgggg ggttcgtgca cacagcccag cttggagcga     5580

acgacctaca ccgaactgag atacctacag cgtgagctat gagaaagcgc cacgcttccc     5640

gaagggagaa aggcggacag gtatccggta agcggcaggg tcggaacagg agagcgcacg     5700

agggagcttc cagggggaaa cgcctggtat ctttatagtc ctgtcgggtt tcgccacctc     5760

tgacttgagc gtcgattttt gtgatgctcg tcaggggggc ggagcctatg gaaaaacgcc     5820

agcaacgcgg cctttttacg gttcctggcc ttttgctggc cttttgctca catgt          5875


<210> 28
<211> 218
<212> DNA
<213> Homo sapiens


<220> 
<223> RPK CDS (Fig 1)

<400> 28
gttctggggg agggattctg tggggacagg gtggcctact gggtgtgccc cttttctctt     60

ctctgtctcc cttagataag accagcagtt ttgtcatcct ctccctctca ttccatggtc    120

ccgcagcccc aggcccacac tgaaagcatg tcgatccagg agaacatatc atccctgcag    180

cttcggtcat gggtctctaa gtcccaaaga gacttagc                            218


<210> 29
<211> 107
<212> DNA
<213> Artificial Sequence


<220> 
<223> PKLR locus (Fig 7)

<400> 29
tctgtctccc ttagataaga ccagcagttt tgtcatcctc tccctctcat tccatggtcc     60

cgcagcccca ggcccacact gaaagcatgt cgatccagga gaacata                  107


<210> 30
<211> 88
<212> DNA
<213> Artificial Sequence


<220> 
<223> Genomic region of the starting site of the RPK transcript variant
      (Fig 8, 9, 10, 11)

<400> 30
aagaccagca gttttgtcat cctctccctc tcattccatg gtcccgcagc cccaggccca     60

cactgaaagc atgtcgatcc aggagaac                                        88


<210> 31
<211> 158
<212> DNA
<213> Artificial Sequence


<220> 
<223> 5’UTR position with respect to LHA and coRPK without start condon
      in coRPK- AAV vector (Fig 12)

<400> 31
agggtggcct actgggtgtg ccccttttct cttctctgtc tcccttagat aagaccagca     60

gttttgtcat cctctccctc tcttccatgg tcccgcagcc ccaggcccac actgaaagca    120

tgagcatcca ggaaaatatc agctctctgc agctgcgg                            158


<210> 32
<211> 154
<212> DNA
<213> Artificial Sequence


<220> 
<223> FLAG-Tag position with respect to coRPK without STOP condon and
      bGH poly(A) signal in coRPK-AAV vector (Fig 13)

<400> 32
gcgacctggt gatcgtggtg acaggctggc ggcctggcag cggctacacc aacatcatga     60

gggtgctgtc catcagcgac tacaaagacg atgacgataa atgaacgcgt gagttacaaa    120

taaagcactg tgccttctag ttgccagcca tctg                                154


<210> 33
<211> 480
<212> DNA
<213> Artificial Sequence


<220> 
<223> bGH poly(A) signal with respect to coRPK-FLAG and RHA in
      coRPK-AAV vector (Fig 14)

<400> 33
agtgggcgac ctggtgatcg tggtgacagg ctggcggcct ggcagcggct acaccaacat      60

catgagggtg ctgtccatca gcgactacaa agacgatgac gataaatgaa cgcgtgagtt     120

acaaataaag cactgtgcct tctagttgcc agccatctgt tgtttgcccc tcccccgtgc     180

cttccttgac cctggaaggt gccactccca ctgtcctttc ctaataaaat gaggaaattg     240

catcgcattg tctgagtagg tgtcattcta ttctgggggg tggggtgggg caggacagca     300

agggggagga ttgggaagac aatagcaggc atgctgggga tgcggtgggc tctatgggtc     360

ccgcagcccc aggcccacac tgaaagcatg tcgatccagg agaacatatc atccctgcag     420

cttcggtcat gggtctctaa gtcccaaaga gacttagcaa agtccatcct gattggggct     480


<210> 34
<211> 23
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. RPK (Fig 1)

<400> 34
Met Ser Ile Gln Glu Asn Ile Ser Ser Leu Gln Leu Arg Ser Trp Val 
1               5                   10                  15      
Ser Lys Ser Gln Arg Asp Leu 
            20              

<210> 35
<211> 6
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. RPK (Figs 8-11)

<400> 35
Met Ser Ile Gln Glu Asn 
1               5       

<210> 36
<211> 35
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. from aminoacids 748 to 782 (Fig 14)

<400> 36
Val Gly Asp Leu Val Ile Val Val Thr Gly Trp Arg Pro Gly Ser Gly 
1               5                   10                  15      
Tyr Thr Asn Ile Met Arg Val Leu Ser Ile Ser Asp Tyr Lys Asp Asp 
            20                  25                  30          
Asp Asp Lys 
        35  

<210> 37
<211> 11
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. from aminoacids 784 to 794 (Fig 14)

<400> 37
Thr Arg Glu Leu Gln Ile Lys His Cys Ala Phe 
1               5                   10      

<210> 38
<211> 28
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. from aminoacids 796 to 823 (Fig 14)

<400> 38
Leu Pro Ala Ile Cys Cys Leu Pro Leu Pro Arg Ala Phe Leu Asp Pro 
1               5                   10                  15      
Gly Arg Cys His Ser His Cys Pro Phe Leu Ile Lys 
            20                  25              

<210> 39
<211> 8
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. from aminoacids 825 to 832 (Fig 14)

<400> 39
Gly Asn Cys Ile Ala Leu Ser Glu 
1               5               

<210> 40
<211> 20
<212> PRT
<213> Artificial Sequence


<220> 
<223> Aminoacid seq. from aminoacids 834 to 853 (Fig 14)

<400> 40
Val Ser Phe Tyr Ser Gly Gly Trp Gly Gly Ala Gly Gln Gln Gly Gly 
1               5                   10                  15      
Gly Leu Gly Arg 
            20  

