LISTADO DE SECUENCIAS <110> AveXis Inc. <120> MEDIOS Y MÉTODO PARA PREPARAR VECTORES VIRALES Y USOS DE LOS MISMOS <130> AVEX-003/001WO <150> 62/583,035 <151> 2017-11-08 <160> 4 <170> PatentIn version 3.5 <210> 1 <211> 2359 <212> ADN <213> Artificial <220> <223> constructo de vector <400> 1 ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt 60 ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggaatt cacgcgtgga 120 tctgaattca attcacgcgt ggtacctctg gtcgttacat aacttacggt aaatggcccg 180 cctggctgac cgcccaacga cccccgccca ttgacgtcaa taatgacgta tgttcccata 240 gtaacgccaa tagggacttt ccattgacgt caatgggtgg agtatttacg gtaaactgcc 300 cacttggcag tacatcaagt gtatcatatg ccaagtacgc cccctattga cgtcaatgac 360 ggtaaatggc ccgcctggca ttatgcccag tacatgacct tatgggactt tcctacttgg 420 cagtacatct actcgaggcc acgttctgct tcactctccc catctccccc ccctccccac 480 ccccaatttt gtatttattt attttttaat tattttgtgc agcgatgggg gcgggggggg 540 ggggggggcg cgcgccaggc ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga 600 gaggtgcggc ggcagccaat cagagcggcg cgctccgaaa gtttcctttt atggcgaggc 660 ggcggcggcg gcggccctat aaaaagcgaa gcgcgcggcg ggcgggagcg ggatcagcca 720 ccgcggtggc ggcctagagt cgacgaggaa ctgaaaaacc agaaagttaa ctggtaagtt 780 tagtcttttt gtcttttatt tcaggtcccg gatccggtgg tggtgcaaat caaagaactg 840 ctcctcagtg gatgttgcct ttacttctag gcctgtacgg aagtgttact tctgctctaa 900 aagctgcgga attgtacccg cggccgatcc accggtccgg aattcccggg atatcgtcga 960 cccacgcgtc cgggccccac gctgcgcacc cgcgggtttg ctatggcgat gagcagcggc 1020 ggcagtggtg gcggcgtccc ggagcaggag gattccgtgc tgttccggcg cggcacaggc 1080 cagagcgatg attctgacat ttgggatgat acagcactga taaaagcata tgataaagct 1140 gtggcttcat ttaagcatgc tctaaagaat ggtgacattt gtgaaacttc gggtaaacca 1200 aaaaccacac ctaaaagaaa acctgctaag aagaataaaa gccaaaagaa gaatactgca 1260 gcttccttac aacagtggaa agttggggac aaatgttctg ccatttggtc agaagacggt 1320 tgcatttacc cagctaccat tgcttcaatt gattttaaga gagaaacctg tgttgtggtt 1380 tacactggat atggaaatag agaggagcaa aatctgtccg atctactttc cccaatctgt 1440 gaagtagcta ataatataga acagaatgct caagagaatg aaaatgaaag ccaagtttca 1500 acagatgaaa gtgagaactc caggtctcct ggaaataaat cagataacat caagcccaaa 1560 tctgctccat ggaactcttt tctccctcca ccacccccca tgccagggcc aagactggga 1620 ccaggaaagc caggtctaaa attcaatggc ccaccaccgc caccgccacc accaccaccc 1680 cacttactat catgctggct gcctccattt ccttctggac caccaataat tcccccacca 1740 cctcccatat gtccagattc tcttgatgat gctgatgctt tgggaagtat gttaatttca 1800 tggtacatga gtggctatca tactggctat tatatgggtt ttagacaaaa tcaaaaagaa 1860 ggaaggtgct cacattcctt aaattaagga gaaatgctgg catagagcag cactaaatga 1920 caccactaaa gaaacgatca gacagatcta gaaagcttat cgataccgtc gactagagct 1980 cgctgatcag cctcgactgt gccttctagt tgccagccat ctgttgtttg cccctccccc 2040 gtgccttcct tgaccctgga aggtgccact cccactgtcc tttcctaata aaatgaggaa 2100 attgcatcgc attgtctgag taggtgtcat tctattctgg ggggtggggt ggggcaggac 2160 agcaaggggg aggattggga agacaatagc aggcatgctg gggagagatc gatctgagga 2220 acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca ctgaggccgg 2280 gcgaccaaag gtcgcccgac gcccgggctt tgcccgggcg gcctcagtga gcgagcgagc 2340 gcgcagagag ggagtggcc 2359 <210> 2 <211> 294 <212> PRT <213> Homo sapiens <400> 2 Met Ala Met Ser Ser Gly Gly Ser Gly Gly Gly Val Pro Glu Gln Glu 1 5 10 15 Asp Ser Val Leu Phe Arg Arg Gly Thr Gly Gln Ser Asp Asp Ser Asp 20 25 30 Ile Trp Asp Asp Thr Ala Leu Ile Lys Ala Tyr Asp Lys Ala Val Ala 35 40 45 Ser Phe Lys His Ala Leu Lys Asn Gly Asp Ile Cys Glu Thr Ser Gly 50 55 60 Lys Pro Lys Thr Thr Pro Lys Arg Lys Pro Ala Lys Lys Asn Lys Ser 65 70 75 80 Gln Lys Lys Asn Thr Ala Ala Ser Leu Gln Gln Trp Lys Val Gly Asp 85 90 95 Lys Cys Ser Ala Ile Trp Ser Glu Asp Gly Cys Ile Tyr Pro Ala Thr 100 105 110 Ile Ala Ser Ile Asp Phe Lys Arg Glu Thr Cys Val Val Val Tyr Thr 115 120 125 Gly Tyr Gly Asn Arg Glu Glu Gln Asn Leu Ser Asp Leu Leu Ser Pro 130 135 140 Ile Cys Glu Val Ala Asn Asn Ile Glu Gln Asn Ala Gln Glu Asn Glu 145 150 155 160 Asn Glu Ser Gln Val Ser Thr Asp Glu Ser Glu Asn Ser Arg Ser Pro 165 170 175 Gly Asn Lys Ser Asp Asn Ile Lys Pro Lys Ser Ala Pro Trp Asn Ser 180 185 190 Phe Leu Pro Pro Pro Pro Pro Met Pro Gly Pro Arg Leu Gly Pro Gly 195 200 205 Lys Pro Gly Leu Lys Phe Asn Gly Pro Pro Pro Pro Pro Pro Pro Pro 210 215 220 Pro Pro His Leu Leu Ser Cys Trp Leu Pro Pro Phe Pro Ser Gly Pro 225 230 235 240 Pro Ile Ile Pro Pro Pro Pro Pro Ile Cys Pro Asp Ser Leu Asp Asp 245 250 255 Ala Asp Ala Leu Gly Ser Met Leu Ile Ser Trp Tyr Met Ser Gly Tyr 260 265 270 His Thr Gly Tyr Tyr Met Gly Phe Arg Gln Asn Gln Lys Glu Gly Arg 275 280 285 Cys Ser His Ser Leu Asn 290 <210> 3 <211> 736 <212> PRT <213> Virus adeno asociado 9 <400> 3 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> 4 <211> 1621 <212> ADN <213> Homo sapiens <400> 4 ccacaaatgt gggagggcga taaccactcg tagaaagcgt gagaagttac tacaagcggt 60 cctcccggcc accgtactgt tccgctccca gaagccccgg gcggcggaag tcgtcactct 120 taagaaggga cggggcccca cgctgcgcac ccgcgggttt gctatggcga tgagcagcgg 180 cggcagtggt ggcggcgtcc cggagcagga ggattccgtg ctgttccggc gcggcacagg 240 ccagagcgat gattctgaca tttgggatga tacagcactg ataaaagcat atgataaagc 300 tgtggcttca tttaagcatg ctctaaagaa tggtgacatt tgtgaaactt cgggtaaacc 360 aaaaaccaca cctaaaagaa aacctgctaa gaagaataaa agccaaaaga agaatactgc 420 agcttcctta caacagtgga aagttgggga caaatgttct gccatttggt cagaagacgg 480 ttgcatttac ccagctacca ttgcttcaat tgattttaag agagaaacct gtgttgtggt 540 ttacactgga tatggaaata gagaggagca aaatctgtcc gatctacttt ccccaatctg 600 tgaagtagct aataatatag aacagaatgc tcaagagaat gaaaatgaaa gccaagtttc 660 aacagatgaa agtgagaact ccaggtctcc tggaaataaa tcagataaca tcaagcccaa 720 atctgctcca tggaactctt ttctccctcc accacccccc atgccagggc caagactggg 780 accaggaaag ccaggtctaa aattcaatgg cccaccaccg ccaccgccac caccaccacc 840 ccacttacta tcatgctggc tgcctccatt tccttctgga ccaccaataa ttcccccacc 900 acctcccata tgtccagatt ctcttgatga tgctgatgct ttgggaagta tgttaatttc 960 atggtacatg agtggctatc atactggcta ttatatgggt ttcagacaaa atcaaaaaga 1020 aggaaggtgc tcacattcct taaattaagg agaaatgctg gcatagagca gcactaaatg 1080 acaccactaa agaaacgatc agacagatct ggaatgtgaa gcgttataga agataactgg 1140 cctcatttct tcaaaatatc aagtgttggg aaagaaaaaa ggaagtggaa tgggtaactc 1200 ttcttgatta aaagttatgt aataaccaaa tgcaatgtga aatattttac tggactcttt 1260 tgaaaaacca tctgtaaaag actggggtgg gggtgggagg ccagcacggt ggtgaggcag 1320 ttgagaaaat ttgaatgtgg attagatttt gaatgatatt ggataattat tggtaatttt 1380 atggcctgtg agaagggtgt tgtagtttat aaaagactgt cttaatttgc atacttaagc 1440 atttaggaat gaagtgttag agtgtcttaa aatgtttcaa atggtttaac aaaatgtatg 1500 tgaggcgtat gtggcaaaat gttacagaat ctaactggtg gacatggctg ttcattgtac 1560 tgtttttttc tatcttctat atgtttaaaa gtatataata aaaatattta attttttttt 1620 a 1621