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'recombinant': [197, 1104, 2144], 'adeno-associated': [198, 408, 957, 1017, 1105, 1347, 1617, 1754, 1927, 2051, 2125, 2187], 'viral': [199, 483, 958, 1327, 1618, 1755, 2188], 'vectors': [200, 1019, 1085, 1349, 1596, 2189], 'trialsMolecular': [203], '-': [205, 880], 'Methods': [206], 'Clinical': [208, 609, 1002, 2113, 2220], 'Development,': [209, 2110], '3Challenges': [211], 'Prospects': [213], 'Alpha-1': [215, 2320], 'Antitrypsin': [216, 2321], 'Deficiency': [217, 540], 'Joanna': [220, 227], 'Wozniak,': [221], 'Tomasz': [222], 'Wandtke,': [223], 'Piotr': [224], 'Kopinski,': [225], 'Chorostowska-Wynimko5': [228], '2015': [230], '26,': [236], '11Current': [238], 'status': [239, 1510], 'α-1': [244, 1344, 1369], 'deficiency3': [246], '2014': [248, 271], '3Efficiency': [258], 'Protease-Activatable': [260], 'Nanonodes': [262], 'Tuned': [263], 'Through': [264, 1698, 1796, 1829], 'Incorporation': [265], 'Wild-Type': [267], 'Capsid': [268], 'Subunits15': [269], 'July': [270, 2323], 'Cellular': [273, 716], 'Molecular': [275, 387, 797, 1457, 1649, 2350], 'Bioengineering,': [276, 1334], '7,': [278], '3Birth': [280], 'a': [282, 415, 493, 606, 1132, 1237, 1843, 1852, 1945, 1954, 1974, 2070, 2105, 2339, 2360], 'New': [283], 'Therapeutic': [284, 837, 2340], 'Platform:': [285], '47': [286], 'Years': [287], 'Adeno-associated': [289, 1523], 'Biology': [291], 'From': [292], 'Discovery': [294], 'Licensed': [296], 'TherapyMolecular': [298], '21,': [301, 320, 741, 784, 874], '11NADH-dehydrogenase': [303], 'Type-2': [304], 'Suppresses': [305], 'Irreversible': [306], 'Visual': [307], 'Loss': [308], 'Neurodegeneration': [310], 'EAE': [313], 'Animal': [314], 'Model': [315, 1138], 'MSMolecular': [317], '10Recombinant': [322, 407], 'Integration': [325, 1247], 'Sites': [326, 1480], 'Murine': [328, 1133, 1230, 1257, 1679, 1777, 1810], 'After': [330, 1037, 1254], 'Ornithine': [331], 'Transcarbamylase': [332], 'Correction': [334, 1841, 1943], 'Li': [335, 732, 768, 849, 1147, 1189, 1263, 1700], 'Zhong,': [336, 769, 850, 1148, 1190, 1264, 1701], 'Nirav': [337], 'Malani,': [338], 'Mengxin': [339, 718, 857], 'Li,': [340, 348, 673, 719, 724, 858, 860, 1144, 1192, 1270, 1703, 1705, 1711, 1727], 'Troy': [341], 'Brady,': [342], 'Jun': [343], 'Xie,': [344], 'Peter': [345], 'Bell,': [346], 'Shaoyong': [347], 'Haven': [349], 'Jones,': [350], 'James': [351, 611, 1058, 2287], 'M.': [352, 619, 634, 762, 2015, 2272, 2275, 2288], 'Wilson,': [353], 'Terence': [354, 556, 1053, 1481, 1877, 2170], 'Flotte,': [356, 1055, 1483], 'Frederic': [357], 'D.': [358, 2000, 2024], 'Bushman,': [359], 'Guangping': [361], 'Gao26': [362], 'April': [363, 698, 814, 1092, 1108, 1164, 1219, 1454, 1495, 1621, 2244], '2013': [364], '24,': [370, 1537], '5Insulin': [372], 'from': [375, 577], 'design': [376], 'beta': [378], 'cell': [379], 'generation15': [380], 'October': [381, 886, 2115], '2012': [382], 'Reviews': [385], 'Medicine,': [388, 459, 1672, 1770], '14Preclinical': [390], 'safety': [391], 'evaluation': [392], 'subretinal': [394], 'AAV2.sFlt-1': [395], 'non-human': [397], 'primates10': [398], '2011': [400, 500, 585, 644, 686], '19,': [405, 505, 986, 1080, 1171, 1226, 1296], 'virus:': [409], 'application': [411], 'development': [413], 'as': [414, 517, 2104, 2338], 'gene-therapy': [416], 'vectorTherapeutic': [417], 'Delivery,': [418], '3,': [420], '7Sustained': [422], 'miRNA-mediated': [423], 'Knockdown': [424], 'Mutant': [426], 'AAT': [427, 433], 'With': [428], 'Simultaneous': [429], 'Augmentation': [430], 'Wild-type': [432], 'Has': [434], 'Minimal': [435], 'Effect': [436], 'Global': [438], 'miRNA': [440], 'ProfilesMolecular': [441], '3A': [446], 'Review': [447, 2318], 'α': [449, 1657], '1': [450, 1638, 1658, 1697, 1795, 1828, 2128], '-Antitrypsin': [451, 1659], 'DeficiencyAmerican': [452], 'Respiratory': [455, 794, 1668, 2347], 'Critical': [457, 1670], 'Care': [458, 1671], '185,': [461], '3Exploiting': [463], 'Natural': [464], 'Diversity': [465], 'Design': [470], 'with': [473, 492, 581, 755, 811, 1925, 1931, 2050, 2191, 2367], 'Novel': [474, 1653], 'Properties27': [475], '2011Successful': [477], 'attenuation': [478], 'humoral': [480], 'immunity': [481], 'capsid': [484, 939, 2197], 'transgenic': [486], 'protein': [487], 'following': [488], 'AAV-mediated': [489, 1233], 'transfer': [491, 1759], 'non-depleting': [494], 'CD4': [495], 'antibody': [496, 2235], 'cyclosporine30': [498], '1rAAV': [507], 'Vector': [508, 604, 844, 1047, 1252, 1467, 1476, 1525, 1695, 1793, 1826, 1856, 2109], 'Product': [509], 'Characterization': [510, 1459], 'Stability': [512], 'Studies27': [513], '2011Glucan': [515], 'Particles': [516], 'Carriers': [518], 'Nanoparticles': [520], 'Macrophage-Targeted': [522], 'Delivery7': [523], 'December': [524, 559, 943, 1959, 2093], '2012Recombinant': [525], 'Virus-Mediated': [527, 907, 2164], 'Transfer': [529, 1183, 1906, 2166, 2263], 'Potential': [532, 713], 'Adenosine': [535], 'Deaminase-Deficient': [536], 'Severe': [537], 'Combined': [538], 'Immune': [539], 'Jared': [541], 'N.': [542], 'Silver,': [543], 'Melissa': [544], 'Elder,': [545], 'Thomas': [546, 1062, 1484, 1857], 'Conlon,': [547, 1064, 1486, 1859], 'Pedro': [548], 'Cruz,': [549], 'Amy': [550, 1487], 'Wright,': [552], 'Arun': [553, 679, 729, 770, 865, 1162, 1217, 1287, 1573, 1735], 'Srivastava,': [554, 680, 730, 771], 'Flotte13': [558], '2010': [560, 735, 778, 815, 868], '22,': [566, 650, 692], '8Recombinant': [568], 'AAV2-mediated': [569], 'β-globin': [570], 'expression': [571, 969, 1585, 2236], 'human': [573, 1614], 'fetal': [574], 'hematopoietic': [575], 'cells': [576, 1921], 'aborted': [579], 'fetuses': [580], 'β-thalassemia': [582], 'major27': [583], 'International': [587], 'Hematology,': [590], '93,': [592], '6Good': [594], 'Manufacturing': [595], 'Practice': [596], 'Production': [597, 2111], 'Self-Complementary': [599, 1117, 1634], 'Serotype': [600, 760, 1045, 1120, 1901], '8': [601, 2196], 'Hemophilia': [607], 'B': [608], 'Trial': [610], 'A.': [612, 1152, 1204, 1277, 1565, 1719, 2377], 'Allay,': [613], 'Susan': [614], 'Sleep,': [615], 'Scott': [616], 'Long,': [617], 'David': [618], 'Tillman,': [620], 'Rob': [621], 'Clark,': [622], 'Gael': [623], 'Carney,': [624], 'Paolo': [625], 'Fagone,': [626], 'Jenny': [627], 'H.': [628, 1724], 'McIntosh,': [629], 'Arthur': [630], 'W.': [631, 862], 'Nienhuis,': [632], 'Andrew': [633], 'Davidoff,': [635], 'Amit': [636], 'C.': [637, 1159, 1214, 1284, 1568, 1732], 'Nathwani,': [638], 'John': [640], 'Gray17': [642], '5A': [652, 1435], 'Simple': [653], 'Method': [654], 'Increase': [656], 'Efficiency': [659], 'Single-Stranded': [661, 834], 'In': [665, 668, 1686, 1689, 1784, 1787, 1817, 1820], 'Vitro': [666, 1687, 1785, 1818], 'Vivo': [669, 1690, 1788, 1821], 'Wenqin': [670, 727], 'Ma,': [671, 728], 'Baozheng': [672, 723, 859, 1710], 'Chen': [674, 725], 'Ling,': [675, 726], 'Giridhara': [676, 720, 846], 'Jayandharan,': [678, 722, 848], 'Barry': [682, 1070, 1492], 'Byrne10': [684], 'January': [685, 867, 1329, 1387, 1428, 1646, 1763, 1831, 1880, 2030, 2055], '5Introduction': [694], 'Vectors2': [697], '2011High-Efficiency': [699], 'Fibroblasts': [702], 'Mesenchymal': [704], 'Stem': [705, 752, 1123, 1259, 1681, 1779, 1812], 'by': [707, 804, 833, 956, 970, 1302, 1346, 1616, 1633, 1691, 1789, 1822, 1848, 1922, 1950, 2047], 'Tyrosine-Mutant': [708], 'AAV2': [709, 1407, 1447, 2145], 'Their': [712], 'Use': [714], 'Zhong29': [733], '11Enhanced': [743], 'Long-Term': [744, 1128, 1630], 'Multilineage': [747], 'Engraftment': [748], 'Hematopoietic': [751, 1122, 1258, 1680, 1778, 1811], 'Transduced': [754], 'Tyrosine-Modified': [756], '2': [761, 1046, 1593], 'Ariel': [763], 'Kauss,': [764], 'Laura': [765], 'Smith,': [767], 'K.K.': [772], 'Wong,': [773], 'Saswati': [775], 'Chatterjee4': [776], '9Secretory': [786], 'Leukocyte': [787], 'Protease': [788], 'Inhibitor': [789], 'Elafin/Trappin-2American': [791], 'Cell': [795, 1124, 1684, 1782, 1815, 2348], 'Biology,': [798, 2351], '42,': [800], '6IL-10': [802], 'delivery': [803, 973], 'AAV5': [805], 'vector': [806, 938, 2155], 'attenuates': [807], 'inflammation': [808], 'mice': [810, 2046], 'pseudomonas': [812], 'pneumonia1': [813], '17,': [820, 1582, 1744, 1887], '5Optimized': [822], '(AAV)–Protein': [825], 'Phosphatase-5': [826], 'Helper': [827], 'Viruses': [828], 'Efficient': [830], 'Expression': [838, 1130, 1186, 1235, 1632, 2255], 'Factor': [840], 'IX': [841], 'at': [842, 2237], 'Reduced': [843], 'Doses': [845], 'Brandon': [851], 'K.': [852], 'Sack,': [853], 'Angela': [854, 1207], 'E.': [855, 1208, 2005], 'Rivers,': [856, 1209], 'Roland': [861], 'Herzog,': [863], 'Srivastava21': [866], '3Alpha-1': [876], '(AAT)': [878], 'deficiency': [879], 'what': [881], 'are': [882], 'options?15': [885], '2009': [887, 927, 2324], 'Pharmacotherapy,': [892], '10,': [894], '16Role': [896], 'Cyclic': [898], 'AMP-Dependent': [899], 'Kinase': [900, 1402, 1442], 'Response': [901], 'Element-Binding': [902], 'Protein': [903, 1400, 1440], 'Heart': [910], 'Jarrod': [913], 'Dean,': [914], 'Jeremy': [915], 'Plante,': [916], 'Gordon': [917], 'S.': [918, 1272, 2021], 'Huggins,': [919], 'Richard': [920], 'O.': [921], 'Snyder,': [922], 'Ryuichi': [924], 'Aikawa31': [925], '9Tailoring': [935], 'therapy4': [942], '2008': [944, 961, 982, 1000, 1026, 1074, 1093, 1109, 1165, 1220, 1290], '16,': [949, 966, 1244, 2037, 2180, 2232, 2297], '3Enhancing': [951], 'transduction': [952, 1013, 1915, 2202], 'vectors14': [959], '1GFP': [968], 'intracellular': [971], 'GFP-coding': [975], 'fragments': [976], 'using': [977, 1103, 2124, 2240], 'nanocrystal': [978], 'quantum': [979], 'dots18': [980], 'Nanotechnology,': [984], '49Human': [988], 'alpha': [989], '1-antitrypsin': [990], 'induces': [992], 'fatal': [993], 'anaphylaxis': [994], 'non-obese': [996], 'diabetic': [997], 'mice28': [998], 'Experimental': [1004], 'Immunology,': [1005], '154,': [1007], '1Strategies': [1009], 'improving': [1011], 'efficiency': [1014], 'single-stranded': [1016], 'virus': [1018, 1106, 1348, 1928, 2126], 'vitro': [1021], 'vivo22': [1024], '18Apparently': [1033], 'Nonspecific': [1034], 'Enzyme': [1035], 'Elevations': [1036], 'Portal': [1038], 'Vein': [1039], 'Delivery': [1040], 'Hepatitis': [1049], 'C': [1050], 'Virus-Infected': [1051], 'Chimpanzees': [1052], 'Jason': [1056], 'Goetzmann,': [1057], 'Caridi,': [1059], 'Joseph': [1060], 'Paolillo,': [1061], 'Mark': [1065], 'Potter,': [1066], 'Christian': [1067], 'Mueller,': [1068], 'Byrne16': [1072], '7Potential': [1082], 'metabolic': [1090], 'disease10': [1091], '11Clinical': [1100], 'vectors17': [1107], '11Recombinant': [1116], 'Vector-Mediated': [1121], 'Lineage-Restricted,': [1127], 'Transgene': [1129, 1185], 'Serial': [1134], 'Bone': [1135], 'Marrow': [1136], 'Transplantation': [1137], 'Njeri': [1139, 1187, 1265, 1712], 'Maina,': [1140, 1188, 1266, 1713], 'Zongchao': [1141, 1195, 1261], 'Han,': [1142, 1196, 1262], 'Xiaomiao': [1143, 1191, 1269, 1726], 'Zhongbo': [1145, 1267], 'Hu,': [1146, 1268], 'Daniela': [1149, 1197, 1274, 1714], 'Bischof,': [1150, 1198, 1275, 1715], 'Kirsten': [1151, 1203, 1276, 1564, 1718, 1866], 'Weigel-Van': [1153, 1205, 1278], 'Aken,': [1154, 1206, 1279], 'William': [1155, 1210, 1280, 1728], 'B.': [1156, 1211, 1281, 1729, 2380], 'Slayton,': [1157, 1212, 1282, 1730], 'Mervin': [1158, 1213, 1283, 1567, 1731], 'Yoder,': [1160, 1215, 1285, 1569, 1733], 'Srivastava25': [1163, 1218], '4Optimization': [1173], 'β-Globin': [1181], 'Weihong': [1193, 1706], 'Zhao,': [1194, 1707], 'George': [1199], 'Aslanidi,': [1200], 'Sergei': [1201], 'Zolotukhin,': [1202, 1722], '4Correction': [1228], 'PKU': [1231], 'Following': [1232], 'Intramuscular': [1234, 1849, 1951], 'Complete': [1238], 'Phenylalanine': [1239], 'Hydroxylating': [1240], 'SystemMolecular': [1241], '4Stable': [1246], 'Genomes': [1253, 1477], 'Nitin': [1271], 'Chouthai,': [1273], 'Srivastava24': [1288], '3Lack': [1298], 'Repeat': [1300], 'Type': [1306, 1465], '5/5': [1307], 'Mouse': [1311], 'AirwayJournal': [1312], 'Virology,': [1314], '81,': [1316], '22Library': [1318], 'selection': [1319], 'directed': [1321], 'evolution': [1322], 'approaches': [1323], 'engineering': [1325], 'targeted': [1326], 'vectors1': [1328], '2007': [1330, 1353, 1388, 1429, 1455, 1496], 'Biotechnology': [1332], '98,': [1336], '3In': [1338], 'vivo': [1339], 'post-transcriptional': [1340], 'silencing': [1342], 'expressing': [1350], 'siRNA25': [1351], 'Laboratory': [1355], 'Investigation,': [1356], '87,': [1358], '9The': [1360], 'promise': [1361], 'deficiencyPharmacogenomics,': [1371], '9Efficiency': [1375], 'eight': [1377], 'different': [1378], 'serotypes': [1380], 'transducing': [1382], 'rat': [1383], 'myocardium': [1384], 'vivo25': [1386], '14,': [1393, 1520], '13A': [1395], 'Dual': [1396, 1436], 'Role': [1397, 1437, 1655], 'EGFR': [1399, 1439], 'Tyrosine': [1401, 1441], 'Signaling': [1403, 1443], 'Ubiquitination': [1405, 1445], 'Capsids': [1408, 1448], 'Second-strand': [1411, 1451], 'DNA': [1412, 1452], 'SynthesisMolecular': [1413], '7Molecular': [1418], 'pathogenesis': [1419], 'alpha-1-antitrypsin': [1421], 'deficiency-associated': [1422], 'disease:': [1424], 'A': [1425, 1461, 1527], 'meeting': [1426], 'review1': [1427], 'Hepatology,': [1431], '45,': [1433], 'Synthesis17': [1453], 'TherapyPreclinical': [1458], '1-Pseudotyped': [1466], 'Demonstrates': [1468], 'Dose-Dependent': [1469], 'Injection': [1470, 1850, 1952], 'Site': [1471], 'Inflammation': [1472, 2337], 'And': [1473], 'Dissemination': [1474], 'Distant': [1479], 'Poirier,': [1488], 'Martha': [1489], 'Campbell-Thompson,': [1490], 'Byrne5': [1494], '18,': [1502], '3Gene': [1504], 'wound': [1507], 'healing:': [1508], 'present': [1509], 'directions24': [1513], '2006': [1515, 1548, 1576, 1605, 1622, 1647, 1738, 1764, 1799, 1832, 1881, 2031], '1Expanding': [1522], 'Capacity:': [1526], 'Tale': [1528], 'Two': [1530], 'VectorsBiotechnology': [1531], 'Genetic': [1533], 'Engineering': [1534], 'Reviews,': [1535], '1Role': [1539], 'Integrin': [1541, 1558], 'Cross-Regulation': [1542, 1559], 'Parvovirus': [1544, 1561], 'B19': [1545, 1562], 'Targeting15': [1546], '0,': [1554, 1805, 1838, 1966, 2100], '0Role': [1556], 'Targeting': [1563], 'Weigel-Kelley,': [1566, 1720], 'Linyuan': [1570], 'Chen,': [1571], 'Srivastava14': [1574], '9Long-term': [1584], 'repeated': [1587], 'administration': [1588], 'type': [1591, 1637, 2122], '1,': [1592], '5': [1595, 1699], 'skeletal': [1598, 1761], 'muscle': [1599], 'immunocompetent': [1601], 'adult': [1602], 'mice11': [1603], '13,': [1610, 1912, 1940], '17Treatment': [1612], 'disease': [1615, 2121], 'transfer13': [1620], 'Genetics,': [1625], '119,': [1627], '6Intrathecal': [1629], 'Suitable': [1639], 'Chronic': [1641, 2329], 'Pain': [1642], 'Studies': [1643], 'Rats30': [1645], 'Pain,': [1650], '2A': [1652, 2242, 2299, 2317], 'Antiapoptotic': [1654], 'Prevention': [1662], 'Pulmonary': [1664, 2331], 'EmphysemaAmerican': [1665], '173,': [1674], '11Evaluation': [1676], 'Primitive': [1678, 1776, 1809], 'Progenitor': [1683, 1781, 1814], 'Serotypes': [1696, 1794, 1827], 'Weiming': [1702], 'Yanjun': [1704], 'Jianqing': [1708], 'Wu,': [1709], 'Keyun': [1716], 'Qing,': [1717], 'Irene': [1721], 'Kenneth': [1723], 'Warrington,': [1725], 'Srivastava17': [1736], '3Viral': [1746], 'serotype': [1747, 2127, 2192], 'transgene': [1750], 'sequence': [1751], 'influence': [1752], 'overlapping': [1753], '(AAV)': [1756], 'vector-mediated': [1757], 'muscle1': [1762], 'The': [1766], '3Evaluation': [1774], '51': [1797], '0Evaluation': [1807], '524': [1830], '0Systemic': [1840], 'Fatty': [1844, 1946], 'Acid': [1845, 1947], 'Oxidation': [1846, 1948], 'Defect': [1847, 1949], 'Glenn': [1860], 'Walter,': [1861], 'Renius': [1862], 'Owen,': [1863], 'Travis': [1864], 'Cossette,': [1865], 'Erger,': [1867], 'Greg': [1868], 'Gutierrez,': [1869], 'Eric': [1870], 'Goetzman,': [1871], 'Dietrich': [1872], 'Matern,': [1873], 'Jerry': [1874], 'Vockley,': [1875], 'Flotte12': [1879], '1Gene': [1889], 'Late': [1893, 1984, 2080], 'Infantile': [1894, 1985, 2081], 'Form': [1895], 'Batten': [1897], 'DiseaseBiology': [1898], 'Liver-Directed': [1904], 'Nonhuman': [1908, 1995, 2091, 2265], 'PrimatesMolecular': [1909], '1Enhanced': [1914], 'mouse': [1917], 'bone': [1918], 'marrow-derived': [1919], 'dendritic': [1920], 'repetitive': [1923], 'infection': [1924], 'self-complementary': [1926], '6': [1929], 'combined': [1930], 'immunostimulatory': [1932], 'ligands1': [1933], '2005': [1935, 1960, 2056, 2094, 2131, 2174, 2226, 2245, 2291], '1Systemic': [1942], 'Vector14': [1958], '0Safety': [1968], 'Direct': [1970, 2066], 'Administration': [1971, 2067], 'AAV2CUhCLN2,': [1973, 2069], 'Candidate': [1975, 2071], 'Central': [1979, 2075], 'Nervous': [1980, 2076], 'System': [1981, 2077], 'Manifestations': [1982, 2078], 'Neuronal': [1986, 2082], 'Ceroid': [1987, 2083], 'Lipofuscinosis,': [1988, 2084], 'Brain': [1991, 2087], 'Rats': [1993, 2089], 'Primates': [1996, 2266], 'Neil': [1997], 'Hackett,': [1999], 'Eugene': [2001], 'Redmond,': [2002], 'Dolan': [2003], 'Sondhi,': [2004], 'Lela': [2006], 'Giannaris,': [2007], 'Elizabeth': [2008], 'Vassallo,': [2009], 'Jamie': [2010], 'Stratton,': [2011], 'Jianping': [2012], 'Qiu,': [2013], 'Stephen': [2014], 'Kaminsky,': [2016], 'Martin': [2017], 'L.': [2018, 2280, 2396], 'Lesser,': [2019], 'Fisch,': [2022], 'Serge': [2023], 'Rouselle,': [2025], 'Ronald': [2027], 'G.': [2028], 'Crystal3': [2029], '12Prevention': [2039], 'reversal': [2041], 'lupus': [2043], 'NZB/NZW': [2045], 'costimulatory': [2048], 'blockade': [2049], 'virus-mediated': [2052], 'transfer1': [2054], 'Arthritis': [2058], 'Rheumatism,': [2060], '52,': [2062], '12Safety': [2064], 'Primates1': [2092], '0Adeno-associated': [2102], 'Vector:': [2108], 'Applications25': [2114], '2005Sustained': [2116], 'correction': [2117], 'glycogen': [2119], 'storage': [2120], 'II': [2123], 'vectors26': [2129], '12,': [2136, 2161], '18Identification': [2138], 'factors': [2140], 'that': [2141], 'contribute': [2142], 'particle': [2146], 'aggregation': [2147], 'methods': [2149], 'prevent': [2151], 'its': [2152], 'occurrence': [2153], 'during': [2154], 'purification': [2156], 'formulationMolecular': [2158], '1Adeno-Associated': [2163], 'Lung': [2168], 'Diseases': [2169], 'Flotte16': [2172], '6Comparison': [2182], 'ability': [2185], 'pseudotyped': [2190], '2,': [2193, 2334], '5,': [2194], 'proteins': [2198], 'mediate': [2200], 'efficient': [2201], 'murine': [2207], 'nonhuman': [2209], 'primate': [2210], 'modelsMolecular': [2211], '11,': [2214], '6Adeno-Associated': [2216], 'Trials': [2221], 'Barrie': [2222], 'Carter25': [2224], '5Stable': [2234], 'therapeutic': [2238], 'levels': [2239], 'peptide17': [2243], 'Nature': [2247], 'Biotechnology,': [2248], '5Long-Term': [2252], 'Inducible': [2253], 'Eye': [2258], 'via': [2259], 'Corinna': [2267], 'Lebherz,': [2268], 'Alberto': [2269], 'Auricchio,': [2270], 'Albert': [2271], 'Maguire,': [2273], 'Victor': [2274], 'Rivera,': [2276], 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