Publication Information

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1219, 1280, 1391, 1645, 2058, 2210, 2382, 2449, 2578, 2756, 2848, 2918, 3210, 3349, 3562, 3702, 3818, 3921, 4132], 'The': [271, 796, 1108, 2059, 2757, 3003], 'severest': [272], 'PIDs': [273], 'present': [274, 2767], 'early': [275, 1021, 1399, 1456], 'life': [277], 'failure': [279], 'thrive': [281], 'infections': [284], '(often': [285], 'opportunistic),': [286], 'they': [288, 2252], 'usually': [290], 'fatal': [291], 'unless': [292, 2860], 'definitive': [293, 1300], 'implemented': [297], 'rapidly.2Fischer': [298], 'Notarangelo': [300, 1528, 2174, 2896], 'L.D.': [301, 1529, 1625, 1755, 2175, 2897], 'Neven': [302, 2898], 'B.': [303, 1040, 1241, 1623, 1794, 1846, 1989, 2525, 2653, 2899, 3264, 4026, 4096, 4109], 'Cavazzana': [304, 2900], 'M.': [305, 1042, 1317, 1323, 1497, 1614, 1700, 1798, 1810, 1854, 1941, 1943, 2409, 2527, 2534, 2661, 2722, 2901, 3034, 3088, 3324, 3434, 3476, 3488, 3525, 3623, 3791, 3875, 4028, 4035, 4063, 4100, 4162], 'Puck': [306, 2902], 'J.M.': [307, 2903], 'Severe': [308, 2904], 'related': [312, 945, 2908], 'disorders.Nat.': [313, 2909], 'Rev.': [314, 532, 2910], 'Dis.': [315, 2911], 'Primers.': [316, 2912], '1:': [318, 1601, 2914], '15061Crossref': [319, 2915], 'Fortunately,': [323], 'many': [324, 3987], 'immunological': [327, 401, 554, 1227, 2044, 2426], 'defects': [328, 1228, 2977], 'intrinsic': [330, 2285], 'system,': [334], 'making': [335], 'them': [336], 'tractable': [337], 'transplantation': [344, 420, 517, 1347], '(HSCT).': [345], 'Since': [346], 'transplants': [352], 'two': [354, 2352, 3005, 3456], 'PIDs,': [355], 'X-linked': [356, 2840, 2878, 3098, 3148, 3197, 3278, 3338, 3773], '(SCID-X1)': [361], 'Wiskott-Aldrich': [363, 426], 'syndrome': [364], '(WAS),': [365], 'HSCT': [366, 3243], 'methodologies': [367], 'technologies': [369], '(including': [370], 'conditioning': [371, 1679, 3827], 'regimens)': [372], 'improved,': [374], 'morbidity': [377], 'mortality': [379], 'this': [382, 617, 2218, 2760, 2850, 3820], 'procedure': [383, 1301], 'diminished': [385], 'considerably.3Gatti': [386], 'R.A.': [387, 395, 642, 698], 'Meuwissen': [388], 'H.J.': [389], 'Allen': [390], 'H.D.': [391], 'Hong': [392], 'R.': [393, 990, 1253, 1887, 1931, 2317, 3446], 'Good': [394], 'reconstitution': [397, 713, 861, 1758, 1814, 2091, 3895], 'sex-linked': [399], 'lymphopenic': [400], 'deficiency.Lancet.': [402], '1968;': [403, 428], '2:': [404, 429], '1366-1369Abstract': [405], 'Scholar,': [408, 433, 508, 661, 690, 731, 760, 1173, 1363, 1525, 1564, 1605, 1735, 1790, 1836, 1877, 1923, 1969, 2008, 2343, 2362, 2556, 2702, 3072, 3121, 3162, 3304, 3470, 3515, 3660, 3781, 4057, 4092, 4199], '4Bach': [409], 'F.H.': [410, 2319], 'Albertini': [411], 'R.J.': [412, 1177], 'Joo': [413], 'P.': [414, 441, 445, 514, 1313, 1505, 1537, 1545, 1977, 2183, 2191, 2313, 3046, 3314, 3440], 'Anderson': [415], 'Bortin': [417], 'M.M.': [418], 'Bone-marrow': [419], 'patient': [423, 3336, 3932], 'syndrome.Lancet.': [427], '1364-1366Abstract': [430], '5Gennery': [434], 'A.R.': [435], 'Slatter': [436, 1322], 'M.A.': [437], 'Grandin': [438], 'L.': [439, 1501, 1800, 1848, 1883, 1929, 2026, 2309, 2345, 2401, 2413, 2704, 3181, 3676, 3869, 3873, 4215], 'Taupin': [440], 'Cant': [442], 'A.J.': [443, 3883], 'Veys': [444, 513, 1312], 'Amrolia': [446], 'P.J.': [447, 3793], 'Gaspar': [448, 1154, 1366, 1622, 2475, 2647], 'H.B.': [449, 1155, 1367, 1737, 2010, 3074, 3164], 'Davies': [450, 2027, 3182], 'E.G.': [451, 2028, 3183], 'Friedrich': [452, 1318, 3315], 'al.Inborn': [455, 1325], 'Errors': [456, 1326], 'Working': [457, 1327], 'Party': [458, 1328], 'European': [461, 468, 1331, 1339, 2158], 'Group': [462, 1332], 'Blood': [464, 1334], 'Marrow': [466, 1336], 'Transplantation;': [467], 'Society': [469, 1340], 'ImmunodeficiencyTransplantation': [471], 'cells': [475, 610, 679, 709, 749, 851, 885, 1444, 1592, 1996, 2333, 2593, 2687, 2781, 3389, 3995, 4119], 'long-term': [477, 712, 2043, 3395], 'survival': [478, 858, 1439, 2062, 2077, 3222, 3369], 'Europe:': [483], 'entering': [484], 'century,': [487], 'do': [488, 490], 'better?.J.': [491], 'Allergy': [492], '2010;': [495, 3155], '126': [496], '(e1–e11):': [497], '602-610Abstract': [498], 'Full': [499, 501, 724, 904, 906, 1066, 1068, 1781, 1783, 2440, 2442, 3112, 3114, 3912, 3914], 'Text': [500, 502, 725, 905, 907, 1067, 1069, 1782, 1784, 2441, 2443, 3113, 3115, 3913, 3915], 'PDF': [503, 726, 908, 1070, 1785, 2444, 3116, 3916], '(183)': [506], '6Booth': [509], 'Silva': [511], 'J.': [512, 992, 998, 1000, 1245, 1249, 1747, 1868, 2024, 2523, 2532, 2818, 3084, 3125, 3153, 3179, 3295, 3668, 3751, 4024, 4033, 4061, 4102, 4207], 'Stem': [515, 899], 'treatment': [520, 1914], 'children:': [524], 'current': [525, 2243], 'status': [526], 'hopes': [528], 'future.Expert': [531], '2016;': [535, 1166, 1962, 2355, 2375], '12:': [536], '713-723Crossref': [537], '(0)': [540, 793, 1217, 1361, 1733, 1967, 2360, 2380, 3302], 'Even': [543, 1281], 'so,': [544, 1282], 'without': [547, 1815, 3951], 'HLA-matched': [548, 3242], 'donors,': [549], 'graft-versus-host': [550], 'disease': [551], '(GvHD),': [552], 'delayed': [553], 'reconstitution,': [555], 'graft': [557], 'rejection': [558], 'remain': [559], 'significant': [560, 3726], 'problems.': [561], 'Some': [562], 'conditions': [563, 845, 954], 'better': [565], 'candidates': [566], 'others': [568], 'due': [569, 1862, 1957, 3282], 'disease-specific': [571, 2761], 'characteristics,': [572], 'risks': [574, 825], 'heightened': [576], 'who': [579, 584, 3413], 'actively': [581], 'infected': [582], 'or': [583, 1052, 2084, 2793, 2872, 2986], 'developed': [586, 3421], 'chronic': [587], 'complications': [588], 'at': [589, 873, 964, 1287, 2109, 2275, 2766], 'time': [591, 967], 'treatment.': [593], '30': [596], 'years': [597, 2385], 'ago,': [598], 'demonstration': [600, 2420], 'retroviral': [602, 1022, 1425, 1911, 1952, 2286, 2546, 2565, 2588, 2676, 2728, 3603, 4047, 4079, 4122], 'transfer': [604, 646, 782, 960, 1632, 2250], '(HSC/Ps)': [611], 'led': [612], 'suggestion': [615], 'technology': [618, 1024, 2230], 'could': [619, 2771, 3839], 'provide': [620, 1471], 'alternative': [622], 'platform': [623], 'therapies': [627], 'number': [630, 916, 2512, 2985, 3016], 'diseases': [632], 'were': [634, 802, 1395, 2461, 3021, 3414, 3924], 'amenable': [635], 'HSCT.7Joyner': [637], 'Keller': [639], 'G.': [640, 733, 1048, 1499, 1533, 1535, 1580, 1621, 1710, 1796, 1852, 2179, 2181, 2657, 3040, 3086, 3268, 3276], 'Phillips': [641, 697], 'Bernstein': [643, 699], 'Retrovirus': [645], 'bacterial': [649], 'into': [651, 675, 706, 783], 'mouse': [652], 'haematopoietic': [653, 677, 753], 'cells.Nature.': [655], '1983;': [656], '305:': [657], '556-558Crossref': [658], '8Williams': [662], 'D.A.': [663, 889, 983, 1050, 2399], 'Lemischka': [664], 'I.R.': [665], 'Nathan': [666], 'D.G.': [667], 'Mulligan': [668, 2316], 'R.C.': [669], 'Introduction': [670, 701], 'genetic': [673, 897, 987, 3289, 3587], 'material': [674], 'pluripotent': [676], 'mouse.Nature.': [682], '1984;': [683], '310:': [684], '476-480Crossref': [685], '(263)': [688], '9Dick': [691], 'J.E.': [692, 772], 'Magli': [693], 'M.C.': [694], 'Huszar': [695], 'D.': [696, 1243, 1541, 1745, 1885, 1979, 1981, 2187, 2315, 2323, 2347, 2708, 3080, 3536, 3634, 4173], 'selectable': [704], 'primitive': [707], 'capable': [710, 3981], 'hemopoietic': [716], 'system': [717], 'W/Wv': [719], 'mice.Cell.': [720], '1985;': [721, 755, 788], '42:': [722], '71-79Abstract': [723], '(315)': [729], '10Keller': [732], 'Paige': [734], 'Gilboa': [736], 'E.': [737, 1187, 1539, 1739, 1893, 1973, 2022, 2185, 2655, 2665, 3044, 3310, 3448, 3672, 3674, 3678, 3785, 4211, 4213, 4217], 'Wagner': [738], 'E.F.': [739], 'Expression': [740], 'foreign': [743], 'myeloid': [746, 2113, 2858], 'lymphoid': [748, 2836, 3769], 'derived': [750], 'multipotent': [752], 'precursors.Nature.': [754], '318:': [756], '149-154Crossref': [757], '11Gruber': [761], 'H.E.': [762], 'Finley': [763], 'K.D.': [764], 'Hershberg': [765], 'R.M.': [766, 1487], 'Katzman': [767], 'S.S.': [768, 2822, 3755], 'Laikind': [769], 'P.K.': [770], 'Seegmiller': [771], 'Friedmann': [773], 'Yee': [775], 'J.K.': [776], 'Jolly': [777], 'D.J.': [778, 3490], 'Retroviral': [779, 2367], 'vector-mediated': [780, 2423], 'human': [784, 951, 3059], 'cells.Science.': [787], '230:': [789], '1057-1061Crossref': [790], 'advantages': [797, 2257], 'autologous': [799], 'anticipated': [803, 877, 1433, 2605], 'lie': [805], 'lack': [808, 2283, 2856], 'need': [810, 1448, 3952], 'identify': [812], 'suitable': [814], 'donor,': [815], 'obviation': [816], 'GvHD,': [818], 'potential': [821], 'reduce': [823, 3830], 'myelosuppressive': [827], 'immunosuppressive': [829], 'preconditioning': [830, 1450], 'patient,': [833], 'which': [834, 973, 1416, 1481, 2115, 2164, 2959, 3591], 'is': [835, 1079, 1089, 1140, 2116, 2165, 2498, 2735, 2765, 2882, 2939, 3496, 3724, 3738, 3821], 'required': [836, 3608], 'make': [838], 'space': [839], 'engrafting': [841], 'HSC/Ps.': [842], 'For': [843], 'it': [846, 2770, 2938, 3861], 'was': [847, 876, 974, 1421, 1431, 2072, 2131, 3402, 3574, 3606, 4135], 'expected': [848], 'corrected': [850, 1668, 3225], 'would': [852, 931, 1026, 1445, 3228, 3828], 'profound': [855, 1138, 2976], 'growth': [856, 1437], 'advantage,': [859], 'allowing': [860], 'relatively': [863, 3927], 'low': [864, 2984], 'numbers': [865], 'cells.': [867], 'In': [868, 2324, 3379, 3563], 'addition,': [869], 'expression': [870, 1420, 2740, 2927, 3848, 4015], 'transgenes': [872], 'supraphysiological': [874], 'levels': [875, 2111], 'allow': [879, 3229], '"cross-correction"': [880], 'deficient': [883], 'non-hematopoietic': [884, 2784], 'diseases.12Williams': [888], 'Broadening': [890], 'indications': [892, 2451, 2631], 'therapies.Cell': [898], 'Cell.': [900], '2013;': [901, 2571], '13:': [902], '263-264Abstract': [903], '(7)': [911], 'Over': [914], 'years,': [918], 'correction': [920, 2424, 3493], 'cellular': [922], 'animal': [924], 'models': [925, 2431], 'boosted': [926], 'expectation': [927, 3217], 'rapidly': [932], 'become': [933], 'mainstream,': [934], 'but': [935, 1232, 1685, 2769, 2799, 3901, 3930], 'translation': [937], 'proved': [938, 1290], 'more': [939, 1160, 2087, 2265, 2453, 3585], 'difficult': [940], '(Figure': [941], '1).': [942], 'This': [943, 2497, 3957, 4133], 'partly': [944], 'limited': [948], 'understanding': [949], 'HSC/P': [952, 2121, 2248], 'culture': [953], 'necessary': [955], 'achieve': [957], 'high-level': [958, 2098], 'ex': [961, 2220, 2875], 'vivo,': [962], 'yet': [963], 'same': [966], 'retaining': [968], 'vivo': [970, 2221, 2325, 2876], 'engraftment': [971, 1693, 3385, 3737], 'capability,': [972], 'not': [975, 2853, 3392], 'well': [976, 3416], 'modeled': [977], 'murine': [979, 1409, 2839, 3772], 'preclinical': [980, 2387], 'assays.13Williams': [982], 'Principles': [984, 1003], 'cell-based': [986], 'therapies.in:': [988], 'Hoffman': [989], 'Benz': [991], 'Silberstein': [993], 'L.E.': [994], 'Heslop': [995], 'H.': [996, 3270, 3482, 3527, 3625, 4164], 'Weitz': [997], 'Anastasi': [999], 'Hematology:': [1001], 'Basic': [1002], 'Practice.': [1005], 'Elsevier,': [1006], '2013:': [1007], '1503-1512Google': [1008], 'Furthermore,': [1010], 'occurrence': [1012], 'clinically': [1014, 2483, 3415, 3833], 'manifesting': [1015, 2484, 3834], 'insertional': [1016, 4083], 'mutagenesis': [1017, 1055, 2507, 3539, 3637, 4176], 'highlighted': [1018, 2519], 'deficiencies': [1019], 'vector': [1023, 2169, 2229, 2244, 2368, 2729, 3500, 3604, 4080], 'resolved.14Baum': [1030], 'von': [1032, 2528, 2718, 3794, 4029, 4064, 4105], 'Kalle': [1033, 2529, 2719, 3090, 3431, 3795, 4030, 4065], 'Staal': [1035], 'F.J.': [1036], 'Li': [1037, 4110], 'Z.': [1038, 1311, 2521, 4022, 4111], 'Fehse': [1039, 4095], 'Schmidt': [1041, 2526, 2721, 3087, 3433, 3475, 3483, 3790, 4027, 4062], 'Weerkamp': [1043], 'F.': [1044, 1179, 1193, 1617, 1702, 1714, 1749, 1804, 1840, 1879, 1927, 1933, 1937, 2018, 2562, 3042, 3048, 3137, 3260], 'Karlsson': [1045], 'S.': [1046, 1698, 1704, 1708, 1753, 1808, 1842, 1850, 1935, 1975, 1983, 1987, 2012, 2020, 2407, 2663, 3036, 3052, 3078, 3123, 3139, 3166, 3172, 3429, 3484, 3662, 3879, 4068, 4201], 'Wagemaker': [1047], 'Williams': [1049], 'Chance': [1051], 'necessity?': [1053], 'Insertional': [1054], 'its': [1060], 'consequences.Mol.': [1061], 'Ther.': [1062, 1640, 1777, 2436, 2570, 2844, 3777, 3908], '2004;': [1063, 3109], '9:': [1064], '5-13Abstract': [1065], '(174)': [1073], 'Adenosine': [1076, 1093, 1156], 'deaminase': [1077, 1094, 1157, 1266, 1597, 1638, 1865, 1960, 2434], '(ADA)': [1078], 'housekeeping': [1081], 'enzyme': [1082, 1261], 'purine': [1085, 1121], 'metabolic': [1086, 1109, 2047, 2428], 'pathway': [1087], 'widely': [1090], 'expressed.15Hershfield': [1091], 'M.S.': [1092, 1247], 'deficiency:': [1095, 1907], 'expression,': [1097], 'molecular': [1098, 4152], 'basis,': [1099], 'therapy.Semin.': [1101], 'Hematol.': [1102], '1998;': [1103], '291-298PubMed': [1105], 'environment': [1110], 'resulting': [1111, 2981], 'deficiency': [1113, 1158, 1376, 2789], 'ADA': [1115, 1222, 1417, 2788], 'accumulation': [1118, 3583], 'toxic': [1120], 'metabolites': [1122], 'causes': [1123, 3545, 3643, 4182], 'skeletal,': [1125], 'lung,': [1126], 'liver,': [1127], 'gastrointestinal,': [1128], 'neurodevelopmental,': [1129], 'sensorineural': [1131], 'defects,': [1132], 'whereas': [1133], 'consistent': [1136], 'abnormality': [1139], 'lymphocytes,': [1145, 1462, 2994], 'T,': [1147], 'B,': [1148], 'natural': [1150, 2991], 'killer': [1151, 2992], 'cells.16Whitmore': [1152], 'K.V.': [1153], '-': [1159], 'just': [1162], 'immunodeficiency.Front.': [1164], '7:': [1167, 1213], '314Crossref': [1168], '(4)': [1171], '17Sauer': [1174], 'A.V.': [1175], 'Hernandez': [1176], 'Fumagalli': [1178], 'Bianchi': [1180], 'V.': [1181, 2669, 3255, 3257, 3789], 'Poliani': [1182], 'P.L.': [1183], 'Dallatomasina': [1184], 'Riboni': [1186], 'Politi': [1188], 'L.S.': [1189], 'Tabucchi': [1190, 1711], 'Carlucci': [1192, 1713], 'al.Alterations': [1195], 'brain': [1198], 'adenosine': [1199, 1265, 1349, 1371, 1596, 1637, 1864, 1902, 1959, 1998, 2036, 2433], 'metabolism': [1200], 'cause': [1201], 'behavioral': [1202], 'neurological': [1204], 'impairment': [1205], 'ADA-deficient': [1207], 'mice': [1208, 3992], 'patients.Sci.': [1210], 'Rep.': [1211], '2017;': [1212, 1381, 2003, 2845, 3778], '40136Crossref': [1214], 'Exogenous': [1220], 'polyethylene-glycol-conjugated': [1221], '(PEG-ADA)': [1223], 'rescue': [1225], 'through': [1229, 3854], 'extracellular': [1230], 'detoxification,': [1231], 'effects': [1234], 'often': [1236], 'partial': [1237], 'poorly': [1239], 'sustained.18Chan': [1240], 'Wara': [1242], 'Bastian': [1244], 'Hershfield': [1246], 'Bohnsack': [1248], 'Azen': [1250], 'C.G.': [1251], 'Parkman': [1252], 'Weinberg': [1254, 1567], 'K.': [1255, 1315, 1741, 3312, 3320, 3322, 3472, 3521, 3531, 3619, 3629, 3680, 3887, 4104, 4158, 4168], 'Kohn': [1256], 'D.B.': [1257, 1365, 1566, 2477, 2649], 'Long-term': [1258], 'efficacy': [1259, 1950, 1992, 2469], 'replacement': [1262], '(ADA)-deficient': [1267], '(SCID).Clin.': [1271], '2005;': [1273, 4125], '117:': [1274, 1830, 3509], '133-143Crossref': [1275], '(91)': [1278], 'use': [1284, 1674, 1773, 3103, 3825], 'PEG-ADA': [1286, 1661, 1771, 2083], 'diagnosis': [1288], 'has': [1289, 2092, 2231, 2293, 3363, 3704], 'very': [1292, 3030, 3403, 3960], 'useful': [1293], 'way': [1294, 2396], 'stabilize': [1296], 'before': [1298], 'implemented.19Hassan': [1304], 'Booth': [1306], 'Brightwell': [1308], 'Allwood': [1310], 'Rao': [1314], 'Hönig': [1316], 'Gennery': [1320], 'Transplantation': [1337], 'ImmunodeficiencyOutcome': [1342], 'deaminase-deficient': [1350, 1372, 1903, 2037], 'immunodeficiency.Blood.': [1353, 3341], '2012;': [1354, 1916, 3811], '120': [1355], '(quiz': [1356], '3626):': [1357], '3615-3624Crossref': [1358], '20Kohn': [1364], 'How': [1368], 'manage': [1370], 'immune': [1375, 1906], '(ADA': [1377], 'SCID).J.': [1378], '(Published': [1382], 'online': [1383], 'February': [1384], '14,': [1385], '2017)https://doi.org/10.1007/s10875-017-0373-yCrossref': [1386], '(2)': [1389], 'Several': [1392, 2579], 'trials': [1394, 3007], 'initiated': [1396], '1990s': [1400], 'using': [1401, 1458, 1649, 2391, 3023], 'gammaretroviral': [1402, 1651, 2600, 3027, 3107, 3999], 'vectors': [1403, 1912, 2393, 2501, 2677, 3028, 4000], 'had': [1405, 3418], 'evaluated': [1407], 'primate': [1411], 'model': [1412, 3892], 'systems,': [1413], 'cDNA': [1418], 'transgene': [1419, 3851], 'regulated': [1422], 'by': [1423, 1719, 1763, 2334, 2463, 2545, 2943, 3102, 4046, 4121], 'long': [1426, 4143], 'terminal': [1427], 'repeat': [1428], '(LTR).': [1429], 'It': [1430, 3703], 'also': [1432, 2294, 2630, 4136], 'selective': [1436, 1665], 'advantage': [1440, 1666, 3223], 'imparted': [1441], 'gene-corrected': [1443, 3964], 'obviate': [1446], 'patient.': [1453], 'However,': [1454, 2849], 'studies': [1457, 1647, 2071, 2390, 2494, 3990], 'residual': [1459], 'peripheral': [1460, 1550, 2196], 'blood': [1461, 1551, 1591, 2197], 'umbilical': [1463, 1589], 'cord': [1464, 1590], 'blood,': [1465], 'failed': [1469], 'clear': [1472], 'benefit': [1474], 'over': [1475, 4141], 'above': [1477], 'PEG-ADA,': [1480], 'continued': [1484], 'receive.21Blaese': [1486], 'Culver': [1488], 'K.W.': [1489], 'Miller': [1490], 'A.D.': [1491], 'Carter': [1492], 'C.S.': [1493, 3444], 'Fleisher': [1494], 'Clerici': [1496], 'Shearer': [1498], 'Chang': [1500], 'Chiang': [1502], 'Y.': [1503], 'Tolstoshev': [1504], 'al.T': [1507], 'lymphocyte-directed': [1508], 'ADA-': [1512], 'SCID:': [1513], 'initial': [1514], 'trial': [1515], 'results': [1516], 'after': [1517, 3193, 3408, 3458, 3687, 3947, 3973], '4': [1518, 3685], 'years.Science.': [1519], '1995;': [1520, 1559, 1600, 2205], '270:': [1521, 1560, 2206], '475-480Crossref': [1522], '22Bordignon': [1526], 'Nobili': [1530, 2176], 'N.': [1531, 2177, 3438, 4107], 'Ferrari': [1532, 2178], 'Casorati': [1534, 2180], 'Panina': [1536, 2182], 'Mazzolari': [1538, 2184], 'Maggioni': [1540, 2186], 'Rossi': [1542, 2188], 'Servida': [1544, 2190], 'al.Gene': [1547, 1858, 1899, 2193, 3056, 3095], 'lymphocytes': [1552, 2102, 2198], 'ADA-immunodeficient': [1557, 2203], 'patients.Science.': [1558, 2204], '470-475Crossref': [1561, 2207], '23Kohn': [1565], 'K.I.': [1568], 'Nolta': [1569], 'J.A.': [1570], 'Heiss': [1571], 'L.N.': [1572], 'Lenarsky': [1573], 'Crooks': [1575], 'G.M.': [1576], 'Hanley': [1577], 'Annett': [1579], 'Brooks': [1581], 'J.S.': [1582], 'el-Khoureiy': [1583], 'al.Engraftment': [1586], 'gene-modified': [1588, 1994], 'neonates': [1594], 'deficiency.Nat.': [1598], 'Med.': [1599, 1869, 2050, 2748, 3154, 3202, 3296, 3810], '1017-1023Crossref': [1602], '24Hoogerbrugge': [1606], 'P.M.': [1607], 'van': [1608], 'Beusechem': [1609], 'V.W.': [1610], 'Fischer': [1611, 3271], 'Debree': [1613], 'le': [1615], 'Deist': [1616, 3259], 'Perignon': [1618], 'Morgan': [1620], 'Fairbanks': [1624, 1754, 2412], 'Skeoch': [1626], 'C.H.': [1627], 'al.Bone': [1629], 'three': [1634], 'deficiency.Gene': [1639], '1996;': [1641, 2338, 3297], '3:': [1642, 2052, 2750, 3204], '179-183PubMed': [1643], 'Subsequent': [1646], '(again': [1648], 'LTR-intact': [1650, 2599, 3026], 'vectors)': [1652], 'introduced': [1653], 'changes,': [1656], 'withdrawal': [1659, 2127], 'enhance': [1663], 'cells,': [1669, 2114, 3976], 'importantly': [1672], 'low-': [1676], 'and/or': [1677], 'reduced-intensity': [1678], 'alkylating': [1681], 'agents': [1682], '(usually': [1683], 'busulphan,': [1684], 'few': [1688], 'melphalan)': [1690], 'promote': [1692], 'HSC/Ps.25Aiuti': [1695], 'Slavin': [1697], 'Aker': [1699, 1809], 'Ficara': [1701], 'Deola': [1703], 'Mortellaro': [1705], 'Morecki': [1707], 'Andolfi': [1709, 1795, 1851], 'al.Correction': [1716], 'ADA-SCID': [1718, 1762, 1819, 2595, 2689], 'nonmyeloablative': [1726], 'conditioning.Science.': [1727], '2002;': [1728, 2549, 4050], '296:': [1729, 2550, 4051], '2410-2413Crossref': [1730], '26Gaspar': [1736], 'Bjorkegren': [1738, 2021], 'Parsley': [1740, 2015, 3075, 3169], 'Gilmour': [1742, 2013, 3081, 3167, 3319], 'K.C.': [1743, 2014, 3082, 3168], 'King': [1744, 3079, 3489], 'Sinclair': [1746, 3083], 'Zhang': [1748, 2017, 2400], 'Giannakopoulos': [1750], 'Adams': [1752, 2019, 3171], 'al.Successful': [1757], 'immunity': [1760, 3946], 'following': [1768, 3241, 3547, 3645, 4184], 'cessation': [1769], 'mild': [1775], 'preconditioning.Mol.': [1776], '2006;': [1778, 4085], '14:': [1779], '505-513Abstract': [1780], '(158)': [1788], '27Aiuti': [1791], 'Cassani': [1793, 1845], 'Mirolo': [1797, 1853, 2660], 'Biasco': [1799], 'Recchia': [1801], 'Urbinati': [1803], 'Valacca': [1805], 'Scaramuzza': [1807, 1849], 'al.Multilineage': [1812], 'clonal': [1816, 2635, 3451], 'selection': [1817], 'treated': [1821, 2068, 2456, 2597, 2691, 2733, 3925], 'therapy.J.': [1826], 'Invest.': [1828, 2002, 3507, 3554, 3652, 3694, 4191], '2007;': [1829, 3508], '2233-2240Crossref': [1831], '(180)': [1834], '28Aiuti': [1837], 'Cattaneo': [1839], 'Galimberti': [1841], 'Benninghoff': [1843], 'U.': [1844, 2311, 3262, 3308, 4059, 4098], 'Callegaro': [1847], 'Brigida': [1855, 1938, 2711], 'I.': [1856, 1939, 2667, 2712, 3266], 'deficiency.N.': [1866], 'Engl.': [1867, 3152, 3294], '2009;': [1870, 2695, 3909], '360:': [1871], '447-458Crossref': [1872], '(585)': [1875], '29Candotti': [1878], 'Shaw': [1880], 'K.L.': [1881, 2016, 3076, 3170], 'Muul': [1882], 'Carbonaro': [1884, 1980], 'Sokolic': [1886], 'Choi': [1888], 'Schurman': [1890], 'S.H.': [1891], 'Garabedian': [1892, 1972], 'Kesserwan': [1894], 'Jagadeesh': [1896], 'G.J.': [1897], 'comparison': [1909], 'plans.Blood.': [1915], '120:': [1917], '3635-3646Crossref': [1918], '(114)': [1921], '30Cicalese': [1924], 'M.P.': [1925, 2826, 3436, 3759], 'Ferrua': [1926], 'Castagnaro': [1928], 'Pajno': [1930], 'Barzaghi': [1932], 'Giannelli': [1934], 'Dionisio': [1936], 'Bonopane': [1940], 'Casiraghi': [1942], 'al.Update': [1945], 'safety': [1948], 'deficiency.Blood.': [1961], '128:': [1963], '45-54Crossref': [1964], '31Shaw': [1970], 'K.K.': [1971], 'Mishra': [1974], 'Barman': [1976], 'Davila': [1978], 'Shupien': [1982], 'Silvin': [1984], 'Geiger': [1986, 2406], 'Nowicki': [1988], 'al.Clinical': [1991, 3326], 'deaminase-deficiency': [1999], 'immunodeficiency.J.': [2000], 'https://doi.org/10.1172/JCI90367Crossref': [2004], '(1)': [2006], '32Gaspar': [2009], 'Cooray': [2011, 3165], 'Bayford': [2023, 3178], 'Brown': [2025, 3180], 'al.Hematopoietic': [2030], 'leads': [2041], 'recovery': [2045, 3351], 'correction.Sci.': [2048], 'Transl.': [2049, 3201], '2011;': [2051, 2749, 3203], '97ra80Crossref': [2053], 'overall': [2061, 3376], 'rate': [2063, 2078, 3370], 'around': [2065], '50': [2066], '100%,': [2073], 'disease-free': [2076, 3368], '(not': [2079], 'requiring': [2080], 're-introduction': [2081], 'HSCT)': [2085], '70%.': [2089], 'general': [2094], 'robust,': [2096], 'marking': [2100, 2122, 3401], 'and,': [2103], 'time,': [2107], 'albeit': [2108], 'lower': [2110, 2775], 'effective': [2118], 'surrogate': [2119], 'marrow.': [2125], 'Permanent': [2126], 'immunoglobulin': [2129], 'supplementation': [2130], 'achieved': [2132], 'approximately': [2134], '50%': [2135], 'patients,': [2137, 2457, 3567], 'suggesting': [2138], 'further': [2140, 3955], 'still': [2143, 2851], 'possible.': [2144], 'Recently,': [2145], 'collaboration': [2147], 'among': [2148], 'GlaxoSmithKline': [2149], '(GSK),': [2150], 'Fondazione': [2151], 'Telethon,': [2152], 'Ospedale': [2154], 'San': [2155], 'Raffaele': [2156], 'secured': [2157], 'Marketing': [2159], 'Authorisation': [2160], 'Strimvelis': [2162], 'TM,': [2163], 'original': [2167], 'LTR-based': [2168], 'studied': [2170], 'Milan.22Bordignon': [2172], 'From': [2211], 'proof': [2212], 'concept': [2214], 'humans': [2216], 'market': [2222], 'authorization': [2223], 'product,': [2228], 'advanced': [2232], 'considerably.': [2233], 'HIV-1-based': [2234], 'lentiviruses': [2235], 'particular': [2237], 'adopted': [2240], 'as': [2241, 2516, 4008], 'choice': [2246], 'because': [2251, 2500, 2937, 3735], 'appear': [2253], 'offer': [2255], 'terms': [2259], 'mutagenicity': [2261], '(because': [2262], 'favorable': [2266], 'genome': [2267], 'insertion': [2268, 3605], 'profile': [2269, 2726], 'within': [2270], 'transcriptional': [2271, 2683], 'units': [2272], 'rather': [2273], 'transcription': [2276], 'start': [2277], 'sites': [2278, 2590, 2616], '[TSSs],': [2279], 'theoretical': [2281], 'advantage),': [2282], 'LTR': [2287, 4007], 'enhancer': [2288, 4003], 'activity': [2289, 2684, 4140], '(a': [2290], 'modification': [2291], 'duplicated': [2296, 4002], 'generation': [2300], 'gammaretroviruses),': [2302], 'higher': [2304], 'efficiency': [2305], 'transfer.33Naldini': [2308], 'Blömer': [2310], 'Gallay': [2312], 'Ory': [2314], 'Gage': [2318], 'Verma': [2320, 2348], 'I.M.': [2321, 2349], 'Trono': [2322, 2346], 'delivery': [2327], 'stable': [2329], 'transduction': [2330, 3032], 'nondividing': [2332], 'lentiviral': [2336, 2392, 2422], 'vector.Science.': [2337], '272:': [2339], '263-267Crossref': [2340], '34Naldini': [2344], 'Lentiviral': [2350], 'vectors,': [2351, 2601], 'decades': [2353], 'later.Science.': [2354], '353:': [2356], '1101-1102Crossref': [2357], '35Everson': [2363], 'E.M.': [2364], 'Trobridge': [2365], 'G.D.': [2366], 'interactions': [2369], 'cells.Curr.': [2372], 'Opin.': [2373], 'Virol.': [2374], '21:': [2376], '41-46Crossref': [2377], 'Following': [2383], 'several': [2384], 'development,': [2388], 'under': [2395], 'ADA-SCID.36Carbonaro': [2398], 'Jin': [2402], 'X.': [2403], 'Montiel-Equihua': [2404], 'Carmo': [2408], 'Cooper': [2410], 'Kaufman': [2414], 'M.L.': [2415], 'Sebire': [2416], 'N.J.': [2417], 'al.Preclinical': [2419], 'abnormalities': [2429], 'deficiency.Mol.': [2435], '2014;': [2437], '22:': [2438], '607-622Abstract': [2439], '(27)': [2447, 3347], 'Early': [2450], '32': [2455], 'whom': [2460], 'excellent': [2468, 3375], 'no': [2471, 2482, 3902], 'toxicity': [2473, 2764, 2859], '(H.B.': [2474, 2646], 'Kohn,': [2478, 2650], 'personal': [2479, 2651], 'communication).': [2480], 'Interestingly,': [2481], 'vector-related': [2485], 'toxicities': [2486], 'observed': [2489, 3240], 'any': [2491, 3954], 'date.': [2496], 'surprising': [2499], 'similar': [2503, 2862, 3031], 'configurations': [2504], 'caused': [2506], 'malignancy': [2509, 2837, 3770], 'conditions,': [2515], 'will': [2517], 'later.37Li': [2520], 'Düllmann': [2522, 4023, 4101], 'Schiedlmeier': [2524, 4025], 'Meyer': [2531, 4032], 'Forster': [2533, 4034], 'Stocking': [2535, 4036], 'Wahlers': [2537, 4038], 'Frank': [2539, 4040], 'O.': [2540, 4041, 4094], 'al.Murine': [2542, 4043], 'leukemia': [2543, 3427, 4044], 'induced': [2544, 4045], 'marking.Science.': [2548, 4049, 4124], '497Crossref': [2551, 4052], '(514)': [2554, 4055], '38Cavazza': [2557], 'Moiani': [2559], 'Mavilio': [2561], 'Mechanisms': [2563], 'integration': [2566, 2589, 2608, 3501], 'mutagenesis.Hum.': [2568], 'Gene': [2569], '24:': [2572], '119-131Crossref': [2573], '(45)': [2576, 3816], 'reports': [2580], 'detailed': [2583], 'comprehensive': [2585], 'analysis': [2586], '(RISs)': [2591], 'although': [2603, 2637, 3581], 'patterns': [2606], 'observed,': [2610], 'namely': [2611], 'preference': [2612], 'TSSs,': [2614], 'near': [2617], 'proto-oncogenes': [2619], 'Lim': [2620, 3126, 3441, 3669, 4208], 'domain': [2621], 'only': [2622], '2': [2623], '(LMO2),': [2624], 'BCL2,': [2625], 'CCND2,': [2627], 'there': [2628], 'subtle': [2633], 'mutagenic': [2634], 'disturbances,': [2636], 'apparently': [2638], 'insufficient': [2639], 'drive': [2641], 'frank': [2643], 'malignant': [2644], 'program': [2645, 3734], 'communication).39Cassani': [2652], 'Montini': [2654], 'Maruggi': [2656], 'Ambrosi': [2658, 2705], 'Selleri': [2662], 'Biral': [2664], 'Frugnoli': [2666], 'Hernandez-Trujillo': [2668], 'Di': [2670, 2715], 'Serio': [2671, 2716], 'al.Integration': [2674], 'induces': [2678], 'minor': [2679], 'changes': [2680, 3588], 'T': [2686, 2780, 2989, 3190, 3236, 3292, 3355, 3423, 3452, 3716, 3800, 3864, 3944, 3965], 'therapy.Blood.': [2694], '114:': [2696], '3546-3556Crossref': [2697], '(48)': [2700], '40Biasco': [2703], 'Pellin': [2707], 'Bartholomae': [2709, 3522, 3620, 4159], 'Roncarolo': [2713], 'M.G.': [2714], 'Aiuti': [2723], 'Integration': [2725], 'cell-specific': [2736], 'according': [2737], 'chromatin': [2742], 'conformation': [2743], 'target': [2745], 'cell.EMBO': [2746], 'Mol.': [2747], '89-101Crossref': [2751], '(59)': [2754], 'reason': [2758], 'difference': [2762], 'unclear,': [2768], 'relate': [2772], 'replicative': [2776, 3710], 'stress': [2777, 3711], 'reconstituting': [2779], 'environmental': [2785], 'background': [2786], 'thymus,': [2792], 'presence': [2795], 'competing': [2797], 'non-transduced': [2798], 'detoxified': [2800], 'thymocytes': [2801], 'successfully': [2803, 3419], 'complete': [2804], 'their': [2805, 3943], 'maturation': [2806, 3866], 'program.41Ginn': [2807], 'S.L.': [2808, 3741], 'Hallwirth': [2809, 3742], 'C.V.': [2810, 3743], 'Liao': [2811, 3744], 'S.H.Y.': [2812, 3745], 'Teber': [2813, 3746], 'E.T.': [2814, 3747], 'Arthur': [2815, 3748], 'J.W.': [2816, 3749], 'Wu': [2817, 3750], 'Lee': [2819, 3752], 'H.C.': [2820, 3753], 'Tay': [2821, 3754], 'Reddell': [2823, 3756], 'R.R.': [2824, 3757], 'McCormack': [2825, 3435, 3758], 'al.Limiting': [2828, 3761], 'thymic': [2829, 3762], 'precursor': [2830, 3763], 'supply': [2831, 3764], 'increases': [2832, 3765], 'risk': [2834, 3767, 3831], 'immunodeficiency.Mol.': [2843, 3776], '6:': [2846, 3779], '1-14Google': [2847, 3780], 'does': [2852], 'explain': [2854], 'argument': [2863], 'applied': [2866], 'microenvironment': [2871], 'even': [2873], 'during': [2874], 'culture.': [2877], '(for': [2880], 'SCID-X1)': [2881], 'common': [2885, 2930], 'form': [2886], 'SCID,': [2888], 'accounting': [2889], '40%–50%': [2891], 'all': [2893, 2969], 'cases.2Fischer': [2894], 'Mutations': [2919], 'IL2RG': [2922], 'lead': [2924], 'defective': [2926], 'cytokine': [2931, 2945], 'receptor': [2932, 2957, 3856], 'gamma': [2933], 'chain': [2934], '(gc),': [2935], 'so-called': [2936], 'subunit': [2941], 'shared': [2942], 'multiple': [2944], 'receptors,': [2946], 'interleukin': [2949], '(IL)-2,': [2950], 'IL-4,': [2951], 'IL-7,': [2952], 'IL-9,': [2953], 'IL-15,': [2954], 'IL-21': [2956], 'complexes,': [2958], 'variably': [2961], 'involved': [2962], 'function': [2967, 2999], 'lymphocytes.': [2970, 3002], 'As': [2971], 'consequence,': [2973], 'show': [2975], 'adaptive': [2979], 'immunity,': [2980], 'absence': [2987, 3381, 3714, 3805], '(NK)': [2993], 'loss': [2997], 'B': [3001, 3396], 'SCID-X1': [3012, 3495, 3551, 3649, 3894, 4188], 'included': [3013], 'total': [3015], '20': [3018], 'subjects': [3019], 'conducted': [3022], 'virtually': [3024], 'identical': [3025], 'protocols.42Cavazzana-Calvo': [3033], 'Hacein-Bey': [3035], 'de': [3037, 3273, 3534, 3597, 3632, 4171], 'Saint': [3038, 3274], 'Basile': [3039, 3275], 'Gross': [3041], 'Yvon': [3043], 'Nusbaum': [3045], 'Selz': [3047], 'Hue': [3049], 'Certain': [3051], '(SCID)-X1': [3063], 'disease.Science.': [3064], '2000;': [3065], '288:': [3066], '669-672Crossref': [3067], '(1861)': [3070], '43Gaspar': [3073], 'Howe': [3077, 3173, 3473], 'Brouns': [3085], 'Von': [3089, 3430], 'Barington': [3092], 'pseudotyped': [3106], 'vector.Lancet.': [3108], '364:': [3110], '2181-2187Abstract': [3111], '(469)': [3119], '44Hacein-Bey-Abina': [3122], 'Hauer': [3124], 'Picard': [3128], 'Wang': [3130, 3665, 4204], 'G.P.': [3131, 3666, 4205], 'Berry': [3132], 'C.C.': [3133], 'Martinache': [3134], 'Rieux-Laucat': [3136], 'Latour': [3138], 'Belohradsky': [3140], 'B.H.': [3141], 'al.Efficacy': [3143], 'immunodeficiency.N.': [3151], '363:': [3156], '355-364Crossref': [3157], '(342)': [3160], '45Gaspar': [3163], 'S.J.': [3174, 3474, 3517, 3533, 3615, 3631, 4154, 4170], 'Al': [3175], 'Ghonaium': [3176], 'al.Long-term': [3185], 'persistence': [3186], 'polyclonal': [3189], 'repertoire': [3192, 3238], 'immunodeficiency.Sci.': [3200], '97ra79Crossref': [3205], 'Neither': [3211], 'study': [3212], 'used': [3213], 'preconditioning,': [3214], 'extremely': [3220], 'high': [3221], 'lymphocyte': [3226], 'precursors': [3227, 3967], 'highly': [3234], '(as': [3239], 'infusions,': [3244], 'variant': [3248], 'rare': [3251], 'somatic': [3252, 3332, 3543, 3641, 4180], 'reversion': [3253, 3286, 3333], 'events).46Stephan': [3254], 'Wahn': [3256], 'Le': [3258], 'Dirksen': [3261], 'Broker': [3263], 'Müller-Fleckenstein': [3265], 'Horneff': [3267], 'Schroten': [3269], 'Atypical': [3277], 'possible': [3284], 'spontaneous': [3285], 'defect': [3290], 'cells.N.': [3293], '335:': [3298], '1563-1567Crossref': [3299], '47Speckmann': [3305], 'Pannicke': [3307], 'Wiech': [3309], 'Schwarz': [3311], 'Fisch': [3313], 'Niehues': [3317], 'Buiting': [3321], 'Schlesier': [3323], 'immunologic': [3328], 'consequences': [3329], '2008;': [3342, 3555, 3653, 3695, 4192], '112:': [3343], '4090-4097Crossref': [3344], 'Successful': [3350], 'functional': [3354], 'compartment': [3357], 'occurred': [3358], 'sustained': [3365, 3384], 'greater': [3371], '80%': [3373], 'outcome.': [3378], 'conditioning,': [3383], '(HSCs)': [3390], 'did': [3391], 'occur;': [3393], 'consequently,': [3394], 'NK': [3399], 'low.': [3404], 'Unfortunately,': [3405], '31–68': [3406], 'months': [3407], 'five': [3411, 3566], 'reconstituted': [3420], 'acute': [3425, 3613], 'lymphoblastic': [3426], '(T-ALL).48Hacein-Bey-Abina': [3428], 'Wulffraat': [3437], 'Leboulch': [3439], 'Osborne': [3443], 'Pawliuk': [3445], 'Morillon': [3447, 3671, 4210], 'al.LMO2-associated': [3450], 'proliferation': [3454], 'SCID-X1.Science.': [3462], '2003;': [3463], '302:': [3464], '415-419Crossref': [3465], '(2288)': [3468], '49Schwarzwaelder': [3471], 'Brugman': [3477, 3528, 3626, 4165], 'M.H.': [3478, 3529, 3627, 4166], 'Deichmann': [3479], 'Glimm': [3481], 'Prinz': [3485], 'Wissler': [3487], 'al.Gammaretrovirus-mediated': [3492], 'skewed': [3499], 'site': [3502], 'distribution': [3503], 'vivo.J.': [3505], '2241-2249Crossref': [3510], '(152)': [3513], '50Howe': [3516], 'Mansour': [3518, 3616, 4155], 'M.R.': [3519, 3617, 4156], 'Schwarzwaelder': [3520, 3618, 4157], 'Hubank': [3524, 3622, 4161], 'Kempski': [3526, 3624, 4163], 'Pike-Overzet': [3530, 3628, 4167], 'Chatters': [3532, 3630, 4169], 'Ridder': [3535, 3633, 4172], 'al.Insertional': [3538, 3636, 3682, 4175], 'acquired': [3542, 3640, 4179], 'mutations': [3544, 3642, 4181], 'leukemogenesis': [3546, 3644, 3853, 4183], 'patients.J.': [3552, 3650, 4189], '118:': [3556, 3654, 3696, 4193], '3143-3150Crossref': [3557, 3655, 4194], '(615)': [3560, 3658, 4197], 'four': [3564, 3939], 'enhancer-mediated': [3568], 'upregulation': [3569], 'LMO-2': [3572], 'proto-oncogene': [3573], 'directly': [3575, 3601], 'implicated': [3576], 'leukemic': [3579, 3733], 'process,': [3580], 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