Orchard Therapeutics Gains First US Approval for a Metachromatic Leukodystrophy Gene Therapy

被引:6
作者
Philippidis, Alex [1 ]
机构
[1] Mary Ann Liebert Inc, New Rochelle, NY 10801 USA
关键词
D O I
10.1089/hum.2024.29268.bfs
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
[No abstract available]
引用
收藏
页码:215 / 218
页数:4
相关论文
共 50 条
[21]   Lentiviral vectors for in vivo gene therapy of metachromatic leukodystrophy [J].
Consiglio, A ;
Dolcetta, D ;
Follenzi, A ;
Luca, T ;
Marchesini, S ;
Gritti, A ;
Vescovi, A ;
Naldini, L ;
Bordignon, C .
JOURNAL OF NEUROCHEMISTRY, 2003, 87 :5-5
[22]   Brain gene therapy for metachromatic leukodystrophy: towards clinical trial [J].
Piguet, F. ;
Sondhi, D. ;
Colle, M. A. ;
Raoul, S. ;
Roujeau, T. ;
Vanier, M. T. ;
Bouquet, C. ;
Moullier, P. ;
Cherel, Y. ;
Hackett, N. ;
Zerah, M. ;
Aubourg, P. ;
Crystal, R. G. ;
Cartier, N. ;
Sevin, C. .
HUMAN GENE THERAPY, 2010, 21 (10) :1449-1450
[23]   AAVrh.10 intracerebral gene therapy for metachromatic leukodystrophy [J].
Piguet, Francoise ;
Sondhi, Dolan ;
Vanier, Marie ;
Piraud, Monique ;
Ahouansou, Ornella ;
Fouquet, Francoise ;
Crystal, Ronald G. ;
Aubourg, Patrick ;
Cartier, Nathalie ;
Sevin, Caroline .
HUMAN GENE THERAPY, 2009, 20 (11) :1447-1447
[24]   Brain Gene Therapy for Metachromatic Leukodystrophy: Towards Clinical Trial [J].
Piguet, Francoise ;
Sondhi, Dolan ;
Colle, Marie-Anne ;
Raoul, Sylvie ;
Roujeau, Thomas ;
Piraud, Monique ;
Ahouansou, Ornella ;
Bouquet, Celine ;
Vanier, Marie-Therese ;
Moullier, Philippe ;
Cherel, Yan ;
Hackett, Neil R. ;
Zerah, Michel ;
Aubourg, Patrick ;
Crystal, Ronald G. ;
Cartier, Nathalie ;
Sevin, Caroline .
HUMAN GENE THERAPY, 2010, 21 (06) :771-771
[25]   Gene therapy for metachromatic leukodystrophy using foamy virus vector [J].
Sweeney, Nathan ;
Regan, Cathy ;
Liu, Jiahui ;
Rupar, Tony ;
McClure, Myra .
HUMAN GENE THERAPY, 2013, 24 (05) :A27-A27
[26]   Gene therapy offers new hope for children with metachromatic leukodystrophy [J].
Kurtzberg, Joanne .
LANCET, 2022, 399 (10322) :338-339
[28]   FDA panel recommends approval of first gene therapy in US [J].
Dyer, Owen .
BMJ-BRITISH MEDICAL JOURNAL, 2017, 358
[29]   Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits mice [J].
Biffi, Alessandra ;
Capotondo, Alessia ;
Fasano, Stefania ;
del Carro, Ubaldo ;
Marchesini, Sergio ;
Azuma, Hisaya ;
Malaguti, Maria Chiara ;
Arnadio, Stefano ;
Brambilla, Riccardo ;
Grompe, Markus ;
Bordignon, Claudio ;
Quattrini, Angelo ;
Naldini, Luigi .
JOURNAL OF CLINICAL INVESTIGATION, 2006, 116 (11) :3070-3082
[30]   Brain Gene Therapy of Metachromatic Leukodystrophy: From Mice to Human Patients [J].
Piguet, Francoise ;
Sondhi, Dolan ;
Vanier, Marie ;
Piraud, Monique ;
Ahouansou, Ornella ;
Fouquet, Francoise ;
Crystal, Ronald G. ;
Aubourg, Patrick ;
Cartier, Nathalie ;
Sevin, Caroline .
HUMAN GENE THERAPY, 2009, 20 (06) :685-685