Concepedia
Methods in enzymology on CD-ROM/Methods in enzymology · 2012 · 23 citations · 8 references
Gene TherapiesEngineeringNovel TherapyMedicineDrug DiscoveryRegulatory StructuresGenetic EngineeringEuropean UnionTherapeutic AreaAntisense TherapyPharmacologyGenome EditingBiomolecular Engineering
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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text
Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Alessandro Aiuti, Shimon Slavin, Memet Aker et al. · Science · 2002 · 1.1K citations
Cell Therapy, Immunology, Nonmyeloablative Conditioning +17
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
Michael G. Kaplitt, Andrew Feigin, Chengke Tang et al. · The Lancet · 2007 · 1.1K citations
Adeno-associated Virus, Medical Treatment, Borne Gad Gene +4
Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
Elizabeth M. Kang, Uimook Choi, Narda Theobald et al. · Blood · 2009 · 190 citations · Full text
Phase I Study of Dystrophin Plasmid-Based Gene Therapy in Duchenne/Becker Muscular Dystrophy
Norma B. Romero, Serge Braun, Olivier Benvéniste et al. · Human Gene Therapy · 2004 · 149 citations