Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

Gene transfer into autologous hematopoietic stem cells by γ-retroviral vectors (gRV) is an effective treatment for adenosine deaminase (ADA)–deficient severe combined immunodeficiency (SCID). However, current gRV have significant potential for insertional mutagenesis as reported in clinical trials f...

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Main Authors: Carbonaro, Denise A, Zhang, Lin, Jin, Xiangyang, Montiel-Equihua, Claudia, Geiger, Sabine, Carmo, Marlene, Cooper, Aaron, Fairbanks, Lynette, Kaufman, Michael L, Sebire, Neil J, Hollis, Roger P, Blundell, Michael P, Senadheera, Shantha, Fu, Pei-Yu, Sahaghian, Arineh, Chan, Rebecca Y, Wang, Xiaoyan, Cornetta, Kenneth, Thrasher, Adrian J, Kohn, Donald B, Gaspar, H Bobby
Formáid: Artigo
Teanga:Inglês
Foilsithe: Nature Publishing Group 2014
Ábhair:
Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3944341/
https://ncbi.nlm.nih.gov/pubmed/24256635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.265
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