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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Nature Publishing Group 2014
Gaiak:
Original Article
Sarrera elektronikoa: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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