Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the interleukin-2 receptor γ chain gene (IL2RG), and it is characterized by profound defects in T, B, and natural killer (NK) cell functions. Transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically corre...

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發表在:Mol Ther Methods Clin Dev
Main Authors: Poletti, Valentina, Charrier, Sabine, Corre, Guillaume, Gjata, Bernard, Vignaud, Alban, Zhang, Fang, Rothe, Michael, Schambach, Axel, Gaspar, H. Bobby, Thrasher, Adrian J., Mavilio, Fulvio
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2018
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5918176/
https://ncbi.nlm.nih.gov/pubmed/29707600
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.03.002
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