A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

To develop safer and more effective vectors for gene therapy of X-linked severe combined immunodeficiency (SCID-X1), we have evaluated new self-inactivating lentiviral vectors based on the HIV virus. The CL20i4-hγ(c)-Revgen vector contains the entire human common γ chain (γ(c)) genomic sequence driv...

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Bibliographische Detailangaben
Hauptverfasser: Zhou, Sheng, Mody, Disha, DeRavin, Suk See, Hauer, Julia, Lu, Taihe, Ma, Zhijun, Hacein-Bey Abina, Salima, Gray, John T., Greene, Michael R., Cavazzana-Calvo, Marina, Malech, Harry L., Sorrentino, Brian P.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Hematology 2010
Schlagworte:
Gene Therapy
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2924228/
https://ncbi.nlm.nih.gov/pubmed/20457870
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-10-250209
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