Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

Chronic granulomatous disease (CGD) is associated with significant morbidity and mortality from infection. The first CGD gene therapy trial resulted in only short-term marking of 0.01% to 0.1% of neutrophils. A recent study, using busulfan conditioning and an SFFV retrovirus vector, achieved more th...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Kang, Elizabeth M., Choi, Uimook, Theobald, Narda, Linton, Gilda, Long Priel, Debra A., Kuhns, Doug, Malech, Harry L.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Hematology 2010
Pynciau:
Gene Therapy
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC2815517/
https://ncbi.nlm.nih.gov/pubmed/19965657
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-05-222760
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