Phenotypic correction of Fanconi anemia cells in the murine bone marrow after carrier cell mediated delivery of lentiviral vector

Abstract Fanconi anemia (FA) is an autosomal-recessive disorder associated with hematopoietic failure and it is a candidate for hematopoietic stem cell (HSC)-directed gene therapy. However, the characteristically reduced HSC numbers found in FA patients, their ineffective mobilization from the marro...

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Автори:	Santhosh Chakkaramakkil Verghese, Natalya A. Goloviznina, Peter Kurre
Формат:	Artigo
Мова:	Inglês
Опубліковано:	BMC 2016-11-01
Серія:	Stem Cell Research & Therapy
Предмети:	Fanconi Anemia Gene Therapy In situ gene delivery Lentiviral vector Hematopoietic stem cells FA gene therapy
Онлайн доступ:	http://link.springer.com/article/10.1186/s13287-016-0431-z
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