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

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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Bibliographic Details
Main Authors: Santhosh Chakkaramakkil Verghese, Natalya A. Goloviznina, Peter Kurre
Format: Article
Language:English
Published: BMC 2016-11-01
Series:Stem Cell Research & Therapy
Subjects:
Fanconi Anemia
Gene Therapy
In situ gene delivery
Lentiviral vector
Hematopoietic stem cells
FA gene therapy
Online Access:http://link.springer.com/article/10.1186/s13287-016-0431-z

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http://link.springer.com/article/10.1186/s13287-016-0431-z

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