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Stem Cell Week

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Gene Therapy



Studies from Columbus Children's Hospital in the area of gene therapy described



August 25th, 2008

"Fanconi anemia ( FA) is a rare recessive syndrome, characterized by congenital anomalies, bone marrow failure, and predisposition to cancer. Two earlier clinical trials utilizing gamma-retroviral vectors for the transduction of autologous FA hematopoietic stem cells ( HSCs) required extensive in vitro manipulation and failed to achieve detectable long-term engraftment of transduced HSCs," scientists in the United States report.

"As a strategy for minimizing ex vivo manipulation, we investigated the use of a ''rapid'' lentiviral transduction protocol in a murine Fanca(-/-) model. Importantly, while this and most murine models of FA fail to completely mimic the human...


Source: Stem Cell Week (2008-08-25)

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