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Search results for "Hemophilia"

Researchers at the Mount Sinai School of Medicine developed a technique for inserting genes into specific sites on the genome in liver cells. The genes are inserted into non-coding regions of the ...


A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy. The study is published in the Proceedings of the Nation ...


Researchers have previously reported the preliminary safety and efficacy data of a novel gene transfer approach in six subjects with severe haemophilia B ( Nathwani et al,NEJM365:2357–65, 2011 ). In b ...