Gene Therapy For Hemophilia A Mice

Hemophilia A is an inherited bleeding disorder caused by a lack of the blood clotting protein Factor VIII. Current gene therapy approaches to treat the condition have had little clinical success. However, hope is provided by researchers who have developed a new approach to target genes to the cells that are the main source of Factor VIII and used it to provide long-term expression of Factor VIII in hemophilia A mice, markedly reducing their disease.

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