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Advances and challenges for hemophilia gene therapy

19 Jul 2019

Abstract excerpt

Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8 (hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding episodes into the soft tissues and joints, which are treated with self-infusion of factor VIII or IX concentrates. Hemophilia provides an attractive target for gene therapy studies, due to the monogenic nature of these disorders and easily...

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PMID: 31332444
DOI: 10.1093/hmg/ddz157

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