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Adeno-Associated Virus Gene Therapy for Hemophilia

14 Sept 2022

Abstract excerpt

In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy. This effort is soon to bear fruit with completed pivotal adeno-associated viral (AAV) vector gene addition trials reporting encouraging results and regulatory approval widely anticipated...

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PMID: 36103998
DOI: 10.1146/annurev-med-043021-033013

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