Adeno-associated Virus Gene Therapy for Hemophilia

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Abstract

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 in the near future for the current generation of HA and HB AAV vectors. Here we review the clinical development of AAV gene therapy for HA and HB and examine outstanding questions that have recently emerged from AAV clinical trials for hemophilia and other monogenic disorders.

Expected final online publication date for the Annual Review of Medicine, Volume 74 is January 2023. Please see http://www.annualreviews.org/page/journal/pubdates for revised estimates.

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Journal

Title: Annual Review of Medicine

Abbreviated Title: Annu. Rev. Med.

Publisher: Annual Reviews

ISSN (Print): 0066-4219

ISSN (Electronic): 1545-326X

Publication date Created: January 27 2023

Publication date (Electronic): September 14 2022

Publication date (Print): January 27 2023

Volume: 74

Issue: 1

Affiliations

[1 ]Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA;

[2 ]Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, the Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA

Article

DOI: 10.1146/annurev-med-043021-033013

SO-VID: 635880c2-f5c1-4890-a877-48edeee39a01

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