Internal medicine

Virus

Adeno-associated virus

Bioinformatics

Biology

Gene

Genetic enhancement

Virology

Recombinant DNA

Factor IX

Vector (molecular biology)

Viral vector

Clinical trial

Genetics

Medicine

Annual Review of Medicine

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.

Adeno-Associated Virus Gene Therapy for Hemophilia