Adeno-Associated Virus Gene Therapy for Hemophilia
Benjamin J. Samelson‐Jones, Lindsey A. George
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.
Topics & Concepts
Adeno-associated virusGenetic enhancementMedicineFactor IXClinical trialVector (molecular biology)VirusGeneVirologyViral vectorBioinformaticsBiologyGeneticsInternal medicineRecombinant DNAVirus-based gene therapy researchCAR-T cell therapy researchViral Infectious Diseases and Gene Expression in Insects