# Adeno-Associated Virus Gene Therapy for Hemophilia

Review · Samelson-Jones BJ, George LA

- Archive id: doi:10.1146/annurev-med-043021-033013
- Published: 2022-09-14
- Venue: Annual review of medicine
- DOI: https://doi.org/10.1146/annurev-med-043021-033013
- PMID: 36103998
- PMCID: PMC9892335
- Citations: 106
- Source URL: https://doi.org/10.1146/annurev-med-043021-033013
- PDF: https://europepmc.org/articles/PMC9892335/pdf
- Provenance: europepmc

## 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.

_Abstract reproduced under CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)._

Topics: [Gene & Cell Therapy](https://space-kitty.com/topics/gene-cell-therapy)

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