Hemophilia clinical gene therapy: brief review

Christopher E Walsh; Katherine M Batt

doi:10.1016/j.trsl.2012.12.016

Hemophilia clinical gene therapy: brief review

Transl Res. 2013 Apr;161(4):307-12. doi: 10.1016/j.trsl.2012.12.016. Epub 2013 Jan 23.

Authors

Christopher E Walsh¹, Katherine M Batt

Affiliation

¹ Mount Sinai School of Medicine, New York City, NY 10029, USA. Christopher-e.walsh@msnyuhealth.org

PMID: 23352600
DOI: 10.1016/j.trsl.2012.12.016

Abstract

Genetic correction of hemophilia A and B was long considered amenable to the available gene transfer technologies. This assumption has come to fruition with the recent results of a phase I/II trial for hemophilia B. Here we review the clinical application of gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer science and technology. This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Animals
Bone Transplantation
Clinical Trials as Topic
Dependovirus / genetics
Factor VIII / genetics
Factor VIII / therapeutic use
Genetic Therapy*
Hemophilia A / genetics*
Hemophilia A / therapy*
Humans

Substances

Factor VIII