Hemophilia clinical gene therapy: brief review.
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.
Mount Sinai School of Medicine, New York City, NY 10029, USA. Christopher-e.walsh@msnyuhealth.org
Christopher E
CE
Walsh
Katherine M
KM
Batt
D000818:Animals; D016025:Bone Transplantation; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D005169:Factor VIII / Q000235:genetics / Q000627:therapeutic use; D015316:Genetic Therapy; D006467:Hemophilia A / Q000235:genetics* / Q000628:therapy*; D006801:Humans
D005169:Factor VIII