Preclinical and clinical gene therapy for haemophilia

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia Vol. 10; no. s4; pp. 119 - 125
• Main Authors: Chuah, M. K. L., Collen, D., Vandendriessche, T.
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.10.2004
• Subjects: Adenoviridae - genetics; Animals; Clinical Trials as Topic; Factor IX - genetics; Factor VIII - genetics; gene therapy; Genetic Therapy - methods; Genetic Vectors - genetics; haemophilia; Hemophilia A - genetics; Hemophilia A - therapy; Hemophilia B - genetics; Hemophilia B - therapy; Humans; Lentivirus - genetics; Moloney murine leukemia virus - genetics; vector
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/j.1365-2516.2004.00984.x

The goal of all haemophilia therapy is to prevent bleeding and its associated complications. Replacement by factor concentrates can only ever be suboptimum, and efforts are being made to correct the genetic cause of the disorder. Haemophilia is an ideal candidate for gene therapy, as it is caused by mutations in a single gene. A number of vectors have been used in an attempt to obtain therapeutic levels of factor VIII and factor IX in animal models, with some success. A number of phase 1 clinical trials have been conducted, and, although connection of the bleeding disorder was neither complete nor long‐lasting, they do offer hope for a permanent gene‐therapy cure for the disease.