Title: Preclinical and clinical gene therapy for haemophilia
Authors: Chuah, M K L
Collen, Desire
Vandendriessche, Thierry # ×
Issue Date: Oct-2004
Publisher: Blackwell Science
Series Title: Haemophilia vol:10 Suppl 4 pages:119-25
Abstract: 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.
ISSN: 1351-8216
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Molecular and Vascular Biology
× corresponding author
# (joint) last author

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