Correction of genetic blood defects by gene transfer.
Recent clinical trials in patients with a severe combined immunodeficiency disease demonstrate that gene therapy is a powerful tool in the treatment of genetic blood defects. Recent identification of the genes involved in the pathogenesis of inherited lymphohemopoietic disorders led to animal models of gene transfer. Extensive preclinical studies have overcome some of the obstacles involved in the transduction of hemopoietic cells. These promising results led to the approval of several clinical trials that are currently underway. This review focuses on the clinical outcome in patients with genetic blood defects treated by gene transfer and examines the progress achieved to date and the problems that have been encountered. Despite the obstacles, improved clinical results for several of these diseases are expected within the next 5 years.
Cavazzana-Calvo
M
M
Laboratoire de Thérapie Cellulaire et Génique AP-HP and INSERM U429, Hôpital Necker-Enfants Malades, Paris, France. cavazzan@necker.fr
Hacein-Bey-Abina
S
S
D000818:Animals; D001796:Blood Protein Disorders / Q000235:genetics / Q000628:therapy*; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D006801:Humans