Application des techniques de thérapie génique aux maladies ostéo-articulaires

Abstract

L’apparition du concept de gène médicament a considérablement élargi le champ d’application de la thérapie génique. Initialement développées pour les maladies génétiques ou certaines formes de cancer, les stratégies de transfert génique sont désormais étudiées en vue du traitement d’autres pathologies telles que les maladies ostéo-articulaires. La polyarthrite rhumatoïde a ainsi fait l’objet du premier essai clinique de thérapie génique pour le traitement d’une maladie non létale.

Osteoarticular disorders are the major cause of disability in Europe and North America. It is estimated that rheumatoid arthritis affects 1 % of the population and that more than two third of people over age 55 develop osteoarthritis. Because there are no satisfactory treatments, gene therapy offers a new therapeutic approach. The delivery of cDNA encoding anti-arthritic proteins to articular cells has shown therapeutic efficacy in numerous animal models in vivo. Through the development and the experimentalprogresses that have been made for both rheumatoid arthritis and osteoarthritis, this review discusses the different gene therapy strategies available today and the safety issues with which they may be associated. Among the different vectors available today, adeno-associated virus seems the best candidate for a direct in vivo gene delivery approach for the treatment of joint disorders.