Article

This article considers the development of gene therapies as a potentially viable means of enhancing skeletal muscle function for the treatment of dystrophin-related muscular dystrophies. As has been discussed, however, a clearer understanding of the causative mechanisms of muscle weakness in a given disease model and identification of the precise mechanisms of action associated with promising interventions may enable the rational design of gene therapies for many additional conditions characterized by impaired muscle function. The authors anticipate that these developments also would benefit from continuing improvements in vector design that enhance gene transfer efficiency and intervention safety. Assessing technical improvements in animal disease models of varying size and physiology would be crucial for verifying their efficacy and safety and should provide solid platforms from which to identify promising intervention strategies that warrant translation to clinical application. As with all experimental approaches being devised for life-threatening conditions for which no cure currently exists, thorough but sympathetic oversight of the development process by responsible regulatory agencies will prove invaluable for the ultimate realization of what it is hoped will be the next generation of therapies for serious neuromuscular disorders.

Langue : ANGLAIS