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Cellular Therapies for Muscular Dystrophies: Frustrations and Clinical Successes.

Research paper by Elisa E Negroni, Anne A Bigot, Gillian S GS Butler-Browne, Capucine C Trollet, Vincent V Mouly

Indexed on: 15 Dec '15Published on: 15 Dec '15Published in: Human gene therapy



Abstract

Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic strategy. In this review we summarize progress in preclinical cell therapy approaches, with a special emphasis on human cells potentially attractive for human clinical trials. Future perspectives for cell therapy in skeletal muscle are discussed, including the perspective of combined therapeutic approaches.