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Mary Ann Liebert, Human Gene Therapy, 2(27), p. 117-126, 2016

DOI: 10.1089/hum.2015.139

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Cellular therapies for muscular dystrophies: frustrations and clinical successes

Journal article published in 2015 by Elisa Negroni, Anne Bigot, Gillian S. Butler-Browne, Capucine Trollet ORCID, Vincent Mouly
This paper is available in a repository.
This paper is available in a repository.

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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 recent progresses in pre-clinical 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.