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© Marie Prévost, Institut Pasteur
Image of a portion of a Xenopus oocyte expressing a channel receptor.
Publication : Critical reviews in eukaryotic gene expression

Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview

Scientific Fields
Diseases
Organisms
Applications
Technique

Published in Critical reviews in eukaryotic gene expression - 01 Jan 2015

Bajek A, Porowinska D, Kloskowski T, Brzoska E, Ciemerych MA, Drewa T

Link to Pubmed [PMID] – 25955813

Crit. Rev. Eukaryot. Gene Expr. 2015;25(1):1-11

Duchenne muscular dystrophy (DMD), the most common and most severe form of all muscular dystrophies, leads to progressive muscle fiber necrosis, fibroblast proliferation, and growth of fibrous tissue and fat. The most common cause of death in DMD patients is cardiac and respiratory failure. Current pharmacological and other treatment methods do not lead to full recovery. For this reason, new alternatives for skeletal muscle regeneration are being investigated. Transplantation of myoblasts from healthy donors is one studied approach to muscle treatment in DMD patients. However, the results of intramuscular injection of in vitro cultured myoblasts are still not satisfactory. The use of autologous stem cells is also proposed. Despite many ongoing studies, this therapy is still in preliminary testing and requires more experiments.