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Critical Reviews™ in Eukaryotic Gene Expression

Impact factor: 1.660

ISSN Print: 1045-4403
ISSN Online: 2162-6502

Critical Reviews™ in Eukaryotic Gene Expression

DOI: 10.1615/CritRevEukaryotGeneExpr.2015011074
pages 1-11

Cell Therapy in Duchenne Muscular Dystrophy Treatment: Clinical Trials Overview

Anna Bajek
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Dorota Porowinska
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Tomasz Kloskowski
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Edyta Brzoska
Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland
Maria A. Ciemerych
Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland
Tomasz Drewa
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland; Department of Urology, Nicolaus Copernicus Hospital, Torun, Poland

ABSTRACT

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.