title: Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview.
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.
authors.affiliation: Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland.
authors.forename: Anna
authors.initials: A
authors.lastname: Bajek
authors.affiliation: Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland.
authors.forename: Dorota
authors.initials: D
authors.lastname: Porowinska
authors.affiliation: Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland.
authors.forename: Tomasz
authors.initials: T
authors.lastname: Kloskowski
authors.affiliation: Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland.
authors.forename: Edyta
authors.initials: E
authors.lastname: Brzoska
authors.affiliation: Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland.
authors.forename: Maria A
authors.initials: MA
authors.lastname: Ciemerych
authors.affiliation: Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland; Department of Urology, Nicolaus Copernicus Hospital, Torun, Poland.
authors.forename: Tomasz
authors.initials: T
authors.lastname: Drewa
mesh_terms: D000293:Adolescent; D064987:Cell- and Tissue-Based Therapy; D002648:Child; D002675:Child, Preschool; D006801:Humans; D015201:Meta-Analysis as Topic; D020388:Muscular Dystrophy, Duchenne / Q000473:pathology / Q000628:therapy*; D032446:Myoblasts / Q000637:transplantation*; D012038:Regeneration; D033581:Stem Cell Transplantation