Cell based therapy for Duchenne muscular dystrophy.
Mutations in the dystrophin gene cause an X-linked genetic disorder: Duchenne muscular dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small number of cells are required to obtain a therapeutic effect. Here, we discussed about multiple types of myogenic stem cells and their possible use to treat DMD. The identification of a stem cell population providing efficient muscle regeneration is critical for the progression of cell therapy for DMD. We speculated that the most promising possibility for the treatment of DMD is a combination of different approaches, such as gene and stem cell therapy.
Farini
Andrea
A
Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy.
Razini
Paola
P
Erratico
Silvia
S
Torrente
Yvan
Y
Meregalli
Mirella
M
D000071916:AC133 Antigen; D000818:Animals; D015703:Antigens, CD / Q000032:analysis; D015316:Genetic Therapy / Q000379:methods*; D006023:Glycoproteins / Q000032:analysis; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D010455:Peptides / Q000032:analysis; D020286:Pericytes / Q000166:cytology; D032496:Satellite Cells, Skeletal Muscle / Q000166:cytology / Q000637:transplantation; D033581:Stem Cell Transplantation; D013234:Stem Cells / Q000166:cytology / Q000378:metabolism
D000071916:AC133 Antigen; D015703:Antigens, CD; D006023:Glycoproteins; D010455:Peptides