Stem cell therapies for muscle disorders

被引:71
作者
Tedesco, Francesco S. [1 ,2 ,3 ]
Cossu, Giulio [1 ,2 ,3 ]
机构
[1] UCL, Dept Cell & Dev Biol, London WC1E 6DE, England
[2] UCL, Ctr Stem Cells & Regenerat Med, London WC1E 6DE, England
[3] Ist Sci San Raffaele, Div Regenerat Med Stem Cells & Gene Therapy, I-20132 Milan, Italy
基金
英国医学研究理事会; 欧洲研究理事会;
关键词
cell therapy; diseases of skeletal muscle; muscular dystrophy; myogenic stem/progenitor cells; DUCHENNE MUSCULAR-DYSTROPHY; HUMAN SKELETAL-MUSCLE; SATELLITE CELLS; SELF-RENEWAL; MYOBLAST TRANSPLANTATION; ALPHA-SARCOGLYCAN; IN-VITRO; REGENERATION; GENERATION; PERICYTES;
D O I
10.1097/WCO.0b013e328357f288
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Purpose of review This review focuses on stem cell-based therapies to treat skeletal muscle disorders, with a special emphasis on muscular dystrophies. Recent findings We briefly review previous attempts at cell therapy by the use of donor myoblasts, explaining the likely reasons for the poor clinical results; we then describe the use of the same cells in current promising trials for localized treatments of different diseases of skeletal muscle. Moreover, we discuss important novel findings on muscle stem/progenitor cell biology and their promise for future clinical translation. Preclinical and clinical applications of novel myogenic stem/progenitor cells are also described. Summary We summarize several ongoing clinical trials for different muscle disorders and the advances in the understanding of the biology of the myogenic progenitors used in such trials. On the basis of the currently available information, a prediction of developments in the field is proposed.
引用
收藏
页码:597 / 603
页数:7
相关论文
共 77 条
[71]   Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy [J].
Tedesco, Francesco Saverio ;
Gerli, Mattia F. M. ;
Perani, Laura ;
Benedetti, Sara ;
Ungaro, Federica ;
Cassano, Marco ;
Antonini, Stefania ;
Tagliafico, Enrico ;
Artusi, Valentina ;
Longa, Emanuela ;
Tonlorenzi, Rossana ;
Ragazzi, Martina ;
Calderazzi, Giorgia ;
Hoshiya, Hidetoshi ;
Cappellari, Ornella ;
Mora, Marina ;
Schoser, Benedikt ;
Schneiderat, Peter ;
Oshimura, Mitsuo ;
Bottinelli, Roberto ;
Sampaolesi, Maurilio ;
Torrente, Yvan ;
Broccoli, Vania ;
Cossu, Giulio .
SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (140)
[72]   Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy [J].
Tedesco, Francesco Saverio ;
Hoshiya, Hidetoshi ;
D'Antona, Giuseppe ;
Gerli, Mattia F. M. ;
Messina, Graziella ;
Antonini, Stefania ;
Tonlorenzi, Rossana ;
Benedetti, Sara ;
Berghella, Libera ;
Torrente, Yvan ;
Kazuki, Yasuhiro ;
Bottinelli, Roberto ;
Oshimura, Mitsuo ;
Cossu, Giulio .
SCIENCE TRANSLATIONAL MEDICINE, 2011, 3 (96)
[73]   Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells [J].
Tedesco, Francesco Saverio ;
Dellavalle, Arianna ;
Diaz-Manera, Jordi ;
Messina, Graziella ;
Cossu, Giulio .
JOURNAL OF CLINICAL INVESTIGATION, 2010, 120 (01) :11-19
[74]   Development of cellular therapy for the treatment of stress urinary incontinence [J].
Wang, Hung-Jen ;
Chuang, Yao-Chi ;
Chancellor, Michael B. .
INTERNATIONAL UROGYNECOLOGY JOURNAL, 2011, 22 (09) :1075-1083
[75]   Satellite cells, the engines of muscle repair [J].
Wang, Yu Xin ;
Rudnicki, Michael A. .
NATURE REVIEWS MOLECULAR CELL BIOLOGY, 2012, 13 (02) :127-133
[76]   Harnessing the potential of induced pluripotent stem cells for regenerative medicine (vol 13, pg 497, 2011) [J].
Wu, Sean M. ;
Hochedlinger, Konrad .
NATURE CELL BIOLOGY, 2011, 13 (06) :734-734
[77]   A Fresh Look at iPS Cells [J].
Yamanaka, Shinya .
CELL, 2009, 137 (01) :13-17