This article will review myogenic cell transplantation for congenital and acquired diseases of skeletal muscle. There are already a number of excellent reviews on this topic, but they are mostly focused on a specific disease, muscular dystrophies and in particular Duchenne Muscular Dystrophy. There are also recent reviews on cell transplantation for inflammatory myopathies, volumetric muscle loss (VML) (this usually with biomaterials), sarcopenia and sphincter incontinence, mainly urinary but also fecal. We believe it would be useful at this stage, to compare the same strategy as adopted in all these different diseases, in order to outline similarities and differences in cell source, pre-clinical models, administration route, and outcome measures. This in turn may help to understand which common or disease-specific problems have so far limited clinical success of cell transplantation in this area, especially when compared to other fields, such as epithelial cell transplantation. We also hope that this may be useful to people outside the field to get a comprehensive view in a single review. As for any cell transplantation procedure, the choice between autologous and heterologous cells is dictated by a number of criteria, such as cell availability, possibility of in vitro expansion to reach the number required, need for genetic correction for many but not necessarily all muscular dystrophies, and immune reaction, mainly to a heterologous, even if HLA-matched cells and, to a minor extent, to the therapeutic gene product, a possible antigen for the patient. Finally, induced pluripotent stem cell derivatives, that have entered clinical experimentation for other diseases, may in the future offer a bank of immune-privileged cells, available for all patients and after a genetic correction for muscular dystrophies and other myopathies.
Bibliographical noteFunding Information:
Work in the authors laboratories was supported by grants from PHRC National Research Program (P020908/AOM02100), Association Française contre les Myopathies (GB-B, VM, and OB); Fondation de l’Avenir (OB); NIHR Manchester Biomedical Research Centre Funding Scheme (HC); ERC AIG 844952, EC H2020-MSCA-ITN-2019 860034, UMIP No. 20150256, MRC MR/S015116, Duchenne Parent Project (Italy), GOSH V4618, Wellcome Trust HICF 107572 (GC); NIHR Clinical Lecturship MWN/006/025/A (JL); Greg Marzolf Junior Foundation Award (AM); NIH R01AR071439, Department of Defense MD160035, Duchenne UK 2019-08 (RCRP); Fondazione Regionale per la Ricerca Biomedica FRRB CP2_10/2018, EJPRD19-118, Telethon GGP17009, Ministry of Health RF-2016-02361246 (SCP); INTERREG—Euregio Meuse-Rhine, GYM— Generate your muscle 2020-EMR116, FWO (G0D4517N and G066821N), C1-KU Leuven grant 3DMUSYC, C14/17/111 (MS); Deutsche Forschungsgemeinschaft (DFG) KFO192, DFG IGK1631: MyoGrad, the Helmholtz Validation Fund/Helmholtz Association, SPARK-Berlin Institute of Health Validation Fund, the Foundations Gisela Krebs and Else Kröner-Fresenius (VS-W); French Telethon AFM 21104, Ricerca Finalizzata 2016 (YT); Interreg EMR 116, Metakids “ING goede doelen fonds” (2014-055), and the Prinses Beatrix Spierfonds (PBS W.0R15-09) (HS and FV).
© Copyright © 2021 Boyer, Butler-Browne, Chinoy, Cossu, Galli, Lilleker, Magli, Mouly, Perlingeiro, Previtali, Sampaolesi, Smeets, Schoewel-Wolf, Spuler, Torrente, Van Tienen and Study Group.
- cell transplantation
- inflammatory myopathies
- mitochondrial myopathies
- muscle stem cells
- muscular dystrophies
- sphincter incontinence
- volumetric muscle loss