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Myogenic cell transplantation in genetic and acquired diseases of skeletal muscle

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Item Type:Review
Title:Myogenic cell transplantation in genetic and acquired diseases of skeletal muscle
Creators Name:Boyer, O. and Butler-Browne, G. and Chinoy, H. and Cossu, G. and Galli, F. and Lilleker, J.B. and Magli, A. and Mouly, V. and Perlingeiro, R.R. and Previtali, S.C. and Sampaolesi, M. and Smeets, H. and Schoewel-Wolf, V. and Spuler, S. and Torrente, Y. and Van Tienen, F.
Abstract: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.
Keywords:Cell Transplantation, Muscle Stem Cells, Muscular Dystrophies, Mitochondrial Myopathies, Inflammatory Myopathies, Sphincter Incontinence, Volumetric Muscle Loss, Animals, Mice
Source:Frontiers in Genetics
Publisher:Frontiers Media SA
Page Range:702547
Date:August 2021
Additional Information:Elisabetta Gazzerro is part of the Study Group.
Official Publication:https://doi.org/10.3389/fgene.2021.702547
PubMed:View item in PubMed

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