Frontiers in Genetics (Aug 2021)
Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle
- Olivier Boyer,
- Gillian Butler-Browne,
- Hector Chinoy,
- Hector Chinoy,
- Giulio Cossu,
- Giulio Cossu,
- Giulio Cossu,
- Francesco Galli,
- James B. Lilleker,
- James B. Lilleker,
- Alessandro Magli,
- Vincent Mouly,
- Rita C. R. Perlingeiro,
- Stefano C. Previtali,
- Maurilio Sampaolesi,
- Maurilio Sampaolesi,
- Hubert Smeets,
- Hubert Smeets,
- Hubert Smeets,
- Verena Schoewel-Wolf,
- Simone Spuler,
- Yvan Torrente,
- Florence Van Tienen,
- Florence Van Tienen,
- Study Group,
- H. Aldearee,
- A. Bisson,
- L. Bragg,
- V. Bridoux,
- R. Duelen,
- A. Farini,
- E. Gazzero,
- N. Giarratana,
- C. Giverne,
- L. Meggiolaro,
- E. Negroni,
- E. Porrello,
- R. Tonlorenzi,
- C. Villa,
- L. Yedigaryan,
- A. Zamboni
Affiliations
- Olivier Boyer
- Department of Immunology & Biotherapy, Rouen University Hospital, Normandy University, Inserm U1234, Rouen, France
- Gillian Butler-Browne
- Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France
- Hector Chinoy
- Manchester Centre for Clinical Neurosciences, Manchester Academic Health Science Centre, Salford Royal NHS Foundation Trust, Salford, United Kingdom
- Hector Chinoy
- National Institute for Health Research Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, The University of Manchester, Manchester, United Kingdom
- Giulio Cossu
- Division of Cell Matrix Biology & Regenerative Medicine, The University of Manchester, Manchester, United Kingdom
- Giulio Cossu
- Muscle Research Unit, Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Berlin, Germany
- Giulio Cossu
- InSpe and Division of Neuroscience, Istituto di Ricerca e Cura a Carattere Scientifico (IRCCS) Ospedale San Raffaele, Milan, Italy
- Francesco Galli
- National Institute for Health Research Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, The University of Manchester, Manchester, United Kingdom
- James B. Lilleker
- Manchester Centre for Clinical Neurosciences, Manchester Academic Health Science Centre, Salford Royal NHS Foundation Trust, Salford, United Kingdom
- James B. Lilleker
- National Institute for Health Research Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, The University of Manchester, Manchester, United Kingdom
- Alessandro Magli
- Department of Medicine, Lillehei Heart Institute, Stem Cell Institute, University of Minnesota, Minneapolis, MN, United States
- Vincent Mouly
- Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France
- Rita C. R. Perlingeiro
- Department of Medicine, Lillehei Heart Institute, Stem Cell Institute, University of Minnesota, Minneapolis, MN, United States
- Stefano C. Previtali
- InSpe and Division of Neuroscience, Istituto di Ricerca e Cura a Carattere Scientifico (IRCCS) Ospedale San Raffaele, Milan, Italy
- Maurilio Sampaolesi
- Translational Cardiomyology Laboratory, Department of Development and Regeneration, KU Leuven, Leuven, Belgium
- Maurilio Sampaolesi
- 0Human Anatomy Unit, Department of Public Health, Experimental and Forensic Medicine, University of Pavia, Pavia, Italy
- Hubert Smeets
- 1Department of Toxicogenomics, Maastricht University Medical Centre, Maastricht, Netherlands
- Hubert Smeets
- 2School for Mental Health and Neurosciences (MHeNS), Maastricht University, Maastricht, Netherlands
- Hubert Smeets
- 3School for Developmental Biology and Oncology (GROW), Maastricht University, Maastricht, Netherlands
- Verena Schoewel-Wolf
- Muscle Research Unit, Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Berlin, Germany
- Simone Spuler
- Muscle Research Unit, Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Berlin, Germany
- Yvan Torrente
- 4Unit of Neurology, Stem Cell Laboratory, Department of Pathophysiology and Transplantation, Centro Dino Ferrari, Università degli Studi di Milano, Fondazione Istituto di Ricerca e Cura a Carattere Scientifico (IRCCS) Cà Granda Ospedale Maggiore Policlinico, Milan, Italy
- Florence Van Tienen
- 1Department of Toxicogenomics, Maastricht University Medical Centre, Maastricht, Netherlands
- Florence Van Tienen
- 2School for Mental Health and Neurosciences (MHeNS), Maastricht University, Maastricht, Netherlands
- Study Group
- H. Aldearee
- A. Bisson
- L. Bragg
- V. Bridoux
- R. Duelen
- A. Farini
- E. Gazzero
- N. Giarratana
- C. Giverne
- L. Meggiolaro
- E. Negroni
- E. Porrello
- R. Tonlorenzi
- C. Villa
- L. Yedigaryan
- A. Zamboni
- DOI
- https://doi.org/10.3389/fgene.2021.702547
- Journal volume & issue
-
Vol. 12
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