Intrinsic Myogenic Potential of Skeletal Muscle-Derived Pericytes from Patients with Myotonic Dystrophy Type 1

Overview

Journal Mol Ther Methods Clin Dev

Publisher Cell Press

Date 2019 Oct 26

PMID 31649961

Citations 4

Authors

Cornelia Rosanne Maria Ausems

Renee Henrica Lamberta Raaijmakers

Walterus Johannes Antonius Adriana van den Broek

Marieke Willemse

Baziel Gerardus Maria van Engelen

Derick Gert Wansink

Hans van Bokhoven

Affiliations

Soon will be listed here.

Abstract

Pericytes are multipotent, vessel-associated progenitors that exhibit high proliferative capacity, can cross the blood-muscle barrier, and have the ability to home to muscle tissue and contribute to myogenesis. Consequently, pericyte-based therapies hold great promise for muscular dystrophies. A complex multi-system disorder exhibiting muscular dystrophy for which pericytes might be a valuable cell source is myotonic dystrophy type 1 (DM1). DM1 is caused by an unstable (CTG)n repeat in the gene and characterized by skeletal muscle weakness, muscle wasting, and myotonia. We have successfully isolated alkaline phosphatase-positive pericytes from skeletal muscle of DM1 patients and a transgenic mouse model. Intranuclear (CUG)n RNA foci, a pathogenic DM1 hallmark, were identified in human and mouse pericytes. Notably, pericytes from DM1 patients maintained similar growth parameters and innate myogenic characteristics compared to cells from unaffected controls. Our results thus demonstrate the potential of pericytes to ameliorate muscle features in DM1 in a therapeutic setting.

Citing Articles

Ameliorated cellular hallmarks of myotonic dystrophy in hybrid myotubes from patient and unaffected donor cells.

Raaijmakers R, Ausems C, Willemse M, Cumming S, van Engelen B, Monckton D Stem Cell Res Ther. 2024; 15(1):302.

PMID: 39278936 PMC: 11403792. DOI: 10.1186/s13287-024-03913-y.

Approaches for the isolation and long-term expansion of pericytes from human and animal tissues.

Alvino V, Mohammed K, Gu Y, Madeddu P Front Cardiovasc Med. 2023; 9:1095141.

PMID: 36704463 PMC: 9873410. DOI: 10.3389/fcvm.2022.1095141.

Myonuclear transcriptional dynamics in response to exercise following satellite cell depletion.

Wen Y, Englund D, Peck B, Murach K, McCarthy J, Peterson C iScience. 2021; 24(8):102838.

PMID: 34368654 PMC: 8326190. DOI: 10.1016/j.isci.2021.102838.

Systemic cell therapy for muscular dystrophies : The ultimate transplantable muscle progenitor cell and current challenges for clinical efficacy.

Ausems C, van Engelen B, van Bokhoven H, Wansink D Stem Cell Rev Rep. 2020; 17(3):878-899.

PMID: 33349909 PMC: 8166694. DOI: 10.1007/s12015-020-10100-y.

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