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Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy.
NPJ Regen Med. 2016; 1NR

Abstract

Duchenne muscular dystrophy (DMD) is a genetic disease characterised by skeletal muscle degeneration and progressive muscle wasting, which is caused by loss-of-function mutations in the DMD gene that encodes for the protein dystrophin. Dystrophin has critical roles in myofiber stability and integrity by connecting the actin cytoskeleton to the extracellular matrix. Absence of dystrophin leads to myofiber fragility and contributes to skeletal muscle degeneration in DMD patients, however, accumulating evidence also indicate that muscle stem cells (also known as satellite cells) are defective in dystrophic muscles, which leads to impaired muscle regeneration. Our recent work demonstrated that dystrophin is expressed in activated satellite cells, where it regulates the establishment of satellite cell polarity and asymmetric cell division. These findings indicate that dystrophin-deficient satellite cells have intrinsic dysfunctions that contribute to muscle wasting and progression of the disease. This discovery suggests that satellite cells could be targeted to treat DMD. Here we discuss how these new findings affect regenerative therapies for muscular dystrophies. Therapies targeting satellite cells hold great potential and could have long-term efficiency owing to the high self-renewal ability of these cells.

Authors+Show Affiliations

Sprott Center for Stem Cell Research, Ottawa Hospital Research Institute, Regenerative Medicine Program, Ottawa, ON, Canada. Department of Cellular and Molecular Medicine, Faculty of Medicine, University of Ottawa, Ottawa, ON, Canada.Sprott Center for Stem Cell Research, Ottawa Hospital Research Institute, Regenerative Medicine Program, Ottawa, ON, Canada. Department of Cellular and Molecular Medicine, Faculty of Medicine, University of Ottawa, Ottawa, ON, Canada.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

29188075

Citation

Dumont, Nicolas A., and Michael A. Rudnicki. "Targeting Muscle Stem Cell Intrinsic Defects to Treat Duchenne Muscular Dystrophy." NPJ Regenerative Medicine, vol. 1, 2016.
Dumont NA, Rudnicki MA. Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy. NPJ Regenerative medicine. 2016;1.
Dumont, N. A., & Rudnicki, M. A. (2016). Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy. NPJ Regenerative Medicine, 1. https://doi.org/10.1038/npjregenmed.2016.6
Dumont NA, Rudnicki MA. Targeting Muscle Stem Cell Intrinsic Defects to Treat Duchenne Muscular Dystrophy. NPJ Regenerative medicine. 2016;1 PubMed PMID: 29188075.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy. AU - Dumont,Nicolas A, AU - Rudnicki,Michael A, Y1 - 2016/06/09/ PY - 2017/12/1/entrez PY - 2016/1/1/pubmed PY - 2016/1/1/medline JF - NPJ Regenerative medicine VL - 1 N2 - Duchenne muscular dystrophy (DMD) is a genetic disease characterised by skeletal muscle degeneration and progressive muscle wasting, which is caused by loss-of-function mutations in the DMD gene that encodes for the protein dystrophin. Dystrophin has critical roles in myofiber stability and integrity by connecting the actin cytoskeleton to the extracellular matrix. Absence of dystrophin leads to myofiber fragility and contributes to skeletal muscle degeneration in DMD patients, however, accumulating evidence also indicate that muscle stem cells (also known as satellite cells) are defective in dystrophic muscles, which leads to impaired muscle regeneration. Our recent work demonstrated that dystrophin is expressed in activated satellite cells, where it regulates the establishment of satellite cell polarity and asymmetric cell division. These findings indicate that dystrophin-deficient satellite cells have intrinsic dysfunctions that contribute to muscle wasting and progression of the disease. This discovery suggests that satellite cells could be targeted to treat DMD. Here we discuss how these new findings affect regenerative therapies for muscular dystrophies. Therapies targeting satellite cells hold great potential and could have long-term efficiency owing to the high self-renewal ability of these cells. SN - 2057-3995 UR - https://www.unboundmedicine.com/medline/citation/29188075/Targeting_muscle_stem_cell_intrinsic_defects_to_treat_Duchenne_muscular_dystrophy_ L2 - https://doi.org/10.1038/npjregenmed.2016.6 DB - PRIME DP - Unbound Medicine ER -
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