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Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies

O'Neill, HC; Pini, V; Muntoni, F; Morgan, J; (2017) Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies. Current Stem Cell Reports , 3 (2) pp. 137-148. 10.1007/s40778-017-0076-6. Green open access

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Abstract

PURPOSE OF REVIEW: Muscular dystrophies are a group of severe degenerative disorders characterized by muscle fiber degeneration and death. Therapies designed to restore muscle homeostasis and to replace dying fibers are being experimented, but none of those in clinical trials are suitable to permanently address individual gene mutation. The purpose of this review is to discuss genome editing tools such as CRISPR/Cas (clustered regularly interspaced short palindromic repeats/CRISPR-associated), which enable direct sequence alteration and could potentially be adopted to correct the genetic defect leading to muscle impairment. RECENT FINDINGS: Recent findings show that advances in gene therapy, when combined with traditional viral vector-based approaches, are bringing the field of regenerative medicine closer to precision-based medicine. SUMMARY: The use of such programmable nucleases is proving beneficial for the creation of more accurate in vitro and in vivo disease models. Several gene and cell-therapy studies have been performed on satellite cells, the primary skeletal muscle stem cells involved in muscle regeneration. However, these have mainly been based on artificial replacement or augmentation of the missing protein. Satellite cells are a particularly appealing target to address these innovative technologies for the treatment of muscular dystrophies.

Type: Article
Title: Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/s40778-017-0076-6
Publisher version: http://doi.org/10.1007/s40778-017-0076-6
Language: English
Additional information: © The Author(s) 2017. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
Keywords: Stem cells; Muscular dystrophies; CRISPR/Cas; Genome editing; Gene therapy; Precision medicine
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > Inst for Women's Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > Inst for Women's Health > Reproductive Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health > ICH Developmental Neurosciences Prog
URI: http://discovery.ucl.ac.uk/id/eprint/1553276
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