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Updated: Feb 28, 2026
CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
Published on: September 14, 2019
Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies
Veronica Pini1, Jennifer E Morgan1, Francesco Muntoni1
1Molecular and Developmental Neurosciences Program, The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH UK.
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.
Insights
Genome editing tools like CRISPR/Cas offer a promising approach to correct genetic defects in muscular dystrophies. This technology could revolutionize regenerative medicine by permanently addressing gene mutations and improving muscle regeneration.
Area of Science:
- Biomedical Science
- Genetics
- Regenerative Medicine
Background:
- Muscular dystrophies are severe genetic disorders causing progressive muscle degeneration.
- Current therapies aim to restore muscle homeostasis but do not permanently correct underlying gene mutations.
Purpose of the Study:
- To review genome editing tools, specifically CRISPR/Cas, for their potential to correct genetic defects in muscular dystrophies.
- To explore the application of these tools in regenerative medicine for treating muscle impairment.
Main Methods:
- Discussion of CRISPR/Cas (clustered regularly interspaced short palindromic repeats/CRISPR-associated) technology.
- Review of gene and cell-therapy studies, particularly focusing on satellite cells.
Main Results:
- Advances in gene therapy combined with viral vectors are driving precision-based medicine.
- Programmable nucleases like CRISPR/Cas are effective for creating accurate in vitro and in vivo disease models.
Conclusions:
- Genome editing tools offer a direct approach to alter DNA sequences and correct genetic defects.
- Satellite cells, the skeletal muscle stem cells, are a promising target for gene and cell therapies in muscular dystrophies.
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