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CRISPR Correction of Duchenne Muscular Dystrophy.
Annu Rev Med. 2019 01 27; 70:239-255.AR

Abstract

The ability to efficiently modify the genome using CRISPR technology has rapidly revolutionized biology and genetics and will soon transform medicine. Duchenne muscular dystrophy (DMD) represents one of the first monogenic disorders that has been investigated with respect to CRISPR-mediated correction of causal genetic mutations. DMD results from mutations in the gene encoding dystrophin, a scaffolding protein that maintains the integrity of striated muscles. Thousands of different dystrophin mutations have been identified in DMD patients, who suffer from a loss of ambulation followed by respiratory insufficiency, heart failure, and death by the third decade of life. Using CRISPR to bypass DMD mutations, dystrophin expression has been efficiently restored in human cells and mouse models of DMD. Here, we review recent progress toward the development of possible CRISPR therapies for DMD and highlight opportunities and potential obstacles in attaining this goal.

Authors+Show Affiliations

Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, and Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, Texas 75390, USA; email: Eric.Olson@utsouthwestern.edu.Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, and Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, Texas 75390, USA; email: Eric.Olson@utsouthwestern.edu.Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, and Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, Texas 75390, USA; email: Eric.Olson@utsouthwestern.edu.

Pub Type(s)

Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

30379597

Citation

Min, Yi-Li, et al. "CRISPR Correction of Duchenne Muscular Dystrophy." Annual Review of Medicine, vol. 70, 2019, pp. 239-255.
Min YL, Bassel-Duby R, Olson EN. CRISPR Correction of Duchenne Muscular Dystrophy. Annu Rev Med. 2019;70:239-255.
Min, Y. L., Bassel-Duby, R., & Olson, E. N. (2019). CRISPR Correction of Duchenne Muscular Dystrophy. Annual Review of Medicine, 70, 239-255. https://doi.org/10.1146/annurev-med-081117-010451
Min YL, Bassel-Duby R, Olson EN. CRISPR Correction of Duchenne Muscular Dystrophy. Annu Rev Med. 2019 01 27;70:239-255. PubMed PMID: 30379597.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - CRISPR Correction of Duchenne Muscular Dystrophy. AU - Min,Yi-Li, AU - Bassel-Duby,Rhonda, AU - Olson,Eric N, Y1 - 2018/10/31/ PY - 2018/11/1/pubmed PY - 2018/11/1/medline PY - 2018/11/1/entrez KW - CRISPR KW - dystrophin KW - muscular dystrophy KW - skeletal muscle SP - 239 EP - 255 JF - Annual review of medicine JO - Annu. Rev. Med. VL - 70 N2 - The ability to efficiently modify the genome using CRISPR technology has rapidly revolutionized biology and genetics and will soon transform medicine. Duchenne muscular dystrophy (DMD) represents one of the first monogenic disorders that has been investigated with respect to CRISPR-mediated correction of causal genetic mutations. DMD results from mutations in the gene encoding dystrophin, a scaffolding protein that maintains the integrity of striated muscles. Thousands of different dystrophin mutations have been identified in DMD patients, who suffer from a loss of ambulation followed by respiratory insufficiency, heart failure, and death by the third decade of life. Using CRISPR to bypass DMD mutations, dystrophin expression has been efficiently restored in human cells and mouse models of DMD. Here, we review recent progress toward the development of possible CRISPR therapies for DMD and highlight opportunities and potential obstacles in attaining this goal. SN - 1545-326X UR - https://www.unboundmedicine.com/medline/citation/30379597/CRISPR_Correction_of_Duchenne_Muscular_Dystrophy_ L2 - http://arjournals.annualreviews.org/doi/full/10.1146/annurev-med-081117-010451?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -