In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Open accessAbstract
Editing can help build stronger muscles: Much of the controversy surrounding the gene-editing technology called CRISPR/Cas9 centers on the ethics of germline editing of human embryos to correct disease-causing mutations. For certain disorders such as muscular dystrophy, it may be possible to achieve therapeutic benefit by editing the faulty gene in somatic cells. In proof-of-concept studies, Long et al. , Nelson et al. , and Tabebordbar et al. used adeno-associated virus-9 to deliver the CRISPR/Cas9 gene-editing system to young mice with a mutation in the gene coding for dystrophin, a muscle protein deficient in patients with Duchenne muscular dystrophy. Gene editing partially restored dystrophin protein expression in skeletal and cardiac muscle and improved skeletal muscle function. Science , this issue p. 400 , p. 403 , p. 407
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Smaller genome editing proteins, such as S. aureus Cas9, C. jejuni Cas9 and newly identified CRISPR-Cas enzymes, may circumvent the AAV cargo limit.
“Smaller genome editing proteins, such as the S. aureus Cas9, C. jejuni Cas9 and newly identified CRISPR-Cas enzymes, may circumvent this issue 23,61-65 .”
From The promise and challenge of therapeutic genome editing (2020) · cites this paper as [63]
The passage on AAV delivery (section labeled Abstract; page not provided) describes S. aureus Cas9 as a smaller protein that can be packaged in AAV, supporting that example. It does not address C. jejuni Cas9 or newly identified CRISPR-Cas enzymes, so it does not support the full list in the citing sentence. (LLM, conf=0.85)
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A New Class of Medicines through DNA Editing
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AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
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In vivo genome editing improves motor function and extends survival in a mouse model of ALS
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Self-citationThe Ethics of Germline Gene Editing
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- Authors
- Tabebordbar, M., Zhu, K., Cheng, J. K. W., Chew, W. L., Widrick, J. J., Yan, W. X., Maesner, C., Wu, E. Y., Xiao, R., Ran, F. A., Cong, L., Zhang, F., Vandenberghe, L. H., Church, G. M., & Wagers, A. J.
- Affiliations
- Harvard University (US)
- Boston Children's Hospital (US)
- Broad Institute (US)
- Massachusetts Eye and Ear Infirmary (US)
- Corresponding author
- Tabebordbar, Mohammadsharif
- Zhu, Kexian
- Wagers, Amy J.
- ORCID
- Venue
- Science
- Published
- 2016-01-22
- Volume
- 351
- Issue
- 6271
- Pages
- 407-411
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- PMC
- PMC4924477
- OpenAlex
- W2203460689
- Cited by
- 1,046
- References
- 55
- Citations by year
- 2026: 36 · 2025: 50 · 2024: 42 · 2023: 63 · 2022: 80 · 2021: 110 · 2020: 97 · 2019: 125 · 2018: 147 · 2017: 164 · 2016: 130 · 2015: 2
- FWCI
- 64.15
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Digital Access to Scholarship at Harvard (DASH) (Harvard University) (acceptedVersion)
- Funders
- Howard Hughes Medical Institute
- National Institute of General Medical Sciences (NIGMS) (T2GM007753)
- NIH (1DP2OD004345, 5U01HL100402, 5PN2EY018244)
- New York Stem Cell Foundation
- National Institute of Mental Health (5DP1-MH100706)
- National Institute of Diabetes and Digestive and Kidney Diseases (5R01DK097768-03)
- NSF
- Keck
- New York Stem Cell
- Damon Runyon
- Searle Scholars
- Merkin
- Vallee Foundation
- B. Metcalfe
- Agency for Science, Technology, and Research (A*STAR), Singapore
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Muscle Physiology and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- Duchenne muscular dystrophy, dystrophin, CRISPR/Cas9, AAV gene delivery, in vivo gene editing, mdx mice, muscle stem cells, cardiomyocytes, exon deletion
- MeSH terms
- Dependovirus, Animals, Disease Models, Animal, Exons, Myocardium, RNA, Messenger, Transduction, Genetic, Genetic Therapy, Frameshift Mutation, Sequence Deletion, Mice, Inbred mdx, Muscle, Skeletal, Muscular Dystrophy, Duchenne, Satellite Cells, Skeletal Muscle, Mice, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems
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