TY - JOUR
T1 - Myoediting
T2 - Toward prevention of muscular dystrophy by therapeutic genome editing
AU - Zhang, Yu
AU - Long, Chengzu
AU - Bassel-Duby, Rhonda
AU - Olson, Eric N.
N1 - Funding Information:
This work was supported by Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center Grant U54 HD 087351 as well as Parent Project Muscular Dystrophy and Robert A. Welch Foundation Grant 1-0025 (to E. N. Olson).
Funding Information:
This work was supported by Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center Grant U54 HD 087351 as well as Parent Project Muscular Dystrophy and Robert A. Welch Foundation Grant 1– 0025 (to E. N. Olson).
Publisher Copyright:
© 2018 American Physiological Society. All rights reserved.
PY - 2018/7
Y1 - 2018/7
N2 - Muscular dystrophies represent a large group of genetic disorders that significantly impair quality of life and often progress to premature death. There is no effective treatment for these debilitating diseases. Most therapies, developed to date, focus on alleviating the symptoms or targeting the secondary effects, while the underlying gene mutation is still present in the human genome. The discovery and application of programmable nucleases for site-specific DNA doublestranded breaks provides a powerful tool for precise genome engineering. In particular, the CRISPR/Cas system has revolutionized the genome editing field and is providing a new path for disease treatment by targeting the disease-causing genetic mutations. In this review, we provide a historical overview of genome-editing technologies, summarize the most recent advances, and discuss potential strategies and challenges for permanently correcting genetic mutations that cause muscular dystrophies.
AB - Muscular dystrophies represent a large group of genetic disorders that significantly impair quality of life and often progress to premature death. There is no effective treatment for these debilitating diseases. Most therapies, developed to date, focus on alleviating the symptoms or targeting the secondary effects, while the underlying gene mutation is still present in the human genome. The discovery and application of programmable nucleases for site-specific DNA doublestranded breaks provides a powerful tool for precise genome engineering. In particular, the CRISPR/Cas system has revolutionized the genome editing field and is providing a new path for disease treatment by targeting the disease-causing genetic mutations. In this review, we provide a historical overview of genome-editing technologies, summarize the most recent advances, and discuss potential strategies and challenges for permanently correcting genetic mutations that cause muscular dystrophies.
UR - http://www.scopus.com/inward/record.url?scp=85046797549&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85046797549&partnerID=8YFLogxK
U2 - 10.1152/physrev.00046.2017
DO - 10.1152/physrev.00046.2017
M3 - Review article
C2 - 29717930
AN - SCOPUS:85046797549
SN - 0031-9333
VL - 98
SP - 1205
EP - 1240
JO - Physiological reviews
JF - Physiological reviews
IS - 3
ER -