Abstract:
: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 double-stranded 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.
journal_name
Physiol Revjournal_title
Physiological reviewsauthors
Zhang Y,Long C,Bassel-Duby R,Olson ENdoi
10.1152/physrev.00046.2017subject
Has Abstractpub_date
2018-07-01 00:00:00pages
1205-1240issue
3eissn
0031-9333issn
1522-1210journal_volume
98pub_type
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