Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing.

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 Rev

journal_title

Physiological reviews

authors

Zhang Y,Long C,Bassel-Duby R,Olson EN

doi

10.1152/physrev.00046.2017

subject

Has Abstract

pub_date

2018-07-01 00:00:00

pages

1205-1240

issue

3

eissn

0031-9333

issn

1522-1210

journal_volume

98

pub_type

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