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Scientific Articles

Making Gene Editing a Therapeutic Reality

key information

source: F1000Research

year: 2019

authors: Conboy I, Murthy N, Etienne J, Robinson Z


This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9.

Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.

organization: UC Berkeley, USA

DOI: 10.12688/f1000research.16106.1

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