Making gene editing a therapeutic reality [version 1; referees: 2 approved]

Irina Conboy; Niren Murthy; Jessy Etienne; Zachery Robinson

doi:10.12688/f1000research.16106.1

F1000Research (Dec 2018)

Making gene editing a therapeutic reality [version 1; referees: 2 approved]

Irina Conboy,
Niren Murthy,
Jessy Etienne,
Zachery Robinson

Affiliations

Irina Conboy: Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA
Niren Murthy: Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA
Jessy Etienne: Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA
Zachery Robinson: Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA

DOI: https://doi.org/10.12688/f1000research.16106.1
Journal volume & issue: Vol. 7

Abstract

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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.

Published in F1000Research

ISSN: 2046-1402 (Online)
Publisher: F1000 Research Ltd
Country of publisher: United Kingdom
LCC subjects: Medicine; Science
Website: https://f1000research.com

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