Article

Making gene editing a therapeutic reality

2018-12-21

Abstract excerpt

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...

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Literature Corpus work: 0e7c80d1-4b66-59da-8ea2-0eaa1105619d
DOI: 10.12688/f1000research.16106.1

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