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Review
. 2020 Feb;578(7794):229-236.
doi: 10.1038/s41586-020-1978-5. Epub 2020 Feb 12.

The promise and challenge of therapeutic genome editing

Jennifer A Doudna  1   2   3   4   5   6   7
Affiliations
Review

The promise and challenge of therapeutic genome editing

Jennifer A Doudna. Nature. 2020 Feb.

Abstract

Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is-or will soon be-in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.

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See this image and copyright information in PMC

References

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      *This study showed how CRISPR-Cas9 uses a dual RNA guide to bind and cut DNA, and described the composition of Cas9 with single-guide RNAs that can direct DNA nicking or cutting in ways suitable for genome editing. The results provided a road map for labs exploring mechanisms of genome editing to deploy CRISPR-Cas9 in this capacity.

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