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Nature. 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.

Doudna JA1,2,3,4,5,6,7.

Author information

1
Department of Molecular and Cell Biology, University of California Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
2
Department of Chemistry, University of California Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
3
California Institute for Quantitative Biosciences (QB3), University of California Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
4
Innovative Genomics Institute, University of California Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
5
Howard Hughes Medical Institute, University of California Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
6
MBIB Division, Lawrence Berkeley National Laboratory, Berkeley, CA, USA. doudna@berkeley.edu.
7
Gladstone Institutes, University of California San Francisco, San Francisco, CA, USA. doudna@berkeley.edu.

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.

PMID:
32051598
DOI:
10.1038/s41586-020-1978-5

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