The promise and challenge of therapeutic genome editing.

Authors

Jennifer A Doudna

Published in

Nature. Volume 578. Issue 7794. Pages 229-236. Epub Feb 12, 2020.

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
Bibliographic data and abstract were imported from PubMed on 28 Sep 2026.

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