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The promise and challenge of therapeutic genome editing.
Nature. 2020 02; 578(7794):229-236.Nat

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.

Authors+Show Affiliations

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

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

32051598

Citation

Doudna, Jennifer A.. "The Promise and Challenge of Therapeutic Genome Editing." Nature, vol. 578, no. 7794, 2020, pp. 229-236.
Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229-236.
Doudna, J. A. (2020). The promise and challenge of therapeutic genome editing. Nature, 578(7794), 229-236. https://doi.org/10.1038/s41586-020-1978-5
Doudna JA. The Promise and Challenge of Therapeutic Genome Editing. Nature. 2020;578(7794):229-236. PubMed PMID: 32051598.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - The promise and challenge of therapeutic genome editing. A1 - Doudna,Jennifer A, Y1 - 2020/02/12/ PY - 2019/02/10/received PY - 2019/11/20/accepted PY - 2020/2/14/entrez PY - 2020/2/14/pubmed PY - 2020/4/21/medline SP - 229 EP - 236 JF - Nature JO - Nature VL - 578 IS - 7794 N2 - 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. SN - 1476-4687 UR - https://www.unboundmedicine.com/medline/citation/32051598/The_promise_and_challenge_of_therapeutic_genome_editing_ L2 - https://doi.org/10.1038/s41586-020-1978-5 DB - PRIME DP - Unbound Medicine ER -