Genome Editing: Insights from Chemical Biology to Support Safe and Transformative Therapeutic Applications

Programmable nuclease-based genome editing technologies, including the clustered, regularly interspaced, short palindromic repeats (CRISPR)/Cas9 system, are becoming an essential component of many applications ranging from agriculture to medicine. However, fundamental limitations currently prevent t...

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Bibliographic Details
Published in:	ACS chemical biology Vol. 13; no. 2; pp. 333 - 342
Main Authors:	Wegrzyn, Renee D, Lee, Andrew H, Jenkins, Amy L, Stoddard, Colby D, Cheever, Anne E
Format:	Journal Article
Language:	English
Published:	United States American Chemical Society 16-02-2018
Subjects:	Animals Biology - ethics Biology - methods CRISPR-Associated Proteins - metabolism CRISPR-Cas Systems - genetics DNA - genetics DNA Breaks, Double-Stranded DNA End-Joining Repair - genetics Endonucleases - metabolism Gene Editing - ethics Gene Editing - methods Gene Transfer Techniques Genome Humans RNA, Guide, CRISPR-Cas Systems - genetics
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Summary:	Programmable nuclease-based genome editing technologies, including the clustered, regularly interspaced, short palindromic repeats (CRISPR)/Cas9 system, are becoming an essential component of many applications ranging from agriculture to medicine. However, fundamental limitations currently prevent the widespread, safe, and practical use of genome editors, especially for human disease interventions. These limitations include off-target effects, a lack of control over editing activity, suboptimal DNA repair outcomes, insufficient target conversion, and inadequate delivery performance. This perspective focuses on the potential for biological chemistry to address these limitations such that newly developed genome editing technologies can enable the broadest range of potential future applications. Equally important will be the development of these powerful technologies within a relevant ethical framework that emphasizes safety and responsible innovation.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	1554-8929 1554-8937
DOI:	10.1021/acschembio.7b00689