Cited by 2,237CRISPR and Genetic EngineeringRNA regulation and diseaseVirus-based gene therapy research

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

Anzalone, Andrew V., Koblan, Luke W., Liu, David R.·2020·Nature Biotechnology

Abstract

The development of new CRISPR-Cas genome editing tools continues to drive major advances in the life sciences. Four classes of CRISPR-Cas-derived genome editing agents-nucleases, base editors, transposases/recombinases and prime editors-are currently available for modifying genomes in experimental systems. Some of these agents have also moved rapidly into the clinic. Each tool comes with its own capabilities and limitations, and major efforts have broadened their editing capabilities, expanded their targeting scope and improved editing specificity. We analyze key considerations when choosing genome editing agents and identify opportunities for future improvements and applications in basic research and therapeutics.

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Crossref · 10.1038/s41587-020-0561-9

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  • Advances in mRNA-based gene editing tools, including CRISPR-Cas9 and base editing platforms, provide an opportunity for one-and-done treatments for monogenic diseases.

    “Advances in mRNA-based gene editing tools, including CRISPR-Cas9 and base editing platforms, provide an opportunity for “one-and-done” treatments for monogenic diseases (5, 6).”

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Authors
Anzalone, A. V., Koblan, L. W., & Liu, D. R.
Affiliations
Broad Institute (US)
Corresponding author
Liu, David R.
Venue
Nature Biotechnology
Published
2020-06-22
Volume
38
Issue
7
Pages
824-844
ISSN
  • 0733-222X (ISSN-L)
  • 1087-0156
  • 1546-1696
OpenAlex
W3035764705
Cited by
2,237
References
51
Citations by year
2026: 267 · 2025: 316 · 2024: 414 · 2023: 412 · 2022: 412 · 2021: 326 · 2020: 90
FWCI
30.06
Type
journal article
Language
en
Open access
closed
Locations
  • Nature Biotechnology (publishedVersion)
  • PubMed (publishedVersion)
Funders
  • Division of Intramural Research, National Institute of Allergy and Infectious Diseases (U01AI142756)
  • U.S. Department of Health & Human Services | NIH | National Human Genome Research Institute (RM1HG009490)
  • U.S. Department of Health & Human Services | NIH | National Institute of Biomedical Imaging and Bioengineering (R01EB022376)
  • U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (R35GM118062)
  • Howard Hughes Medical Institute (Liu investigatorship)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords
CRISPR/Cas genome editing, CRISPR-Cas nucleases, base editors, transposase, recombinases, prime editors, genome editing tools, editing specificity, tool selection
MeSH terms
Gene Editing, Endonucleases, Humans, Genome, Transposases, Recombinases, DNA Breaks, Double-Stranded, CRISPR-Cas Systems

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