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Cited by 1,336CRISPR and Genetic EngineeringRNA regulation and diseaseVirus-based gene therapy research

Therapeutic genome editing: prospects and challenges

Open access
Cox, David Benjamin Turitz, Platt, Randall Jeffrey, Zhang, Feng·2015·Nature Medicine
DOI 10.1038/nm.3793PMC PMC4492683
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Abstract

Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease-based therapies as well as future prospects and challenges.

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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).”

    From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [5]

    The Abstract describes CRISPR-Cas9 editing as a potential treatment for monogenic disorders, and the Delivery section discusses mRNA delivery of editing nucleases. Our copy does not discuss base-editing platforms or establish that treatment would require only one administration; those parts of the claim are unsupported. (LLM, conf=0.86)

    Experimental — accuracy not yet measured

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Cited by

Sorted by citations

Authors
Cox, D. B. T., Platt, R. J., & Zhang, F.
Affiliations
Broad Institute (US)
Corresponding author
Zhang, Feng
ORCID
Venue
Nature Medicine
Publisher
Springer Science and Business Media LLC
Published
2015-02
Volume
21
Issue
2
Pages
121-131
ISSN
  • 1078-8956 (ISSN-L)
  • 1546-170X
DOI
10.1038/nm.3793
PMC
PMC4492683
OpenAlex
W1996788258
Cited by
1,336
References
23
Citations by year
2026: 47 · 2025: 93 · 2024: 102 · 2023: 126 · 2022: 122 · 2021: 120 · 2020: 148 · 2019: 138 · 2018: 138 · 2017: 136 · 2016: 115 · 2015: 49 · 2014: 1 · 2013: 1
FWCI
14.15
Type
journal article
Language
en
Open access
Licence
www.springer.com/tdm (tdm)
Locations
  • Nature Medicine (publishedVersion)
  • PubMed (publishedVersion)
  • Europe PMC (PubMed Central) (acceptedVersion)
  • PubMed Central (acceptedVersion)
  • DSpace@MIT (Massachusetts Institute of Technology) (submittedVersion, cc-by-nc-sa)
  • PMC
Funders
  • National Institute of Mental Health (DP1MH100706)
  • National Institute of General Medical Sciences (T32GM007753)
  • National Institute of Neurological Disorders and Stroke (R01NS073124)
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
genome editing, programmable nucleases, gene therapy, genetic disorders, disease modeling, animal models, mutation correction, therapeutic genome editing
MeSH terms
Deoxyribonucleases, Endodeoxyribonucleases, Genetic Engineering, Humans, Genetic Therapy, Genome

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