Evidence map›Paper›PMID 33077937›Full record

ReviewNature reviews. Drug discovery2020

Base editing: advances and therapeutic opportunities.

Elizabeth M Porto, Alexis C Komor, Ian M Slaymaker, Gene W Yeo

Abstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 285 papers.

0numbers the graph read from it
0cells of the map it votes in
285citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

285 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Article
  5. The landscape and trajectory of global CRISPR therapeutics.Molecular therapy. Nucleic acids · 2026
    Review
  6. Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. In vivo base editing of Asgr1 reduces blood lipids in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  12. Review
  13. Precision modification of heart failure signaling by CRISPR-Cas9 base editing.Journal of molecular and cellular cardiology · 2026
    Review
  14. Review
  15. Article
  16. Review
  17. Review
  18. Article
  19. Review
  20. Article

225 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Elizabeth M Porto *Department of Chemistry and Biochemistry, University of California, San Diego, La Jolla, CA, USA.
Alexis C Komor *Department of Chemistry and Biochemistry, University of California, San Diego, La Jolla, CA, USA. akomor@ucsd.edu.ORCID http://orcid.org/0000-0001-5043-0998
Ian M SlaymakerSynthetic Biology Department, Beam Therapeutics, Cambridge, MA, USA.
Gene W YeoDepartment of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA, USA.ORCID http://orcid.org/0000-0002-0799-6037

Funding

MOLECULAR BIOPHYSICS TRAINING PROGRAMT32GM008326 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI KOMIVES, ELIZABETH A. · 1989 to 2020
$7.5M
Therapeutic strategies for microsatellite expansion diseases using RNA-targeting CRISPR/CasR01NS103172 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI MAURICE SCOTT SWANSON, Eugene Wei-Ming Yeo · 2017 to 2026
$5.6M
Application of RNA-targeting Cas9 to Fuchs' dystrophyR01EY029166 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI AFSHARI, NATALIE ADEL, YEO, EUGENE WEI-MING · 2018 to 2021
$1.4M
Development of New Genome Editing Agents Using RNA Modifying EnzymesR21GM135736 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI KOMOR, ALEXIS C. · 2019 to 2019
$205k
NEI NIH HHS R01 EY029166NIGMS NIH HHS R21 GM135736NIGMS NIH HHS T32 GM008326NINDS NIH HHS R01 NS103172
6 · The paper itself

Abstract

Base editing - the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells - is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and RNA base editors are revealing exciting therapeutic opportunities for these technologies. We expect the correction of single point mutations will be a major focus of future precision medicine.

Indexed as

CRISPR-Cas SystemsGene EditingDiseaseHearing LossHumansLiver DiseasesMuscular Dystrophy, DuchenneNeoplasmsPolymorphism, Single Nucleotide

Identifiers

PMID33077937
PMCPMC7721651

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.