Evidence map›Paper›PMID 32207531›Full record

ReviewBioscience reports2020

Gene editing and CRISPR in the clinic: current and future perspectives.

Matthew P Hirakawa, Raga Krishnakumar, Jerilyn A Timlin, James P Carney, Kimberly S Butler

Open access · goldAbstract readReview
In one paragraph

Review in Bioscience reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 97 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
97citing papers in PubMed, 1 pooled it
10.9field-weighted citation impact, top 1% of its field
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

97 citing papers in PubMed, 1 synthesis or guideline pooled it, 186 citations in OpenAlex.

  1. Pooled it
  2. Article
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  6. Article
  7. Review
  8. Review
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  10. Article
  11. Article
  12. CRISPR-Cas Systems: A Functional Perspective and Innovations.International journal of molecular sciences · 2025
    Review
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  14. Review
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37 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

5 authors at 1 institution in 1 country.

Matthew P HirakawaSystems Biology, Sandia National Laboratories, Livermore, CA 94551, U.S.A.
Raga KrishnakumarSystems Biology, Sandia National Laboratories, Livermore, CA 94551, U.S.A.
Jerilyn A TimlinMolecular and Microbiology, Sandia National Laboratories, Albuquerque, NM 87185, U.S.A.
James P CarneyAdvanced Materials Laboratory, Sandia National Laboratories, Albuquerque, NM 87185, U.S.A.
Kimberly S ButlerMolecular and Microbiology, Sandia National Laboratories, Albuquerque, NM 87185, U.S.A.
Sandia National Laboratories California · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly interspaced short palindromic repeat DNA sequences)/Cas9 are rapidly progressing into clinical trials. Most clinical use of CRISPR to date has focused on ex vivo gene editing of cells followed by their re-introduction back into the patient. The ex vivo editing approach is highly effective for many disease states, including cancers and sickle cell disease, but ideally genome editing would also be applied to diseases which require cell modification in vivo. However, in vivo use of CRISPR technologies can be confounded by problems such as off-target editing, inefficient or off-target delivery, and stimulation of counterproductive immune responses. Current research addressing these issues may provide new opportunities for use of CRISPR in the clinical space. In this review, we examine the current status and scientific basis of clinical trials featuring ZFNs, TALENs, and CRISPR-based genome editing, the known limitations of CRISPR use in humans, and the rapidly developing CRISPR engineering space that should lay the groundwork for further translation to clinical application.

Indexed as

AnimalsCell Culture TechniquesClinical Trials as TopicCRISPR-Cas SystemsDrug Delivery SystemsGene EditingGenetic TherapyGenetic VectorsHumansImmunotherapy, AdoptiveMiceModels, AnimalNanoparticlesReceptors, Chimeric AntigenTranscription Activator-Like Effector NucleasesTranslational Research, BiomedicalReceptors, Chimeric AntigenTranscription Activator-Like Effector NucleasesZinc Finger Nucleasesclinical trialCRISPRgene activationgenome editingtranscription activator-like effector nucleaseszinc finger nuclease

Identifiers

PMID32207531
PMCPMC7146048
OpenAlexW3012901598

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.