Evidence map›Paper›PMID 31418127›Full record

ReviewCell biology and toxicology2020

Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications.

Emily M Mills, Victoria L Barlow, Louis Y P Luk, Yu-Hsuan Tsai

Abstract readReview
In one paragraph

Review in Cell biology and toxicology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
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.

Emily M MillsSchool of Chemistry, Cardiff University, Cardiff, CF10 3AT, UK.
Victoria L BarlowSchool of Chemistry, Cardiff University, Cardiff, CF10 3AT, UK.
Louis Y P LukSchool of Chemistry, Cardiff University, Cardiff, CF10 3AT, UK.
Yu-Hsuan TsaiSchool of Chemistry, Cardiff University, Cardiff, CF10 3AT, UK. tsaiy5@cardiff.ac.uk.ORCID 0000-0003-0589-5088

Funding

Biology and Biological Science Research Council BB/P009506/1Wellcome Trust 200730/Z/16/Z
6 · The paper itself

Abstract

Progress in targeted gene editing by programmable endonucleases has paved the way for their use in gene therapy. Particularly, Cas9 is an endonuclease with high activity and flexibility, rendering it an attractive option for therapeutic applications in clinical settings. Many disease-causing mutations could potentially be corrected by this versatile new technology. In addition, recently developed switchable Cas9 variants, whose activity can be controlled by an external stimulus, provide an extra level of spatiotemporal control on gene editing and are particularly desirable for certain applications. Here, we discuss the considerations and difficulties for implementing Cas9 to in vivo gene therapy. We put particular emphasis on how switchable Cas9 variants may resolve some of these barriers and advance gene therapy in the clinical setting.

Indexed as

CRISPR-Associated Protein 9CRISPR-Cas SystemsGene EditingGenetic TherapyHumansCRISPR-Associated Protein 9EndonucleaseGene therapyIn vivo gene editingSwitchable Cas9

Identifiers

PMID31418127
PMCPMC7051928

What OpenQuestion holds

Textmetadata
LicenceCC BY
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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.