Evidence map›Paper›PMID 32833535›Full record

ReviewThe CRISPR journal2020

Translating CRISPR-Cas Therapeutics: Approaches and Challenges.

Lavina Sierra Tay, Nathan Palmer, Rebecca Panwala, Wei Leong Chew, Prashant Mali

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in The CRISPR journal, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
1.7field-weighted citation impact, top 16% 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

27 citing papers in PubMed, 40 citations in OpenAlex.

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  15. CRISPR-Cas Genome Editing inHuman gene therapy · 2024
    Article
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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 2 countries.

Lavina Sierra TayLaboratory of Synthetic Biology and Genome Editing Therapeutics, Genome Institute of Singapore, Agency for Science, Technology and Research, Singapore, Singapore.; University of California San Diego, La Jolla, California, USA.
Nathan PalmerDivision of Biological Sciences, University of California San Diego, La Jolla, California, USA.
Rebecca PanwalaDepartment of Bioengineering, University of California San Diego, La Jolla, California, USA.
Wei Leong ChewLaboratory of Synthetic Biology and Genome Editing Therapeutics, Genome Institute of Singapore, Agency for Science, Technology and Research, Singapore, Singapore.; University of California San Diego, La Jolla, California, USA.
Prashant MaliDepartment of Bioengineering, University of California San Diego, La Jolla, California, USA.
University of California San Diego · US

Funding

Next-generation Functional Genetic Screening of Un-screenable TraitsR01HG009285 · NHGRI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI MALI, PRASHANT, TAMAYO, PABLO · 2017 to 2020
$4.2M
Systematic mapping of genetic vulnerabilities in head and neck cancerR01CA222826 · NCI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI MALI, PRASHANT · 2018 to 2022
$1.8M
Dissecting the interplay of gene regulatory networks and cellular niche on cell fate determinationR01GM123313 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI MALI, PRASHANT · 2018 to 2021
$1.2M
NCI NIH HHS R01 CA222826NHGRI NIH HHS R01 HG009285NIGMS NIH HHS R01 GM123313
6 · The paper itself

Abstract

CRISPR-Cas clinical trials have begun, offering a first glimpse at how DNA and RNA targeting could enable therapies for many genetic and epigenetic human diseases. The speedy progress of CRISPR-Cas from discovery and adoption to clinical use is built on decades of traditional gene therapy research and belies the multiple challenges that could derail the successful translation of these new modalities. Here, we review how CRISPR-Cas therapeutics are translated from technological systems to therapeutic modalities, paying particular attention to the therapeutic cascade from cargo to delivery vector, manufacturing, administration, pipelines, safety, and therapeutic target profiles. We also explore potential solutions to some of the obstacles facing successful CRISPR-Cas translation. We hope to illuminate how CRISPR-Cas is brought from the academic bench toward use in the clinic.

Indexed as

CRISPR-Cas SystemsGene EditingClustered Regularly Interspaced Short Palindromic RepeatsGenetic TherapyHumans

Identifiers

PMID32833535
PMCPMC7469700
OpenAlexW3081375952

What OpenQuestion holds

Textmetadata
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.