Evidence map›Paper›PMID 32532987›Full record

ArticleNature communications2020

CRISPR artificial splicing factors.

Menghan Du, Nathaniel Jillette, Jacqueline Jufen Zhu, Sheng Li, Albert Wu Cheng

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 80 papers.

0numbers the graph read from it
0cells of the map it votes in
80citing papers in PubMed
5.6field-weighted citation impact, top 3% 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

80 citing papers in PubMed, 118 citations in OpenAlex.

  1. RNA splicing in health and disease.Molecular biomedicine · 2026
    Review
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  18. Recent advances in therapeutic gene-editing technologies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  19. Review
  20. Article

20 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 2 institutions in 1 country.

Menghan Du *The Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA.
Nathaniel Jillette *The Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA.
Jacqueline Jufen ZhuThe Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA.
Sheng LiThe Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA.ORCID http://orcid.org/0000-0002-9543-6274
Albert Wu ChengThe Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA. albert.cheng@jax.org.ORCID http://orcid.org/0000-0003-2653-5743
Jackson Laboratory · USUniversity of Connecticut · US

Funding

Shared Resource ManagementP30CA034196 · NCI · JACKSON LABORATORY · PI William Carl Skarnes · 1985 to 2026
$61.9M
Modular Platform for Combinatorial Epigenome ManipulationR01HG009900 · NHGRI · JACKSON LABORATORY · PI ADAMS, MARK D · 2018 to 2022
$3.7M
NCI NIH HHS P30 CA034196NHGRI NIH HHS R01 HG009900
6 · The paper itself

Abstract

Alternative splicing allows expression of mRNA isoforms from a single gene, expanding the diversity of the proteome. Its prevalence in normal biological and disease processes warrant precise tools for modulation. Here we report the engineering of CRISPR Artificial Splicing Factors (CASFx) based on RNA-targeting CRISPR-Cas systems. We show that simultaneous exon inclusion and exclusion can be induced at distinct targets by differential positioning of CASFx. We also create inducible CASFx (iCASFx) using the FKBP-FRB chemical-inducible dimerization domain, allowing small molecule control of alternative splicing. Finally, we demonstrate the activation of SMN2 exon 7 splicing in spinal muscular atrophy (SMA) patient fibroblasts, suggesting a potential application of the CASFx system.

Indexed as

Alternative SplicingBase SequenceCell LineCell Line, TumorCRISPR-Cas SystemsExonsFibroblastsHEK293 CellsHeLa CellsHumansMuscular Atrophy, SpinalRNARNA IsoformsRNA Splicing FactorsSurvival of Motor Neuron 2 ProteinRNARNA IsoformsRNA Splicing FactorsSMN2 protein, humanSurvival of Motor Neuron 2 Protein

Identifiers

PMID32532987
PMCPMC7293279
OpenAlexW3035188284

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.