Evidence map›Paper›PMID 41536809›Full record

ArticleMolecular therapy. Nucleic acids2026

Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target

Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Chia-Fei Liu, Sihui Wu, Ida Maria Westin, Irina Golovleva, Guei-Sheung Liu, Satheesh Kumar, Samuel McLenachan, Fred Kuanfu Chen and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. mRNA Vaccines for Influenza: Hope for a Universal Vaccine?BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  3. Article
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

16 authors.

Roxanne Hsiang-Chi LiouCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Daniel Urrutia-CabreraCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Chia-Fei LiuCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Sihui WuCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Ida Maria WestinMedical and Clinical Genetics, Department of Medical Biosciences, Umeå University, Umeå, Sweden.
Irina GolovlevaMedical and Clinical Genetics, Department of Medical Biosciences, Umeå University, Umeå, Sweden.
Guei-Sheung LiuCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Satheesh KumarCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Samuel McLenachanCentre for Ophthalmology and Visual Science, University of Western Australia, Perth, Western Australia, Australia.
Fred Kuanfu ChenCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Fei-Ting HsuDepartment of Life Sciences, National Central University, Taoyuan City, Taiwan.
Chien-Ling HuangDepartment of Health Technology and Informatics, The Hong Kong Polytechnic University, Hong Kong, China.
Tom EdwardsCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Keith R MartinCentre for Eye Research Australia, Melbourne, Victoria, Australia.
Albert Wu ChengState Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, China.
Raymond C B WongCentre for Eye Research Australia, Melbourne, Victoria, Australia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Dysregulation of the alternative splicing process results in aberrant mRNA transcripts, leading to dysfunctional proteins or nonsense-mediated decay that cause a wide range of mis-splicing diseases. Development of therapeutic strategies to target the alternative splicing process could potentially shift the mRNA splicing from disease isoforms to a normal isoform and restore functional protein. As a proof of concept, we focus on Stargardt disease (STGD1), an autosomal recessive inherited retinal disease caused by biallelic genetic variants in the

Indexed as

cas13CRISPRengineered U1exonsMT: RNA/DNA Editingretinal degenerationRNA splicingstargardt disease

Identifiers

PMID41536809
PMCPMC12795662

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.