Evidence map›Paper›PMID 40243436›Full record

ArticleInternational journal of molecular sciences2025

Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel.

Kelly Godbout, Mathieu Dugas, Steven R Reiken, Sina Ramezani, Alexia Falle, Joël Rousseau, Anetta E Wronska, Gabriel Lamothe, Geoffrey Canet, Yaoyao Lu and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of molecular sciences, 2025. 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. Review
  3. Phosphorylation of RYR1 at SerScience signaling · 2025
    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

13 authors.

Kelly GodboutMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.ORCID 0000-0002-7567-7978
Mathieu DugasMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.
Steven R ReikenDepartment of Physiology and Cellular Biophysics, Center for Molecular Cardiology, Columbia University Vagelos College of Physicians and Surgeons, New York, NY 10032, USA.ORCID 0000-0002-5807-3574
Sina RamezaniMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.
Alexia FalleMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.
Joël RousseauCHU de Québec Research Center-Laval University, Quebec, QC G1V 4G2, Canada.
Anetta E WronskaDepartment of Physiology and Cellular Biophysics, Center for Molecular Cardiology, Columbia University Vagelos College of Physicians and Surgeons, New York, NY 10032, USA.
Gabriel LamotheMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.ORCID 0000-0003-4468-7775
Geoffrey CanetMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.ORCID 0000-0001-7291-4512
Yaoyao LuMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.
Emmanuel PlanelMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.
Andrew R MarksDepartment of Physiology and Cellular Biophysics, Center for Molecular Cardiology, Columbia University Vagelos College of Physicians and Surgeons, New York, NY 10032, USA.
Jacques P TremblayMolecular Medicine Department, Laval University, Quebec, QC G1V 0A6, Canada.ORCID 0000-0001-9404-9195

Funding

RYR-1 Foundation 130780
6 · The paper itself

Abstract

RyR1-related myopathies (RyR1-RMs) include a wide range of genetic disorders that result from mutations in the

Indexed as

Gene EditingGenetic TherapyMuscular DiseasesMutationRyanodine Receptor Calcium Release ChannelCalciumHEK293 CellsHumansMyoblastsCalciumRyanodine Receptor Calcium Release ChannelRYR1 protein, humancalcium channelcalcium leakCRISPR/Cas9prime editingprotective mutationRYR1 geneRyR1-RM

Identifiers

PMID40243436
PMCPMC11988564

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.