Evidence map›Paper›PMID 41716008›Full record

ArticleACS chemical biology2026

Modified Polycyclic Compounds Rescue Mis-splicing in Myotonic Dystrophy Type 1 Disease Models.

Jesus A Frias, Sawyer M Hicks, Hormoz Mazdiyasni, Subodh K Mishra, Kahini Sarkar, Clara Yeboah, Noah M LeFever, Marina M Scotti, Hana Zeghal, Naomi Brandt and 13 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in ACS chemical biology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

23 authors.

Jesus A FriasThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Sawyer M HicksThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Hormoz MazdiyasniThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Subodh K MishraThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Kahini SarkarThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Clara YeboahThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Noah M LeFeverThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Marina M ScottiDepartment of Molecular Genetics & Microbiology and Center for Neurogenetics, College of Medicine, University of Florida, Gainesville, Florida 32610, United States.
Hana ZeghalThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Naomi BrandtThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Sweta VangavetiThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Pramita ChakmaThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Ting WangThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.ORCID 0000-0001-7630-4148
Tammy S ReidThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
Omari McMichaelDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia 23284, United States.
Christopher CrumbaughDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia 23284, United States.
Marina ProvenzanoDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia 23284, United States.
Melissa A HaleDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia 23284, United States.
John D ClearyThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.ORCID 0000-0001-7599-5995
Nicholas E JohnsonDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia 23284, United States.
Eric T WangDepartment of Molecular Genetics & Microbiology and Center for Neurogenetics, College of Medicine, University of Florida, Gainesville, Florida 32610, United States.
Kaalak ReddyThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.
J Andrew BerglundThe RNA Institute, College of Arts and Sciences, University at Albany, State University of New York, Albany, New York 12222, United States.ORCID 0000-0002-5198-2724

Funding

Senator Paul D. Wellstone Muscular Dystrophy Specialized Research CenterP50NS048843 · NINDS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2018 to 2022
$7.1M
Identifying the RNA Splicing and Gene Expression Changes that Cause Congenital Myotonic Dystrophy (Renewal)R01NS104010 · NINDS · VIRGINIA COMMONWEALTH UNIVERSITY · PI Nicholas Elwood Johnson · 2018 to 2026
$4.4M
Training CoreP50AR085906 · NIAMS · VIRGINIA COMMONWEALTH UNIVERSITY · PI Nicholas Elwood Johnson · 2025 to 2026
$4.1M
Trial Readiness and Endpoint Assessment in LGMDR1 (TREATing-LGMDR1)U01NS124974 · NINDS · VIRGINIA COMMONWEALTH UNIVERSITY · PI Nicholas Elwood Johnson · 2023 to 2026
$4.0M
Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic DystrophyR01NS120485 · NINDS · STATE UNIVERSITY OF NEW YORK AT ALBANY · PI Andrew Berglund, Masayuki Nakamori · 2022 to 2026
$2.7M
RNA Science and Technology in Health and DiseaseT32GM132066 · NIGMS · STATE UNIVERSITY OF NEW YORK AT ALBANY · PI Thomas J Begley · 2019 to 2026
$1.8M
FDA HHS R01 FD006071NIAMS NIH HHS P50 AR085906NIGMS NIH HHS T32 GM132066NIH HHS R01NS104010NINDS NIH HHS P50 NS048843NINDS NIH HHS R01 NS104010NINDS NIH HHS R01 NS120485NINDS NIH HHS U01 NS124974
6 · The paper itself

Abstract

Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder with no approved therapeutics targeting the disease mechanism. DM1 is caused by the expression of expanded CUG repeat RNA (CUG

Indexed as

Alternative SplicingMyotonic DystrophyPolycyclic CompoundsAnimalsDisease Models, AnimalHumansMiceRNA-Binding ProteinsRNA SplicingPolycyclic CompoundsRNA-Binding Proteins

Identifiers

PMID41716008
PMCPMC13010256

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.