Evidence map›Paper›PMID 40425312›Full record

ArticleRNA (New York, N.Y.)2025

OpenASO: RNA Rescue-designing splice-modulating antisense oligonucleotides through community science.

Victor Tse, Martin Guiterrez, Jill Townley, Jonathan Romano, Jennifer Pearl, Guillermo Chacaltana, Eterna Players, Rhiju Das, Jeremy R Sanford, Michael D Stone

Abstract read
In one paragraph

Article in RNA (New York, N.Y.), 2025. 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

5 · Who and what money

Authors and funding

10 authors.

Victor TseDepartment of Molecular, Cell and Developmental Biology, University of California Santa Cruz, Santa Cruz, California 95064, USA.ORCID 0009-0001-2072-591X
Martin GuiterrezDepartment of Molecular, Cell and Developmental Biology, University of California Santa Cruz, Santa Cruz, California 95064, USA.
Jill TownleyEterna Massive Open Laboratory.ORCID 0000-0001-8528-2227
Jonathan RomanoEterna Massive Open Laboratory.ORCID 0000-0003-4031-0102
Jennifer PearlEterna Massive Open Laboratory.ORCID 0000-0002-6971-4998
Guillermo ChacaltanaCenter for Molecular Biology of RNA, University of California Santa Cruz, Santa Cruz, California 95064, USA.
Eterna PlayersEterna Massive Open Laboratory.ORCID 0000-0002-7508-6705
Rhiju DasEterna Massive Open Laboratory.ORCID 0000-0001-7497-0972
Jeremy R SanfordDepartment of Molecular, Cell and Developmental Biology, University of California Santa Cruz, Santa Cruz, California 95064, USA mds@ucsc.edu jsanfor2@ucsc.edu.ORCID 0000-0002-1131-1627
Michael D StoneCenter for Molecular Biology of RNA, University of California Santa Cruz, Santa Cruz, California 95064, USA mds@ucsc.edu jsanfor2@ucsc.edu.ORCID 0000-0002-2060-4501

Funding

Next-generation computational/chemical methods for complex RNA structuresR35GM122579 · NIGMS · STANFORD UNIVERSITY · PI Rhiju Das · 2017 to 2026
$7.2M
Structure and dynamics of the Tetrahymena telomerase ribonucleoproteinR01GM095850 · NIGMS · UNIVERSITY OF CALIFORNIA SANTA CRUZ · PI STONE, MICHAEL D · 2011 to 2023
$4.4M
Regulation of mRNA FateR35GM130361 · NIGMS · UNIVERSITY OF CALIFORNIA SANTA CRUZ · PI Jeremy Robert Sanford · 2019 to 2026
$3.1M
Structure and Dynamics of Telomeres and TelomeraseR35GM153235 · NIGMS · UNIVERSITY OF CALIFORNIA SANTA CRUZ · PI Michael D Stone · 2024 to 2026
$1.3M
NIGMS NIH HHS R01 GM095850NIGMS NIH HHS R35 GM122579NIGMS NIH HHS R35 GM130361NIGMS NIH HHS R35 GM153235
6 · The paper itself

Abstract

Splice-modulating antisense oligonucleotides (ASOs) are precision RNA-based drugs that are becoming an established modality to treat human disease. Previously, we reported the discovery of ASOs that target a novel, putative intronic RNA structure to rescue splicing of multiple pathogenic variants of

Indexed as

Hemophilia AOligonucleotides, AntisenseRNARNA SplicingCrowdsourcingExonsFactor VIIIHumansIntronsNucleic Acid ConformationFactor VIIIOligonucleotides, AntisenseRNAantisense oligonucleotidescommunity sciencedrug discoveryhemophilia ARNA splicingRNA structure

Identifiers

PMID40425312
PMCPMC12265936

What Socratic holds

Textmetadata
LicenceTDM
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 Socratic graph.