Evidence map›Paper›PMID 40501755›Full record

ArticlebioRxiv : the preprint server for biology2025

Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease.

Matthew P Stratton, Jessica L Centa, Vicki J Swier, Wanda L Pfeifer, Clarissa D Booth, Karlee Albert, John L Hunyara, Mitchell J Rechtzigel, Fox J Duelli, Hannah G Leppert and 6 more

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 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

16 authors.

Matthew P StrattonCenter for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.ORCID 0000-0002-4426-8442
Jessica L CentaCenter for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.ORCID 0000-0003-3900-6568
Vicki J SwierPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.ORCID 0000-0001-7509-9920
Wanda L PfeiferDepartment of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.ORCID 0000-0002-5700-2678
Clarissa D BoothPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.
Karlee AlbertPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.
John L HunyaraIonis Pharmaceuticals, Carlsbad, CA, 92010, USA.ORCID 0000-0002-7885-6525
Mitchell J RechtzigelPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.ORCID 0000-0002-5298-7391
Fox J DuelliDepartment of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA.
Hannah G LeppertPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.ORCID 0000-0002-5331-8499
Frank RigoIonis Pharmaceuticals, Carlsbad, CA, 92010, USA.ORCID 0000-0001-6467-3309
Trisha SmitExemplar Genetics, Coralville, IA 52241, USA.
Paymaan Jafar-NejadIonis Pharmaceuticals, Carlsbad, CA, 92010, USA.ORCID 0000-0001-5176-739X
Jill M WeimerPediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.ORCID 0000-0003-2504-4942
Arlene V DrackDepartment of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.ORCID 0000-0002-8195-8426
Michelle L HastingsCenter for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.ORCID 0000-0002-4253-9261

Funding

Reading Frame Correction for the Treatment of Batten DiseaseR01NS113233 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI HASTINGS, MICHELLE L, WEIMER, JILL M · 2020 to 2023
$2.6M
LTQ Orbitrap Velos Mass Spectrometer with ETDS10OD010662 · OD · ROSALIND FRANKLIN UNIV OF MEDICINE & SCI · PI GLUCKSMAN, MARC J · 2012 to 2012
$600k
NIH HHS S10 OD010662NINDS NIH HHS R01 NS113233
6 · The paper itself

Abstract

CLN3 Batten disease is a lethal pediatric autosomal recessive neurodegenerative disease caused by mutations in the

Identifiers

PMID40501755
PMCPMC12154811

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.