Evidence map›Paper›PMID 40672310›Full record

ArticlebioRxiv : the preprint server for biology2025

Preventing vision loss in a mouse model of Leber Congenital Amaurosis by engineered tRNA.

Enes Akyuz, Pawan K Shahi, Lionel Gissot, Ahmad Al Saneh, Divya Sinha, Giovanni M Hanstad, Meha Kabra, Maria A Fernandez Zepeda, David M Gamm, Samuel M Young and 2 more

Abstract readPreprint
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

12 authors.

Enes AkyuzUniversity of Wisconsin-Madison, Department of Pediatrics, Wisconsin, USA.
Pawan K ShahiUniversity of Wisconsin-Madison, Department of Pediatrics, Wisconsin, USA.
Lionel GissotUniversity of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics, Iowa, USA.
Ahmad Al SanehUniversity of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics, Iowa, USA.
Divya SinhaUniversity of Wisconsin-Madison, McPherson Eye Research Institute, Wisconsin, USA.
Giovanni M HanstadUniversity of Wisconsin-Madison, Waisman Center, Wisconsin, USA.
Meha KabraUniversity of Wisconsin-Madison, Department of Pediatrics, Wisconsin, USA.
Maria A Fernandez ZepedaUniversity of Wisconsin-Madison, Waisman Center, Wisconsin, USA.
David M GammUniversity of Wisconsin-Madison, McPherson Eye Research Institute, Wisconsin, USA.
Samuel M YoungGene Therapy Center, University of North Carolina-Chapel Hill, Chapel Hill, NC 27599, USA. Department of Pediatrics, Department of Pharmacology, University of North Carolina-Chapel Hill, Chapel Hill, NC 27599, USA.
Christopher A AhernUniversity of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics, Iowa, USA.
Bikash R PattnaikUniversity of Wisconsin-Madison, Department of Pediatrics, Wisconsin, USA.ORCID 0000-0002-5385-358X

Funding

UW Vision Research Core - Administrative CoreP30EY016665 · NEI · UNIVERSITY OF WISCONSIN-MADISON · PI AKIHIRO IKEDA · 2005 to 2026
$12.6M
Restoring Vision with High-Fidelity Nonsense Codon CorrectionR24EY032434 · NEI · UNIVERSITY OF WISCONSIN-MADISON · PI AHERN, CHRISTOPHER A, GAMM, DAVID M · 2021 to 2025
$7.8M
Enhancing Basic and Translational Vision Research Using AnimalsS10OD026957 · OD · UNIVERSITY OF WISCONSIN-MADISON · PI BRANDT, CURTIS R · 2019 to 2019
$529k
NEI NIH HHS P30 EY016665NEI NIH HHS R24 EY032434NIH HHS S10 OD026957
6 · The paper itself

Abstract

Premature termination codons (PTCs) are associated with rare genetic disorders. Inducing targeted read-through of these 'nonsense mutations' presents a potential therapeutic strategy for modifying disease outcomes. We previously reported that one such PTC, W53X, in the

Indexed as

Anticodon engineered tRNAelectrophysiologygene therapyiPSCKir7.1Leber Congenital AmaurosisRPE

Identifiers

PMID40672310
PMCPMC12265521

What Socratic 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 Socratic graph.