Evidence map›Paper›PMID 42265079›Full record

ArticleSignal transduction and targeted therapy2026

Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis.

Pawan K Shahi, Enes Akyuz, Lionel Gissot, Ahmad Al Saneh, Sanjai K Pillala, Divya Sinha, Giovanni M Hanstad, Meha Kabra, Maria A Fernandez Zepeda, David M Gamm and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Signal transduction and targeted therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors.

Pawan K Shahi *University of Wisconsin-Madison, Department of Pediatrics, Madison, WI, USA.
Enes Akyuz *University of Wisconsin-Madison, Department of Pediatrics, Madison, WI, USA.
Lionel GissotUniversity of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics, Iowa City, IA, USA.
Ahmad Al SanehUniversity of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics, Iowa City, IA, USA.
Sanjai K PillalaUniversity of Wisconsin-Madison, Department of Pediatrics, Madison, WI, USA.
Divya SinhaUniversity of Wisconsin-Madison, McPherson Eye Research Institute, Madison, WI, USA.ORCID http://orcid.org/0000-0002-5854-0056
Giovanni M HanstadUniversity of Wisconsin-Madison, Waisman Center, Madison, WI, USA.
Meha KabraUniversity of Wisconsin-Madison, Department of Pediatrics, Madison, WI, USA.
Maria A Fernandez ZepedaUniversity of Wisconsin-Madison, Waisman Center, Madison, WI, USA.
David M GammUniversity of Wisconsin-Madison, McPherson Eye Research Institute, Madison, WI, USA.
Samuel M YoungCenter for Molecular Medicine, 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 City, IA, USA.ORCID http://orcid.org/0000-0002-7975-2744
Bikash R PattnaikUniversity of Wisconsin-Madison, Department of Pediatrics, Madison, WI, USA. pattnaik@wisc.edu.ORCID http://orcid.org/0000-0002-5385-358X

Funding

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
NEI NIH HHS R24 EY032434
6 · The paper itself

Abstract

Premature termination codons (PTCs) are a major class of pathogenic variants that underlie rare inherited disorders, including forms of childhood blindness. Therapeutic suppression of these "nonsense mutations" offers a gene- and position-agnostic strategy to restore protein function. Our previous work established that the W53X PTC in the KCNJ13 gene causes Leber congenital amaurosis type 16 (LCA16) by disrupting the inwardly rectifying potassium channel Kir7.1, leading to retinal pigment epithelium (RPE) dysfunction. Here, we present a proof-of-concept approach using anticodon-engineered transfer RNA (ACE-tRNA) to promote targeted translational readthrough. We engineered a suppressor tRNA encoding tryptophan (ACE-tRNA

Indexed as

Leber Congenital AmaurosisPotassium Channels, Inwardly RectifyingRNA, TransferAnimalsCodon, NonsenseDisease Models, AnimalHumansMiceRetinal Pigment EpitheliumCodon, NonsenseKir7.1 channelPotassium Channels, Inwardly RectifyingRNA, Transfer

Identifiers

PMID42265079
PMCPMC13250130

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.