OSCR

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

Overview

Authors: Pawan K. Shahi1,2, Enes Akyuz1,2, Lionel Gissot3, Ahmad Al Saneh3, Sanjai K. Pillala1,2, Divya Sinha2,4, Giovanni M. Hanstad4, Meha Kabra1,2, Maria A. Fernandez Zepeda4, David M. Gamm2,4,5, Samuel M. Young6,7, Christopher A. Ahern3, Bikash R. Pattnaik1,2,5
ORCID iDs: Enes Akyuz, Ahmad Al Saneh, Divya Sinha, Meha Kabra, Christopher A. Ahern, Bikash R. Pattnaik
  1. University of Wisconsin-Madison, Department of Pediatrics,Madison, WI USA
  2. University of Wisconsin-Madison, McPherson Eye Research Institute,Madison, WI USA
  3. University of Iowa, Carver College of Medicine, Department of Molecular Physiology and Biophysics,Iowa City, IA USA
  4. University of Wisconsin-Madison, Waisman Center,Madison, WI USA
  5. University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences,Madison, WI USA
  6. Center for Molecular Medicine, University of North Carolina-Chapel Hill,Chapel Hill, NC 27599 USA
  7. Department of Pediatrics, Department of Pharmacology, Department of Microbiology and Immunology, University of North Carolina-Chapel Hill,Chapel Hill, NC 27599 USA
Institutions: University of Wisconsin–Madison (United States); University of Iowa (United States); University of North Carolina at Chapel Hill (United States)
Journal: Signal transduction and targeted therapy, volume 11, issue 1, article 225
Dates: received 21 November 2025; accepted 11 May 2026; published online 9 June 2026
Type: Research article · Language: English
License: CC BY-NC-ND
Identifiers: DOI 10.1038/s41392-026-02793-3 · PMID 42265079 · PMCID PMC13250130 · OpenAlex W7164049965
Open access: gold, a free copy (OpenAlex)
Status: data only
Categories: human (organism), mouse (organism)
Methods: Statistics, Evoked potentials, Physiology & signal measures
Keywords: Nucleic-acid therapeutics, Gene therapy, Molecular medicine, Diseases of the nervous system
MeSH: Leber Congenital Amaurosis*, Potassium Channels, Inwardly Rectifying*, RNA, Transfer*, Animals, Codon, Nonsense, Disease Models, Animal, Humans, Mice, Retinal Pigment Epithelium (* major topic)
Topic: Retinal Development and Disorders (Molecular Biology, Biochemistry, Genetics and Molecular Biology), according to OpenAlex
Funding: U.S. Department of Health & Human Services | NIH | National Eye Institute (NEI) (R24 EY032434)
Citations: not cited yet (Europe PMC); 57 references in the paper

Abstract

The abstract is not reproduced here: the paper's license (CC BY-NC-ND) does not allow it. Read it in the paper, at the publisher or on Europe PMC.

Code

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Data

Datasets cited

Data availability statement

The paper has a data availability statement. Its license (CC BY-NC-ND) does not allow reproducing it here; in short, from what the harvester recognized in it:

  • it points to a dataset: figshare 32190930
  • it says that the data are available on request

Read it in the paper: doi.org/10.1038/s41392-026-02793-3.

Versions

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Version 1, 27 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 13 authors, 4 keywords, 9 MeSH terms, 1 funder, 57 references.

Cite

This paper

Shahi, P. K., Akyuz, E., Gissot, L., Al Saneh, A., Pillala, S. K., Sinha, D., Hanstad, G. M., Kabra, M., Fernandez Zepeda, M. A., Gamm, D. M., Young, S. M., Ahern, C. A., & Pattnaik, B. R. (2026). Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis. Signal transduction and targeted therapy, 11(1), 225. https://doi.org/10.1038/s41392-026-02793-3

BibTeX

@article{shahi2026engineered,
author = {Shahi, Pawan K. and Akyuz, Enes and Gissot, Lionel and Al Saneh, Ahmad and Pillala, Sanjai K. and Sinha, Divya and Hanstad, Giovanni M. and Kabra, Meha and Fernandez Zepeda, Maria A. and Gamm, David M. and Young, Samuel M. and Ahern, Christopher A. and Pattnaik, Bikash R.},
title = {{Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis}},
journal = {Signal transduction and targeted therapy},
year = {2026},
month = jun,
volume = {11},
number = {1},
pages = {225},
publisher = {Nature Publishing Group},
issn = {2095-9907},
doi = {10.1038/s41392-026-02793-3},
url = {https://doi.org/10.1038/s41392-026-02793-3},
pmid = {42265079},
pmcid = {PMC13250130}
}

RIS

TY - JOUR
AU - Shahi, Pawan K.
AU - Akyuz, Enes
AU - Gissot, Lionel
AU - Al Saneh, Ahmad
AU - Pillala, Sanjai K.
AU - Sinha, Divya
AU - Hanstad, Giovanni M.
AU - Kabra, Meha
AU - Fernandez Zepeda, Maria A.
AU - Gamm, David M.
AU - Young, Samuel M.
AU - Ahern, Christopher A.
AU - Pattnaik, Bikash R.
TI - Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis
T2 - Signal transduction and targeted therapy
J2 - Signal Transduct Target Ther
PY - 2026
DA - 2026/06/09
VL - 11
IS - 1
SP - 225
SN - 2095-9907
PB - Nature Publishing Group
DO - 10.1038/s41392-026-02793-3
UR - https://doi.org/10.1038/s41392-026-02793-3
LA - en
ER -

CSL-JSON

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