Evidence map›Paper›PMID 39556729›Full record

ArticleProceedings of the National Academy of Sciences of the United States of America2024

In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice.

Samuel W Du, Gregory A Newby, David Salom, Fangyuan Gao, Carolline Rodrigues Menezes, Susie Suh, Elliot H Choi, Paul Z Chen, David R Liu, Krzysztof Palczewski

Abstract read
In one paragraph

Article in Proceedings of the National Academy of Sciences of the United States of America, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

17 citing papers in PubMed.

  1. Article
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  5. Article
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  8. International journal of biological sciences · 2026
    Review
  9. Article
  10. Article
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  12. Article
  13. Review
  14. Review
  15. Molecular therapy. Nucleic acids · 2025
    Review
  16. Review
  17. In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice.Proceedings of the National Academy of Sciences of the United States of America · 2024
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Samuel W DuGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.ORCID 0000-0002-9655-3659
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142.
David SalomGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.
Fangyuan GaoGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.
Carolline Rodrigues MenezesGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.
Susie SuhGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.ORCID 0000-0002-4921-1429
Elliot H ChoiGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.
Paul Z ChenMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142.
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142.
Krzysztof PalczewskiGavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA 92617.ORCID 0000-0002-0788-545X

Funding

MEDICAL SCIENTIST TRAINING PROGRAMT32GM007250 · NIGMS · CASE WESTERN RESERVE UNIVERSITY · PI HUANG, ALEX YEE-CHEN · 1985 to 2023
$33.4M
Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
STRUCTURAL STUDIES OF ARRESTINSR01EY009339 · NEI · UNIVERSITY OF WASHINGTON · PI KISER, PHILIP DAVID, PALCZEWSKI, KRZYSZTOF · 1992 to 2025
$16.0M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM008620 · NIGMS · UNIVERSITY OF CALIFORNIA-IRVINE · PI GOLDIN, ALAN L · 1999 to 2023
$8.1M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
Precision genome editing in vivo to treat retinal diseasesR01EY034501 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Audrone Lapinaite, Krzysztof Palczewski · 2023 to 2026
$3.9M
NEI UCI Center Core Grant for Vision ResearchP30EY034070 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Rachel Wagner Martin · 2022 to 2026
$3.7M
Delivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3M
Expanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1M
Interdisciplinary Training Program in Skin BiologyT32AR080622 · NIAMS · UNIVERSITY OF CALIFORNIA-IRVINE · PI Bogi Andersen, Anand K Ganesan · 2022 to 2026
$909k
In vivo precision genome editing to correct genetic diseaseR00HL163805 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEWBY, GREGORY A. · 2023 to 2025
$747k
microRNA-204 and microRNA-211 regulation of RPE phagocytosisF30EY033642 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Samuel Wang Du · 2022 to 2026
$241k
HHS | NIH (NIH) F30EY029136 T32GM007250 T32AR080622HHS | NIH (NIH) R00HL163805HHS | NIH (NIH) R01EY009339 R01EY034501 P30EY034070HHS | NIH (NIH) T32GM008620 F30EY033642HHS | NIH (NIH) UG3AI150551 U01AI142756 R35GM118062 RM1HG009490NEI NIH HHS F30 EY029136NEI NIH HHS F30 EY033642NEI NIH HHS P30 EY034070NEI NIH HHS R01 EY009339NEI NIH HHS R01 EY034501NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS R00 HL163805NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIAMS NIH HHS T32 AR080622NIGMS NIH HHS R35 GM118062NIGMS NIH HHS T32 GM007250NIGMS NIH HHS T32 GM008620
6 · The paper itself

Abstract

Rhodopsin, the prototypical class-A G-protein coupled receptor, is a highly sensitive receptor for light that enables phototransduction in rod photoreceptors. Rhodopsin plays not only a sensory role but also a structural role as a major component of the rod outer segment disc, comprising over 90% of the protein content of the disc membrane. Mutations in

Indexed as

Gene EditingRetinitis PigmentosaRhodopsinAnimalsCRISPR-Cas SystemsDisease Models, AnimalMiceMutationRetinal Rod Photoreceptor CellsRhodopsinbase editingprime editingretinitis pigmentosarhodopsin

Identifiers

PMID39556729
PMCPMC11621631

What OpenQuestion holds

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LicenceCC BY
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.