Evidence map›Paper›PMID 41210588›Full record

ArticleMolecular therapy. Nucleic acids2025

Prime editing for the investigation of aberrant splicing defect associated with a pathogenic

Bruna Lopes da Costa, Kyle M Helms, Keith Theodore, Yi-Ting Tsai, Salvatore Marco Caruso, Siyuan Liu, Jose Ronaldo Lima de Carvalho, Nicholas D Nolan, Saleha Tahir, Christopher D Makinson and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Variant-to-gene mapping identifiesbioRxiv : the preprint server for biology · 2026
    Article
  3. Review
  4. 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

12 authors.

Bruna Lopes da CostaJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Kyle M HelmsDepartment of Neurology, Columbia University Irving Medical Center, New York, NY 10032, USA.
Keith TheodoreFM Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Yi-Ting TsaiJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Salvatore Marco CarusoJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Siyuan LiuJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Jose Ronaldo Lima de CarvalhoFM Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Nicholas D NolanJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Saleha TahirFM Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Christopher D MakinsonDepartment of Neurology, Columbia University Irving Medical Center, New York, NY 10032, USA.
Stephen H TsangJonas Children's Vision Care (JCVC) and Barbara & Donald Jonas Stem Cell Laboratory, New York-Presbyterian Hospital, New York, NY 10032, USA.
Peter M J QuinnFM Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.

Funding

Prime editing for Crumbs homologue 1 (CRB1) Inherited Retinal DystrophiesR01EY034952 · NEI · UNIVERSITY OF PENNSYLVANIA · PI Peter Martin John Quinn · 2023 to 2026
$1.6M
NEI NIH HHS R01 EY034952
6 · The paper itself

Abstract

The human Peripherin 2 (

Indexed as

hiPSCMT: RNA/DNA Editingprime editingPRPH2 c.828+1G>A splice site variantPRPH2-mediated IRDsretinal organoidsretinitis pigmentosa

Identifiers

PMID41210588
PMCPMC12594906

What OpenQuestion holds

Textmetadata
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.