Evidence map›Paper›PMID 40940846›Full record

ArticleCancers2025

Repair of Mutated

André Leier, Xu Han, Jehanne Aghzadi, Erik Westin, Jian Liu, Tatiana T Marquez Lago, Robert A Kesterson, Bruce R Korf, Deeann Wallis, Ulrich F Müller

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cancers, 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

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.

André LeierDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.ORCID 0000-0002-2647-2693
Xu HanDepartment of Chemistry & Biochemistry, University of California San Diego, La Jolla, CA 92093, USA.
Jehanne AghzadiDepartment of Chemistry & Biochemistry, University of California San Diego, La Jolla, CA 92093, USA.ORCID 0009-0001-4694-0917
Erik WestinDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Jian LiuDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Tatiana T Marquez LagoDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Robert A KestersonDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Bruce R KorfDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Deeann WallisDepartment of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.ORCID 0000-0002-8217-0892
Ulrich F MüllerDepartment of Chemistry & Biochemistry, University of California San Diego, La Jolla, CA 92093, USA.ORCID 0000-0002-7473-538X

Funding

Gilbert Gene Therapy Initiative (GGTI) LLC 563635
6 · The paper itself

Abstract

BACKGROUND/

objectivesTherapeutic strategies for Neurofibromatosis Type I (NF1) that correct the underlying pathogenic

methodsSplice sites on the

resultsThe correct trans-splicing product of this ribozyme was validated in HEK293

conclusionsThis study established a splice site and activity-enhancing extended guide sequences for the repair of NF1 mRNA. Further optimization of the ribozyme, as well as improved delivery methods, may establish ribozyme-based RNA repair as a viable strategy for NF1 treatment.

Indexed as

extended guide sequencegroup I intronhNF1mNf1Neurofibromatosis Type IribozymeTetrahymena thermophilatrans-splicing

Identifiers

PMID40940846
PMCPMC12427287

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.