Evidence map›Paper›PMID 39759878›Full record

ArticleMolecular therapy. Nucleic acids2025

Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective.

Joshua E Hung, Reid A Brewer, Lujaina Elbakr, Antonio Mollica, Georgiana Forguson, Wing Suen Chan, Evgueni A Ivakine

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 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. 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

7 authors.

Joshua E HungGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Reid A BrewerGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Lujaina ElbakrGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Antonio MollicaGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Georgiana ForgusonGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Wing Suen ChanGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.
Evgueni A IvakineGenetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON M5G0A4, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Tay-Sachs disease is a fatal neurodegenerative disorder caused by

Indexed as

CRISPR-Cas9 therapeutic editingframe correction therapyGM2 gangliosidosisHEXAmicrohomology-mediated end joiningMT: RNA/DNA Editingmutation correctionPAM-relaxed targetingTay-Sachs disease

Identifiers

PMID39759878
PMCPMC11699244

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.