Evidence map›Paper›PMID 40330856›Full record

ArticlebioRxiv : the preprint server for biology2025

Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidence.

Terence Gall-Duncan, Sangyoon Y Ko, Isabelle K Quick, Mahreen Khan, Kristie Feng, Chase P Kelley, Annabelle Coleman, Alexiane Touze, Shuqian Tang, Mustafa Mehkary and 15 more

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Review
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

25 authors.

Terence Gall-DuncanGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Sangyoon Y KoNeuroscience & Mental Health, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Isabelle K QuickGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Mahreen KhanGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Kristie FengGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Chase P KelleyDepartment of Molecular Genetics & Microbiology, Center for NeuroGenetics, Genetics Institute, University of Florida, Gainesville, FL 32608, USA.
Annabelle ColemanUniversity College of London (UCL) Huntington's Disease Centre, UCL Queen Square Institute of Neurology, Queen Square, London, WC1N 3BG2.
Alexiane TouzeUniversity College of London (UCL) Huntington's Disease Centre, UCL Queen Square Institute of Neurology, Queen Square, London, WC1N 3BG2.
Shuqian TangGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Mustafa MehkaryGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Katsuyuki YokoiGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Casey R HerringtonGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Justin YouGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Scott C LambieGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Tanya K PrasolavaGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Gagan B PanigrahiGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Jeehye ParkGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Kazuhiko NakataniDepartment of Regulatory Bioorganic Chemistry, The Institute of Scientific and Industrial Research (SANKEN), Osaka University, 8-1 Mihogaoka, Ibaraki 567-0047, Japan.
Lauren M ByrneUniversity College of London (UCL) Huntington's Disease Centre, UCL Queen Square Institute of Neurology, Queen Square, London, WC1N 3BG2.
Peixiang WangGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
John S SchneeklothChemical Biology Laboratory, Center for Cancer Research, National Cancer Institute, Frederick, MD, USA.
Masayuki NakamoriDepartment of Neurology and Clinical Neuroscience, Yamaguchi University Graduate School of Medicine, Ube 755-8505, Japan.
Paul W FranklandNeuroscience & Mental Health, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.
Eric T WangDepartment of Molecular Genetics & Microbiology, Center for NeuroGenetics, Genetics Institute, University of Florida, Gainesville, FL 32608, USA.
Christopher E PearsonGenetics & Genome Biology, The Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.ORCID 0000-0001-9545-4205

Funding

Impeding transcription of expanded microsatellite repeats using deactivated Cas9R01AG058636 · NIA · UNIVERSITY OF FLORIDA · PI WANG, ERIC T · 2019 to 2023
$1.9M
NIA NIH HHS R01 AG058636
6 · The paper itself

Abstract

Huntington disease (HD) is caused by inherited CAG expansions, which continue expanding somatically in affected brain regions to hasten disease onset and progression. Therapeutically diminishing somatic expansions is expected to be clinically beneficial. However, it is not known if interventionally modifying somatic CAG expansions will actually modify

Identifiers

PMID40330856
PMCPMC12051495

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.