Evidence map›Paper›PMID 40673060›Full record

ArticleMolecular therapy. Methods & clinical development2025

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.

Katelyn Daman, Jing Yan, Annabelle Biscans, Dimas Echeverria, Taisia Shmushkovich, Alexey Wolfson, Julia F Alterman, Anastasia Khvorova, Charles P Emerson

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 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. 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

9 authors.

Katelyn DamanWellstone Muscular Dystrophy Program, Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Jing YanWellstone Muscular Dystrophy Program, Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Annabelle BiscansRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Dimas EcheverriaRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Taisia ShmushkovichAdvirna, 17 Briden St, Worcester, MA 01605, USA.
Alexey WolfsonAdvirna, 17 Briden St, Worcester, MA 01605, USA.
Julia F AltermanRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Anastasia KhvorovaRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Charles P EmersonWellstone Muscular Dystrophy Program, Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.

Funding

Training Core [Parent Title: NOVEL THERAPEUTICS FOR FSHD]P50HD060848 · NICHD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI EMERSON, CHARLES P. · 2018 to 2023
$8.6M
NICHD NIH HHS P50 HD060848
6 · The paper itself

Abstract

Facioscapulohumeral muscular dystrophy (FSHD) is the third most diagnosed muscular dystrophy. The disease is caused by genetic and epigenetic disruptions that result in misexpression of the germline transcription factor DUX4 in skeletal muscle, leading to muscle toxicity and turnover. As a gene misexpressed exclusively in muscle,

Indexed as

FSHDlipid-conjugated siRNAmuscular dystrophysiRNAxenograft

Identifiers

PMID40673060
PMCPMC12264612

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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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.