Evidence map›Paper›PMID 39944742›Full record

ArticleBrain communications2025

Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia.

Judith Cossins, Imre Kozma, Claudia Canzonetta, Al Hawkins, David Beeson, Patricio Sepulveda, Yin Yao Dong

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Brain communications, 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. Review
  2. Review
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors.

Judith CossinsNeurosciences Group, Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DS, UK.ORCID https://orcid.org/0000-0003-2323-622X
Imre KozmaNeurosciences Group, Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DS, UK.
Claudia CanzonettaAmplo Biotechnology, La Jolla, CA 92037, USA.
Al HawkinsAmplo Biotechnology, La Jolla, CA 92037, USA.
David BeesonNeurosciences Group, Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DS, UK.ORCID https://orcid.org/0000-0001-9395-7132
Patricio SepulvedaAmplo Biotechnology, La Jolla, CA 92037, USA.
Yin Yao DongNeurosciences Group, Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DS, UK.ORCID https://orcid.org/0000-0001-8600-6988

Funding

Targeted DOK7 gene therapy for Congenital Myasthenic SyndromesR44NS124351 · NINDS · AMPLO BIOTECHNOLOGY, INC. · PI SEPULVEDA, PATRICIO · 2021 to 2024
$4.9M
NINDS NIH HHS R44 NS124351
6 · The paper itself

Abstract

Congenital myasthenic syndromes are a group of inherited disorders characterized by defective neuromuscular transmission and fatigable muscle weakness. Causative mutations have been identified in over 30 genes, including

Indexed as

congenital myastheniaDOK7-AAVgene therapyneuromuscular junctionpre-clinical trial

Identifiers

PMID39944742
PMCPMC11814498

What OpenQuestion holds

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Registered trials

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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.