Evidence map›Paper›PMID 41770218›Full record

ArticleFuture science OA2026

Preclinical assessment of GNE

Fabienne Kerneis, Christopher M Jay, Donald Rao, Jeremy Winchester, Alexander Nemunaitis, Emily Nemunaitis, Ernest Bognar, Gladice Wallraven, Laura Stanbery, Adam Walter and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Future science OA, 2026. 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

12 authors.

Fabienne KerneisGradalis, Inc, Dallas, TX, USA.
Christopher M JayGradalis, Inc, Dallas, TX, USA.
Donald RaoGradalis, Inc, Dallas, TX, USA.
Jeremy WinchesterGradalis, Inc, Dallas, TX, USA.
Alexander NemunaitisTaylor Family Cancer Research Center, Maumee, OH, USA.
Emily NemunaitisTaylor Family Cancer Research Center, Maumee, OH, USA.
Ernest BognarGradalis, Inc, Dallas, TX, USA.
Gladice WallravenGradalis, Inc, Dallas, TX, USA.
Laura StanberyGradalis, Inc, Dallas, TX, USA.
Adam WalterGradalis, Inc, Dallas, TX, USA.
Sep SarsharSolve GNE, Los Angeles, CA, USA.
John NemunaitisGradalis, Inc, Dallas, TX, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

aimsGNE myopathy is a heredity disease of unmet medical need associated with progressive skeletal muscle wasting, atrophy and weakness caused by mutations in the GNE gene. GNE plays a pivotal role in sialic acid production. Sialic acid is a critical part of glycoprotein, ganglioside and glycolipid cell-cell interaction which is necessary for normal skeletal muscle function. Previously we demonstrated safety and efficacy of the GNEwt gene lipoplex in one patient.

methodsWe engineered GNE

resultsWe demonstrate effective plasmid function via knockdown of the GNE

conclusionThese results encourage future studies, potentially leading toward clinical testing of GNE

Indexed as

bi-shRNAGNEGNE M743TmyopathyPlasmid

Identifiers

PMID41770218
PMCPMC12962615

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.