Evidence map›Paper›PMID 40518673›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.

David D Fuller, Sabhya Rana, Prajwal P Thakre, Ethan S Benevides, Megan K Pope, Adrian G Todd, Victoria N Jensen, Lauren Vaught, Denise A Cloutier, Roberto A Ribas and 7 more

Erratum issuedAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. 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. Mass Spectrometry-Based Spatial Imaging of the Cochlea.Journal of the American Society for Mass Spectrometry · 2026
    Article
  2. Article
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

17 authors.

David D FullerDepartment of Physical Therapy, University of Florida, Gainesville, FL, USA; Breathing Research and Therapeutics Center, University of Florida, Gainesville, FL, USA; McKnight Brain Institute, University of Florida, Gainesville, FL, USA. Electronic address: ddf@phhp.ufl.edu.
Sabhya RanaDepartment of Physical Therapy, University of Florida, Gainesville, FL, USA; Breathing Research and Therapeutics Center, University of Florida, Gainesville, FL, USA; McKnight Brain Institute, University of Florida, Gainesville, FL, USA.
Prajwal P ThakreDepartment of Physical Therapy, University of Florida, Gainesville, FL, USA; Breathing Research and Therapeutics Center, University of Florida, Gainesville, FL, USA; McKnight Brain Institute, University of Florida, Gainesville, FL, USA.
Ethan S BenevidesBreathing Research and Therapeutics Center, University of Florida, Gainesville, FL, USA; McKnight Brain Institute, University of Florida, Gainesville, FL, USA; Department of Pediatrics, University of Florida, Gainesville, FL, USA.
Megan K PopeDepartment of Pediatrics, University of Florida, Gainesville, FL, USA.
Adrian G ToddDepartment of Pediatrics, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Victoria N JensenDepartment of Physical Therapy, University of Florida, Gainesville, FL, USA.
Lauren VaughtDepartment of Pediatrics, University of Florida, Gainesville, FL, USA.
Denise A CloutierDepartment of Pediatrics, University of Florida, Gainesville, FL, USA.
Roberto A RibasDepartment of Biochemistry & Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, USA.
Reece C LarsonDepartment of Biochemistry & Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, USA.
Matthew S GentryDepartment of Biochemistry & Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, USA.
Ramon C SunMcKnight Brain Institute, University of Florida, Gainesville, FL, USA; Department of Biochemistry & Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, USA.
Vijay ChandranDepartment of Pediatrics, University of Florida, Gainesville, FL, USA.
Manuela CortiDepartment of Pediatrics, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Darin J FalkDepartment of Pediatrics, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Barry J ByrneDepartment of Pediatrics, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.

Funding

Brain Glycogen-Metabolism,Mechanisms, and Therapeutic PotentialR35NS116824 · NINDS · UNIVERSITY OF KENTUCKY · PI Matthew S. Gentry · 2020 to 2026
$8.2M
Control of Breathing & Glycogen Storage DiseaseR01HD052682 · NICHD · UNIVERSITY OF FLORIDA · PI BYRNE, BARRY J, FULLER, DAVID D · 2007 to 2024
$6.4M
Brain Glucose Deficiency: Mechanisms and ModulationRM1NS133593 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI Joseph J. Pancrazio, Juan M. Pascual · 2023 to 2026
$5.3M
Deciphering the Glycan Code in Human Alzheimer’s Disease BrainR01AG078702 · NIA · UNIVERSITY OF KENTUCKY · PI Peggi M Angel, Sean Curtis Bendall · 2022 to 2026
$3.8M
Aberrant Glycogen in Lung Adenocarcinoma TumorigenesisR01CA266004 · NCI · UNIVERSITY OF KENTUCKY · PI Matthew S. Gentry, Ramon C. Sun · 2022 to 2026
$2.6M
Aberrant Glycogen Modulates Cerebral Glucose Metabolism in Aging and Alzheimer's DiseaseR01AG066653 · NIA · UNIVERSITY OF KENTUCKY · PI SUN, RAMON C. · 2020 to 2024
$1.9M
Pathogenesis of the Neuromuscular Synapse in Pompe DiseaseK01AR066077 · NIAMS · UNIVERSITY OF FLORIDA · PI FALK, DARIN J · 2014 to 2017
$439k
NCI NIH HHS R01 CA266004NIAMS NIH HHS K01 AR066077NIA NIH HHS R01 AG066653NIA NIH HHS R01 AG078702NICHD NIH HHS R01 HD052682NINDS NIH HHS R35 NS116824NINDS NIH HHS RM1 NS133593
6 · The paper itself

Abstract

Absence of functional acid-α-glucosidase (GAA) leads to early onset Pompe disease with cardiorespiratory and neuromuscular failure. A novel Pompe rat model (Gaa

Indexed as

alpha-GlucosidasesGenetic TherapyGlycogen Storage Disease Type IIAnimalsAnimals, NewbornDependovirusDiaphragmDisease Models, AnimalGenetic VectorsHumansRatsalpha-GlucosidasesAAVdiaphragmneonatalPomperespiratoryspinal

Identifiers

PMID40518673
PMCPMC12432874

What OpenQuestion holds

Textmetadata
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.