Evidence map›Paper›PMID 40880469›Full record

ArticleScience advances2025

Intravenous AAV9-

Lalitha Venkataraman, Christopher J Jeanty, Tarun Kaniganti, Benjamin J Kennedy, Patricia Sullivan, David S Goldstein, Stephen G Kaler

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Science advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Article
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

7 authors.

Lalitha VenkataramanCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.ORCID 0000-0002-3649-9996
Christopher J JeantyCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.ORCID 0000-0001-9848-9956
Tarun KanigantiCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.ORCID 0009-0008-7617-1662
Benjamin J KennedyCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Patricia SullivanAutonomic Medicine Section, Clinical Neurosciences Program, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD 20892, USA.ORCID 0009-0000-8627-8270
David S GoldsteinAutonomic Medicine Section, Clinical Neurosciences Program, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-5709-9940
Stephen G KalerCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.ORCID 0000-0002-7522-926X

Funding

Biomarkers of Parkinson Disease and Related DisordersZIANS003034 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI GOLDSTEIN, DAVID · 2009 to 2025
$22.8M
Viral Gene Therapy for Menkes DiseaseU01NS129343 · NINDS · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI KALER, STEPHEN GERARD · 2023 to 2025
$3.8M
Intramural NIH HHS ZIA NS003034NINDS NIH HHS U01 NS129343
6 · The paper itself

Abstract

Menkes disease is an X-linked recessive condition characterized by seizures, failure to thrive, and rapid, progressive neurodegeneration beginning within weeks after birth. Death usually occurs by 3 years of age. The disorder is caused by genetic variants in

Indexed as

CopperCopper-Transporting ATPasesDependovirusGenetic TherapyHistidineMenkes Kinky Hair SyndromeAnimalsBrainDisease Models, AnimalGenetic VectorsHumansInjections, SubcutaneousMaleMiceOrganometallic CompoundsATP7A protein, humanAtp7a protein, mouseCoppercopper histidineCopper-Transporting ATPasesHistidineOrganometallic Compounds

Identifiers

PMID40880469
PMCPMC12396322

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.