Evidence map›Paper›PMID 40014475›Full record

ArticleHuman gene therapy2025

Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1.

Avani V Mylvara, Cristin D Davidson, Derek Alexander, Charles P Venditti, Forbes D Porter

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2025. 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. 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

5 authors.

Avani V MylvaraEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, Maryland, USA.ORCID 0000-0002-8741-4008
Cristin D DavidsonEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, Maryland, USA.ORCID 0000-0002-5508-8113
Derek AlexanderEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, Maryland, USA.ORCID 0009-0009-6578-2537
Charles P VendittiNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, Maryland, USA.ORCID 0000-0001-6599-1253
Forbes D PorterEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, Maryland, USA.ORCID 0000-0001-9397-0046

Funding

Basic and Translational Studies of Inborn Errors of MetabolismZIAHD008988 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI PORTER, FORBES · 2020 to 2025
$12.7M
Intramural NIH HHS ZIA HD008988
6 · The paper itself

Abstract

Niemann-Pick disease, type C1 (NPC1), is a rare, fatal neurodegenerative disorder caused by pathological variations in

Indexed as

Antibodies, NeutralizingAntibodies, ViralDependovirusNiemann-Pick Disease, Type CAdolescentAdultChildChild, PreschoolFemaleGenetic TherapyGenetic VectorsHumansInfantMaleNiemann-Pick C1 ProteinYoung AdultAntibodies, NeutralizingAntibodies, ViralNiemann-Pick C1 ProteinAAV2AAV9neurodegenerative diseaseneutralizing antibodiesNiemann-Pick disease type C1seroprevalence

Identifiers

PMID40014475
PMCPMC12042659

What Socratic 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 Socratic graph.