Evidence map›Paper›PMID 38895471›Full record

ArticlebioRxiv : the preprint server for biology2024

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice.

Avani V Mylvara, Alana L Gibson, Tansy Gu, Cristin D Davidson, Art A Incao, Katerina Melnyk, Dominick Pierre-Jacques, Stephanie M Cologna, Charles P Venditti, Forbes D Porter and 1 more

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 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, MD.ORCID 0000-0002-8741-4008
Alana L GibsonNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0003-2247-7064
Tansy GuNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0002-0653-0892
Cristin D DavidsonEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0002-5508-8113
Art A IncaoNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0001-6801-4562
Katerina MelnykEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0001-9167-5801
Dominick Pierre-JacquesUniversity of Illinois Chicago, Chicago, IL.ORCID 0009-0009-1272-0607
Stephanie M ColognaUniversity of Illinois Chicago, Chicago, IL.ORCID 0000-0002-3541-3361
Charles P VendittiNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.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, MD.ORCID 0000-0001-9397-0046
William J PavanNational Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.ORCID 0000-0001-8281-5120

Funding

Investigations of Methylmalonic Acidemia and Related DisordersZIAHG200318 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI VENDITTI, CHARLES P · 2009 to 2025
$29.7M
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
THE USE OF THE MOUSE NPC MODEL TO CLONE THE HUMAN DISEASE GENEZIAHG000068 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI PAVAN, WILLIAM J · 2009 to 2023
$6.0M
Novel Therapeutic Approaches for NPC DiseaseR01NS124784 · NINDS · UNIVERSITY OF ILLINOIS AT CHICAGO · PI Vinay Aakalu, Stephanie M Cologna · 2022 to 2026
$2.9M
Development of autophagy modulators for evaluation as a therapeutic strategy for Niemann-Pick Type CR01NS114413 · NINDS · UNIVERSITY OF ILLINOIS AT CHICAGO · PI COLOGNA, STEPHANIE M · 2020 to 2024
$2.0M
Intramural NIH HHS ZIA HD008988Intramural NIH HHS ZIA HG000068NINDS NIH HHS R01 NS114413NINDS NIH HHS R01 NS124784
6 · The paper itself

Abstract

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

Identifiers

PMID38895471
PMCPMC11185674

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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.