Evidence map›Paper›PMID 36186952›Full record

ArticleMolecular therapy. Methods & clinical development2022

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9.

Randy J Chandler, Giovanni Di Pasquale, Jennifer L Sloan, Samantha McCoy, Brandon T Hubbard, Tina M Kilts, Irini Manoli, John A Chiorini, Charles P Venditti

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing 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

10 citing papers in PubMed.

  1. Article
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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

9 authors.

Randy J ChandlerOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
Giovanni Di PasqualeNational Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
Jennifer L SloanOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
Samantha McCoyOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
Brandon T HubbardOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
Tina M KiltsNational Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
Irini ManoliOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
John A ChioriniNational Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
Charles P VendittiOrganic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.

Funding

Investigations of Methylmalonic Acidemia and Related DisordersZIAHG200318 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI VENDITTI, CHARLES P · 2009 to 2025
$29.7M
Adeno-associated Virus Biology And Utilization For Gene TransferZ01DE000695 · NIDCR · NATIONAL INSTITUTE OF DENTAL & CRANIOFACIAL RESEARCH · PI CHIORINI, JOHN A · 2000 to 2008
$3.4M
Intramural NIH HHS Z01 DE000695Intramural NIH HHS ZIA HG200318
6 · The paper itself

Abstract

Methylmalonic acidemia (MMA) is a severe and potentially lethal autosomal recessive inborn error of metabolism most frequently caused by mutations in the methylmalonyl-CoA mutase (

Indexed as

AAV 44.9liver directed gene therapymethylmalonic acidemiaMMAneutralizing antibodiessystemic gene therapy

Identifiers

PMID36186952
PMCPMC9490190

What OpenQuestion holds

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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.