Evidence map›Paper›PMID 39823061›Full record

ArticleMolecular therapy. Methods & clinical development2024

Design and validation of cell-based potency assays for frataxin supplementation treatments.

Shibani Mukherjee, Larisa Pereboeva, Daniel Fil, Achisha Saikia, Jeon Lee, Jixue Li, M Grazia Cotticelli, Elisabetta Soragni, Robert B Wilson, Marek Napierala and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. 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. Review
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

11 authors.

Shibani MukherjeeDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, 6000 Harry Hines Boulevard, Dallas, TX 75390, USA.
Larisa PereboevaDepartment of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, 1825 University Boulevard, Birmingham, AL 35294, USA.
Daniel FilDepartment of Biochemistry and Molecular Biology, University of Arkansas for Medical Sciences, Little Rock, AR 72205, USA.
Achisha SaikiaDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, 6000 Harry Hines Boulevard, Dallas, TX 75390, USA.
Jeon LeeLyda Hill Department of Bioinformatics, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
Jixue LiDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, 6000 Harry Hines Boulevard, Dallas, TX 75390, USA.
M Grazia CotticelliDepartment of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Elisabetta SoragniFriedreich's Ataxia Research Alliance, 533 W. Uwchlan Avenue, Downingtown, PA 19335, USA.
Robert B WilsonDepartment of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Marek NapieralaDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, 6000 Harry Hines Boulevard, Dallas, TX 75390, USA.
Jill S NapieralaDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, 6000 Harry Hines Boulevard, Dallas, TX 75390, USA.

Funding

Defining the impact of Frataxin point mutations on Friedreich's ataxia pathogenesisR01NS121038 · NINDS · UT SOUTHWESTERN MEDICAL CENTER · PI NAPIERALA, MAREK · 2021 to 2025
$2.0M
Developing models to define the Frataxin G130V-specific pathogenic mechanism in Friedreich's ataxiaR03NS099953 · NINDS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI NAPIERALA, JILL SERGESKETTER · 2017 to 2018
$149k
NINDS NIH HHS R01 NS121038NINDS NIH HHS R03 NS099953
6 · The paper itself

Abstract

Friedreich's ataxia (FRDA) is a multisystem, autosomal recessive disorder caused by mutations in the frataxin (

Indexed as

aerobic glycolysisfrataxinFriedreich’s ataxiagene therapypotency assay

Identifiers

PMID39823061
PMCPMC11735916

What OpenQuestion holds

Textmetadata
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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.