Evidence map›Paper›PMID 41958974›Full record

ArticlebioRxiv : the preprint server for biology2026

Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing.

Elena M Kahn, Hooda Said, Ping Qu, Mohamad-Gabriel Alameh, Xiao Wang, Kiran Musunuru, Rebecca C Ahrens-Nicklas

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2026. 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

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.

Elena M KahnCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Hooda SaidRaymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Ping QuCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Mohamad-Gabriel AlamehDepartment of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Xiao WangCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Kiran MusunuruCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Rebecca C Ahrens-NicklasMetabolic Disease Program, Division of Human Genetics, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

Funding

Replication study of in vivo postnatal gene editing for metabolic liver disease in the nonhuman primate modelU19NS132301 · NINDS · CHILDREN'S HOSP OF PHILADELPHIA · PI Kiran Musunuru, William H. Peranteau · 2023 to 2026
$48.5M
Personalized prime editing as a platform for hepatic inborn errors of metabolismU01TR005355 · NCATS · CHILDREN'S HOSP OF PHILADELPHIA · PI Rebecca Clare Ahrens-Nicklas, Kiran Musunuru · 2024 to 2026
$10.8M
NCATS NIH HHS U01 TR005355NINDS NIH HHS U19 NS132301
6 · The paper itself

Abstract

Methylmalonic acidemia (MMA) is a recessive genetic disease caused by variants in the

Identifiers

PMID41958974
PMCPMC13060892

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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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.