Evidence map›Paper›PMID 40654892›Full record

ArticlebioRxiv : the preprint server for biology2025

A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models.

Jenny Gao, Nathan Bamidele, Debora Pires-Ferreira, April Destefano, Qiushi Tang, Yueying Cao, Jun Xie, Guangping Gao, Alisha Gruntman, Erik Sontheimer and 2 more

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2025. 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

12 authors.

Jenny GaoRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Nathan BamideleRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Debora Pires-FerreiraHorae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, USA.
April DestefanoHorae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Qiushi TangHorae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Yueying CaoRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Jun XieHorae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID 0000-0001-9051-5531
Guangping GaoLi Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Alisha GruntmanHorae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Erik SontheimerRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Terence R FlotteLi Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Wen XueRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID 0000-0002-0881-0310

Funding

Vector Immunology CoreP01HL158506 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Wen Xue · 2021 to 2026
$19.5M
Develop combinatorial non-viral and viral CRISPR delivery for lung diseasesUH3HL147367 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANDERSON, DANIEL G, GAO, GUANGPING · 2021 to 2022
$2.9M
In vivo prime editing for precision cancer mouse modelsR01CA275945 · NCI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Wen Xue · 2023 to 2026
$2.5M
Developing a novel adenine base editor tool for in vivo alpha-1 antitrypsin deficiency gene correction.F30HL176024 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Jenny Gao · 2024 to 2026
$103k
NCI NIH HHS R01 CA275945NHLBI NIH HHS F30 HL176024NHLBI NIH HHS P01 HL158506NHLBI NIH HHS UH3 HL147367
6 · The paper itself

Abstract

Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the

Indexed as

AAVBase editinggene editingmouse liverrare diseases

Identifiers

PMID40654892
PMCPMC12248072

What OpenQuestion 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 OpenQuestion graph.