Evidence map›Paper›PMID 41588875›Full record

ReviewHuman gene therapy2026

Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.

Jenny Gao, Erik Sontheimer, Terence R Flotte, Wen Xue

Abstract readReview
In one paragraph

Review in Human gene therapy, 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

4 authors.

Jenny GaoRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.
Erik SontheimerRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.
Terence R FlotteLi Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.
Wen XueRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.

Funding

Vector Immunology CoreP01HL158506 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Wen Xue · 2021 to 2026
$19.5M
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
NHLBI NIH HHS F30 HL176024NHLBI NIH HHS P01 HL158506
6 · The paper itself

Abstract

Genome and RNA editing modalities have revolutionized precision gene therapy, offering a safer alternative to traditional gene replacement approaches. Alpha-1 antitrypsin deficiency (AATD) is a compelling model for precision medicine because the disease mechanism is well defined-mutations in a single gene are responsible for both liver and lung pathology. In this review, we summarize the current preclinical and clinical efforts for AATD, with an emphasis on genome and RNA editing strategies.

Indexed as

alpha 1-Antitrypsinalpha 1-Antitrypsin DeficiencyGene EditingGenetic TherapyPrecision MedicineAnimalsHumansMutationRNA Editingalpha 1-Antitrypsinalpha-1 antitrypsinbase editinggene editingprime editingRNA editing

Identifiers

PMID41588875
PMCPMC13372069

What OpenQuestion holds

Textmetadata
LicenceTDM
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.