Evidence map›Paper›PMID 41485391›Full record

ArticleMolecular genetics and metabolism2026

Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease.

Baodong Sun, Haiqing Yi, Sang-Oh Han, Songtao Li, William Eisner, Elizabeth D Brooks, Rebecca Bacon, Dwight D Koeberl

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism, 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

8 authors.

Baodong SunDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Haiqing YiDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Sang-Oh HanDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Songtao LiDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
William EisnerDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Elizabeth D BrooksDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Rebecca BaconDepartment of Pathology, Duke University School of Medicine, Durham, NC, USA.
Dwight D KoeberlDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA; Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC, USA. Electronic address: dwight.koeberl@duke.edu.

Funding

Stable therapy in Pompe disease through genome editingR01AR079223 · NIAMS · DUKE UNIVERSITY · PI KOEBERL, DWIGHT D · 2021 to 2025
$3.3M
Skeletal muscle-targeted gene therapy for Pompe diseaseR01AR085087 · NIAMS · DUKE UNIVERSITY · PI Baodong Sun · 2025 to 2026
$1.1M
NIAMS NIH HHS R01 AR079223NIAMS NIH HHS R01 AR085087
6 · The paper itself

Abstract

Systemically administered gene therapy is under development for the treatment of Pompe disease, an inherited lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency. We evaluated recombinant AAV9 vectors expressing GAA driven by the minimal G6PC promoter (AAV9-G6PC.GAA) and by a hybrid CRM4-G6PC promoter (AAV9-MyoG6PC.GAA) and intravenously administered the vectors (1 × 10

Indexed as

alpha-GlucosidasesGenetic TherapyGlycogen Storage Disease Type IIPromoter Regions, GeneticTransgenesAnimalsDependovirusDisease Models, AnimalFemaleGenetic VectorsGlycogenHumansLiverMaleMiceMice, Knockoutalpha-GlucosidasesGlycogenAdeno-associated virus vectorGene therapyGlycogen storage diseasePompe disease

Identifiers

PMID41485391
PMCPMC13133977

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.