Evidence map›Paper›PMID 42633127›Full record

ArticleMolecular therapy. Advances2026

GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease.

Slawomir Wantuch, Steven Tuske, Sara Benedetti, Nickita Mehta, Jon J Brudvig, Hung Do, Russell Gotschall, Jessie Feng, Anna B Gaspar, Chiara Recchi and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 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

16 authors.

Slawomir WantuchMolecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.
Steven TuskeAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Sara BenedettiMolecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.
Nickita MehtaAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Jon J BrudvigAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Hung DoAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Russell GotschallAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Jessie FengAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Anna B GasparOrchard Therapeutics (Europe) Limited, London W6 8PW, UK.
Chiara RecchiOrchard Therapeutics (Europe) Limited, London W6 8PW, UK.
Paul HealOrchard Therapeutics (Europe) Limited, London W6 8PW, UK.
Tyler B JohnsonAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
Fulvio MavilioOrchard Therapeutics (Europe) Limited, London W6 8PW, UK.
Pervinder SagooOrchard Therapeutics (Europe) Limited, London W6 8PW, UK.
Jill M WeimerAmicus Therapeutics, Inc., Princeton, NJ 08542, USA.
H Bobby GasparMolecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pompe disease (PD) is a severe autosomal-recessive lysosomal storage disorder in which deficiency of acid alpha-glucosidase (GAA) enzyme function results in glycogen accumulation and a spectrum of clinically severe neuromuscular disease. Enzyme replacement therapy is the standard of care for PD, although its efficacy is limited by inefficient uptake by cells in target organs. Autologous transplantation of hematopoietic stem cells (HSCs), genetically modified

Indexed as

ex vivo gene therapyhematopoietic stem cell gene therapyPompe diseasevariant insulin growth factor-2 tag

Identifiers

PMID42633127
PMCPMC13499122

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.