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

Overview

Authors: Slawomir Wantuch^1,2, Steven Tuske³, Sara Benedetti¹, Nickita Mehta³, Jon J. Brudvig³, Hung Do³, Russell Gotschall³, Jessie Feng³, Anna B. Gaspar², Chiara Recchi², Paul Heal², Tyler B. Johnson³, Fulvio Mavilio², Pervinder Sagoo², Jill M. Weimer³, H. Bobby Gaspar^1,2

ORCID iDs: Pervinder Sagoo

Institutions: Orchard Therapeutics (United Kingdom) (United Kingdom); University College London (United Kingdom); Amicus Therapeutics (United States) (United States)

Journal: Molecular therapy. Advances, volume 34, issue 3, article 201824

Dates: received 6 February 2026; accepted 28 July 2026; published online 30 July 2026

Type: Research article · Language: English

License: CC BY-NC-ND

Identifiers: DOI 10.1016/j.omta.2026.201824 · PMID 42633127 · PMCID PMC13499122 · OpenAlex W7171767859

Open access: hybrid, a free copy (OpenAlex)

Status: code on request

Categories: mouse (organism)

Methods: Statistics

Keywords: Pompe disease, variant insulin growth factor-2 tag, hematopoietic stem cell gene therapy, ex vivo gene therapy

Topic: Lysosomal Storage Disorders Research (Physiology, Medicine), according to OpenAlex

Funding: Orchard Therapeutics (Europe) Limited; UCL and Amicus Therapeutics; NIHR Biomedical Research Centre; Great Ormond Street Hospital for Children NHS Foundation Trust; UCL Confidence in Concept Fund; UCL Technology Fund; UCL Department of Cardiovascular Imaging

Citations: not cited yet (Europe PMC); 61 references in the paper

Abstract

The abstract is not reproduced here: the paper's license (CC BY-NC-ND) does not allow it. Read it in the paper, at the publisher or on Europe PMC.

Code

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Data

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Code and data availability statement

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Read it in the paper: doi.org/10.1016/j.omta.2026.201824.

Versions

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Version 2, 28 September 2026

Authors: added Pervinder Sagoo (0000-0003-2568-9126); removed Pervinder Sagoo

Version 1, 27 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 16 authors, 4 keywords, 7 funders, 58 references.

Cite

This paper

Wantuch, S., Tuske, S., Benedetti, S., Mehta, N., Brudvig, J. J., Do, H., Gotschall, R., Feng, J., Gaspar, A. B., Recchi, C., Heal, P., Johnson, T. B., Mavilio, F., Sagoo, P., Weimer, J. M., & Gaspar, H. B. (2026). GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease. Molecular therapy. Advances, 34(3), 201824. https://doi.org/10.1016/j.omta.2026.201824

BibTeX

@article{wantuch2026gaa, author = {Wantuch, Slawomir and Tuske, Steven and Benedetti, Sara and Mehta, Nickita and Brudvig, Jon J. and Do, Hung and Gotschall, Russell and Feng, Jessie and Gaspar, Anna B. and Recchi, Chiara and Heal, Paul and Johnson, Tyler B. and Mavilio, Fulvio and Sagoo, Pervinder and Weimer, Jill M. and Gaspar, H. Bobby}, title = {{GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease}}, journal = {Molecular therapy. Advances}, year = {2026}, month = jul, volume = {34}, number = {3}, pages = {201824}, publisher = {American Society of Gene \& Cell Therapy}, issn = {3117-387X}, doi = {10.1016/j.omta.2026.201824}, url = {https://doi.org/10.1016/j.omta.2026.201824}, pmid = {42633127}, pmcid = {PMC13499122} }

RIS

TY - JOUR AU - Wantuch, Slawomir AU - Tuske, Steven AU - Benedetti, Sara AU - Mehta, Nickita AU - Brudvig, Jon J. AU - Do, Hung AU - Gotschall, Russell AU - Feng, Jessie AU - Gaspar, Anna B. AU - Recchi, Chiara AU - Heal, Paul AU - Johnson, Tyler B. AU - Mavilio, Fulvio AU - Sagoo, Pervinder AU - Weimer, Jill M. AU - Gaspar, H. Bobby TI - GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease T2 - Molecular therapy. Advances J2 - Mol Ther Adv PY - 2026 DA - 2026/07/30 VL - 34 IS - 3 SP - 201824 SN - 3117-387X PB - American Society of Gene & Cell Therapy DO - 10.1016/j.omta.2026.201824 UR - https://doi.org/10.1016/j.omta.2026.201824 LA - en ER -

CSL-JSON

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