Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice

Authors

• Ewa A Ziółkowska, Washington University School of Medicine in St. Louis
• Letitia L Williams, Washington University School of Medicine in St. Louis
• Matthew J Jansen, Washington University School of Medicine in St. Louis
• Sophie H Wang, Washington University School of Medicine in St. Louis
• Elizabeth M Eultgen, Washington University School of Medicine in St. Louis
• Jaiprakash Sharma, Washington University School of Medicine in St. Louis
• Marco Sardiello, Washington University School of Medicine in St. Louis
• Rebecca P Bradley, Children's Hospital of Philadelphia (CHOP)
• Ineka T Whiteman, Batten Disease Support, Research and Advocacy Foundation (US)
• Mark S Sands, Washington University School of Medicine in St. Louis
• Robert O Heuckeroth, University of Pensylvania
• Jonathan D Cooper, Washington University School of Medicine in St. Louis

Journal

Acta Neuropathologica Communications

Publication Date

12-22-2025

Volume

13

Issue

1

First Page

260

Document Type

Open Access Publication

DOI

10.1186/s40478-025-02205-7

Rights and Permissions

Ziółkowska, E.A., Williams, L.L., Jansen, M.J. et al. Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice. acta neuropathol commun 13, 260 (2025). https://doi.org/10.1186/s40478-025-02205-7 This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

Recommended Citation

Ziółkowska, Ewa A; Williams, Letitia L; Jansen, Matthew J; Wang, Sophie H; Eultgen, Elizabeth M; Sharma, Jaiprakash; Sardiello, Marco; Bradley, Rebecca P; Whiteman, Ineka T; Sands, Mark S; Heuckeroth, Robert O; and Cooper, Jonathan D, "Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice." Acta Neuropathologica Communications. 13, 1. 260 (2025).
https://digitalcommons.wustl.edu/oa_4/6885

Department

ICTS (Institute of Clinical and Translational Sciences)

Additional Links

Supplemental material is available for this article at publisher site.