Small Molecules to Elevate Rab7-GTPase Activity and Lower Cholesterol Accumulation in Niemann-Pick Type C Disease
Advancing small molecules that can elevate Rab7-GTPase activity could provide opportunities to overcome cholesterol transport defects in NPC mutant cells and offer applications in other Rab7-related neurological diseases.
Authors
- Michael A. Serwetnyk (ORCID: https://orcid.org/0000-0001-5507-2923)
- Christopher Patzke (ORCID: https://orcid.org/0000-0001-7293-8936)
- Jianfeng Du (ORCID: https://orcid.org/0000-0001-6931-3337)
- Ann-Na Cho
- Agata Szulc (ORCID: https://orcid.org/0000-0003-0059-9861)
- Brandon L. Ashfeld (ORCID: https://orcid.org/0000-0002-4552-2705)
- Brian S. J. Blagg (ORCID: https://orcid.org/0000-0002-6200-3480)
- Paul W. Groundwater (ORCID: https://orcid.org/0000-0002-4932-2030)
- Thomas Grewal (ORCID: https://orcid.org/0000-0002-7937-8887)
- Hanna-Loisa Walther
- C. Pinkenburg
- David E. Hibbs
- Shihui Chen
- Ravi Wikramanayake
- Yangjing Liu
- Martje Hartmann
- Eric Sparkes
- Marc Bernaus-Esqué
- Francesc Tebar
- Maya R. Nikenich
- Anna Wenninger
- Felcia Lai
- Kim Seifert
- Andrew J. Hoy
- Mai K. L. Nguyen
- Carles Rentero
Institutions
- University of Notre Dame (US)
- The University of Sydney (AU)
- Departament de Salut (ES)
- Fundació Clínic per a la Recerca Biomèdica (ES)
- Consorci Institut D'Investigacions Biomediques August Pi I Sunyer (ES)
- Fundació de Recerca Clínic Barcelona-Institut d’Investigacions Biomèdiques August Pi i Sunyer (ES)
- Universitat de Barcelona (ES)
Publication Details
- Journal
- Pharmaceutical Research
- Published
- 2026-03-03
- DOI
- https://doi.org/10.1007/s11095-026-04058-8
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00