Protocol for Screening of Small Molecules in a CLN3 Disease Patient-Specific iPSC-Derived Neuronal Progenitor Cell Model

Authors

Ramu Venkatesan, University of Nebraska Medical Center
Paul C. Trippier, University of Nebraska Medical CenterFollow

Document Type

Article

Journal Title

STAR Protocols

Publication Date

2025

Volume

6

Abstract

Although rare, CLN3 disease is the most common neurodegenerative disorder of childhood. Here, we present a protocol for screening small molecules in a CLN3 disease patient-specific induced pluripotent stem cell (iPSC)-derived neuronal progenitor cell (NPC) model. We describe steps for converting human iPSCs (hiPSCs) to neural stem cells (NSCs) and developing NPCs using feeder-free medium and cytokine-based differentiation. We then detail procedures for achieving access to mature neurons. For complete details on the use and execution of this protocol, please refer to Simeon et al.

DOI Link

10.1016/j.xpro.2025.104269

ISSN

2666-1667

Creative Commons License

This work is licensed under a Creative Commons Attribution-NonCommercial-No Derivative Works 4.0 International License.

Recommended Citation

Venkatesan, Ramu and Trippier, Paul C., "Protocol for Screening of Small Molecules in a CLN3 Disease Patient-Specific iPSC-Derived Neuronal Progenitor Cell Model" (2025). Journal Articles: Pharmaceutical Sciences. 50.
https://digitalcommons.unmc.edu/cop_pharmsci_articles/50