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LXR agonist rescues synaptic dysfunction and degeneration in SPG3A patient-specific iPSC-derived neurons

Abstract Hereditary spastic paraplegias (HSPs) comprise a large, heterogeneous group of inherited disorders characterized by length-dependent axonal degeneration of corticospinal motor neurons, leading to lower extremity spasticity and gait impairment. Currently, there are no effective treatments fo...

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Principais autores: Gitika Thakur, Rutuja Dhanukate, Yongchao Mou, Priya Kunhiraman, Archana Khadilkar, Siddharth Srivastava, Julian E. Alecu, Darius Ebrahimi-Fakhari, Zhenyu Chen, Craig Blackstone, Xue-Jun Li
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2025-11-01
Serier:Acta Neuropathologica Communications
Fag:
Hereditary spastic paraplegias
iPSC
Axon degeneration
Lipid homeostasis
Synaptic dysfunction
Online adgang:https://doi.org/10.1186/s40478-025-02134-5
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