ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease

Vasconcelos-Ferreira, A; Martins, IM; Lobo, D; Pereira, D; Lopes, MM; Faro, R; Lopes, SM; Verbeek, D; Schmidt, T; Nobrega, C; de Almeida, LP

de Almeida, LP (通讯作者),Univ Coimbra, Fac Med, CNC Ctr Neurosci & Cell Biol, Mol Therapy Brain Disorders Grp, Rua Larga,Polo 1, P-3004504 Coimbra, Portugal.

MOLECULAR THERAPY, 2022; 30 (1): 370

Abstract

Machado-Joseph disease (MJD) is a fatal neurodegenerative disorder clinically characterized by prominent ataxia. It is caused by an expansion of a CAG......

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