Double administration of self-complementary AAV9(NDUFS4) prevents Leigh disease in Ndufs4(-/-) mice

Corra, S; Cerutti, R; Balmaceda, V; Viscomi, C; Zeviani, M

Zeviani, M (通讯作者),Univ Padua, Clin Sch, Dept Neurosci, Veneto Inst Mol Med, I-35128 Padua, Italy.;Viscomi, C (通讯作者),Univ Padua, Clin Sch, Dept Biomed Sci, I-35131 Padua, Italy.

BRAIN, 2022; 145 (10): 3405

Abstract

Corra et al. show that double administration of a self-complementary-adeno-associated viral 9 vector expressing human NDUFS4, via intra-vascular and i......

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