Therapeutic reduction of GGGGCC repeat RNA levels by hnRNPA3 suppresses neurodegeneration in Drosophila models of C9orf72-linked ALS/FTD

Taminato, T; Takeuchi, T; Ueyama, M; Mori, K; Ikeda, M; Mochizuki, H; Nagai, Y

Takeuchi, T; Nagai, Y (通讯作者)，Kindai Univ, Fac Med, Dept Neurol, 377-2 Ohnohigashi, Osaka, Osaka 5898511, Japan.;Takeuchi, T; Nagai, Y (通讯作者)，Kindai Univ, Life Sci Res Inst, 377-2 Ohnohigashi, Osaka, Osaka 5898511, Japan.

HUMAN MOLECULAR GENETICS, 2023; 32 (10): 1673

Abstract

The abnormal expansion of GGGGCC hexanucleotide repeats within the C9orf72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS......

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