Prospects for gene replacement therapies in amyotrophic lateral sclerosis

I Giovannelli, A Higginbottom, J Kirby… - Nature Reviews …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating and incurable neurodegenerative
disease characterized by the progressive loss of upper and lower motor neurons. ALS …

[HTML][HTML] Autophagy dysfunction in ALS: from transport to protein degradation

M Cozzi, V Ferrari - Journal of Molecular Neuroscience, 2022 - Springer
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting upper and
lower motor neurons (MNs). Since the identification of the first ALS mutation in 1993, more …

OPTN gene therapy increases autophagy and protects mitochondria in SOD1‐G93A‐expressing transgenic mice and cells

D Wen, Y Ji, Y Li, W Duan, Y Wang, Z Li… - The FEBS …, 2024 - Wiley Online Library
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by
progressive motor neuron (MN) death. Mutation of the superoxide dismutase 1 (SOD1) …