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Article

Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy.

Journal of human genetics - 1 Mar 2023

Suzuki Naoki, Nishiyama Ayumi, Warita Hitoshi, Aoki Masashi

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure leading to mortality. The main locus of ALS is motor neurons. The success of antisense oligonucleotide (ASO) therapy in spinal muscular atrophy (SMA), a motor neuron disease, has triggered a paradigm shift in developing ALS therapies. The causative genes of ALS and disease-modifying genes, including those of sporadic...

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Identifiers

PMID: 35691950
DOI: 10.1038/s10038-022-01055-8
PMCID: PMC9968660

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