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

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...

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Detalles Bibliográficos
Autores principales: Suzuki, Naoki, Nishiyama, Ayumi, Warita, Hitoshi, Aoki, Masashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Nature Singapore 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9968660/
https://www.ncbi.nlm.nih.gov/pubmed/35691950
http://dx.doi.org/10.1038/s10038-022-01055-8