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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...
Autores principales: | Suzuki, Naoki, Nishiyama, Ayumi, Warita, Hitoshi, Aoki, Masashi |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Nature Singapore
2022
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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 |
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