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New antisense oligonucleotide therapies reach first base in ALS

Nature.com
 2022-01-24

Two studies highlight the evolution of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for an effective treatment. In 1993, mutations in the superoxide dismutase 1 (SOD1) gene were the first genetic cause identified in amyotrophic lateral sclerosis (ALS), a neurological disease characterized by rapidly progressive paralysis and death from...

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