Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a refractory neurodegenerative disease characterized by the de-generation and loss of motor neurons, leading to death within five years of onset. There have been few effective treatments, making the development of robust therapies an urgent challenge. Genetic mutations have been identified as contributors to ALS, with mutations in superoxide dismutase 1 (SOD1), which neutrali...

Topics

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• Amyotrophic Lateral Sclerosis
• Amyotrophic Lateral Sclerosis Research
• Cholinesterase and Neurodegenerative Diseases
• Mutation
• Oligonucleotides
• Oligonucleotides, Antisense
• Prion Diseases and Protein Misfolding
• Superoxide Dismutase-1

Identifiers and source

Literature Corpus work

25520420-d8a0-5c37-84ff-6a466db91482

DOI

10.20944/preprints202409.2440.v1