Preemptive SOD1 Silencing via Neonatal Intramuscular AAV Therapy Modifies Disease Trajectory in an ALS Mouse Model

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disorder with limited therapeutic options. Mutations in the gene encoding superoxide dismutase 1 ( SOD1 ) represent a major genetic cause of familial ALS, driving motor neuron degeneration through toxic gain-of-function mechanisms. Although gene silencing approaches targeting SOD1 show substantial therapeutic potential, their clinic...

Topics

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• Amyotrophic Lateral Sclerosis
• Amyotrophic Lateral Sclerosis Research
• Dependovirus
• Disease Models, Animal
• Gene Expression Regulation, Developmental
• Gene Silencing
• Superoxide Dismutase
• Superoxide Dismutase-1

Identifiers and source

Literature Corpus work

7efb9218-8b9b-515f-aa32-316d61d75dfd

DOI

10.1101/2025.10.17.682996