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References from The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS. Local targets link to admitted publications; unresolved targets remain external evidence.
Oligonucleotide-siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial
10.1038/s41591-026-04491-7 · 2026 · External reference
Morpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype
10.1038/srep21301 · 2016 · External reference
Aggregation and motor neuron toxicity of an ALS-linked SOD1 mutant independent from wild-type SOD1
10.1126/science.281.5384.1851 · 1998 · External reference
Long-term tofersen in SOD1 amyotrophic lateral sclerosis
10.1001/jamaneurol.2025.4946 · 2026 · External reference
Trial of antisense oligonucleotide tofersen for SOD1 ALS
10.1056/nejmoa2204705 · 2022 · External reference
Human cerebellar organoids with functional Purkinje cells
10.1016/j.stem.2023.11.013 · 2024 · External reference
