The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.

Nucleic Acids Res · 2021 · PMC7826274 · PMID 33367834

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In a recent phase 1–2 trial in SOD1-ALS patients, IT administration of Tofersen, a MOE-gapmer ASO targeting SOD1 , significantly reduced SOD1 concentrations in CSF and despite the low number of patients enrolled in the study showed a trend towards slowing disease progression in ASO-treated patients ( 19 ).

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