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Scn8a antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Scn8a antisense oligonucleotide is an investigational antisense oligonucleotide (ASO) designed to down-regulate the expression of the SCN8A gene, which encodes the voltage-gated sodium channel subunit alpha Nav1.6. Developed by the University of Michigan in collaboration with Ionis Pharmaceuticals, this ASO targets a sequence in the 3' untranslated region (UTR) of the Scn8a transcript, leading to its degradation. In preclinical mouse models of SCN8A developmental and epileptic encephalopathy (DEE) and Dravet syndrome, intracerebroventricular administration of the Scn8a ASO reduced Scn8a expression, delayed seizure onset, and significantly prolonged survival.
- Other names
- SCN8A antisense oligonucleotideSCN-8A antisense oligonucleotideSCN 8A antisense oligonucleotideSCN8A-ASOSCN-8A-ASOSCN 8A-ASOScn8a ASOScn-8a ASOScn 8a ASO
02
Targets
SCN8A (NaV1.6)
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