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AAV-shRNA (Scn8a)
- Development stage
- Preclinical
- Lead developer
- University of Michigan
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Parenteral
01
Overview
AAV-shRNA (Scn8a) is a preclinical gene therapy construct designed to downregulate the expression of the Scn8a gene, which encodes the voltage-gated sodium channel subunit Nav1.6. Developed by researchers at the University of Michigan and the Children's Hospital of Philadelphia, this therapy utilizes an adeno-associated virus (AAV) vector to deliver short hairpin RNA (shRNA) targeting Scn8a. It is being investigated for the treatment of SCN8A-related developmental and epileptic encephalopathy (SCN8A-DEE) and other severe genetic epilepsies, such as Dravet syndrome. In preclinical mouse models, targeted reduction of Scn8a expression in the dentate gyrus of the hippocampus has been shown to reduce neuronal hyperexcitability, increase seizure thresholds, and significantly prolong survival.
- Other names
- AAV-shRNA-Scn8aAAV-shRNA-Scn-8aAAV-shRNA-Scn 8aAAV-shRNA-Scn8a-GFPAAV-shRNA-Scn-8a-GFPAAV-shRNA-Scn 8a-GFPAAV10-Scn8a-shRNAAAV-10-Scn8a-shRNAAAV 10-Scn8a-shRNA
02
Targets
SCN8A (NaV1.6)
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