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scAAV9.miKcnq3
- Development stage
- Preclinical
- Lead developer
- Columbia University
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
scAAV9.miKcnq3 is an investigational gene therapy designed to treat KCNQ3 gain-of-function (GoF) neurodevelopmental disorders (NDD) and associated spontaneous seizures. Developed by Nationwide Children's Hospital and Columbia University, it consists of a self-complementary adeno-associated virus serotype 9 (scAAV9) vector that delivers a microRNA (miKcnq3) targeting KCNQ3 mRNA. By utilizing RNA interference (RNAi) to knock down KCNQ3 expression, the therapy aims to reduce Kcnq3 mRNA and Kv7.3 protein levels, thereby mitigating neuronal hyperexcitability and suppressing spontaneous seizures.
- Other names
- AAV.miKCNQ3scAAV9-U6-miKcnq3a-eGFPscAAV-9-U6-miKcnq3a-eGFPscAAV 9-U6-miKcnq3a-eGFPscAAV9-miKcnq3-AscAAV-9-miKcnq3-AscAAV 9-miKcnq3-A
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