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AAV-CaV1.3-shRNA
- Development stage
- Preclinical
- Lead developer
- CavGene Therapeutics
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral
01
Overview
AAV-CaV1.3-shRNA is an adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of Parkinson's disease. Developed by CavGene Inc., the therapy utilizes RNA interference (RNAi) via a short hairpin RNA (shRNA) to specifically knock down the expression of CaV1.3 L-type voltage-gated calcium channels. These channels are implicated in the metabolic vulnerability of substantia nigra dopamine neurons due to their role in autonomous pacemaking. By delivering the vector intraputaminally, the therapy achieves retrograde transduction of surviving nigrostriatal neurons, aiming to restore the dopaminergic phenotype and reverse motor deficits. Preclinical studies in non-human primates have demonstrated significant behavioral improvements and evidence of neurorestoration.
- Other names
- rAAV-CaV1.3rAAV-CaV-1.3rAAV-CaV 1.3rAAV-shRNA-CaV1.3rAAV-shRNA-CaV-1.3rAAV-shRNA-CaV 1.3
02
Targets
CACNA1D (Voltage-dependent L-type calcium channel subunit alpha-1D)
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