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AAV-ATP6V0C
- Development stage
- Preclinical
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV-ATP6V0C is an adeno-associated virus (AAV)-mediated gene therapy designed to overexpress the ATP6V0C subunit of the V-type ATPase (v-ATPase) proton pump. In Parkinson's disease (PD), lysosomal dysfunction and impaired acidification lead to the accumulation of pathological alpha-synuclein (α-syn). By delivering the ATP6V0C gene, this therapy aims to restore lysosomal pH, enhance the degradation of aggregated proteins, and prevent dopaminergic neuron loss. Preclinical studies in mouse models of PD (alpha-synuclein pre-formed fibril models) have demonstrated that ATP6V0C overexpression mitigates motor deficits and reduces α-syn aggregation.
02
Targets
V-ATPase (Vacuolar-type H+-ATPase)
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