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small interfering RNA targeting alpha-synuclein
- Development stage
- Unknown
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intranasal, Intrathecal, Subcutaneous
01
Overview
Small interfering RNA (siRNA) targeting alpha-synuclein (encoded by the SNCA gene) is a therapeutic platform designed to reduce the expression of alpha-synuclein, a protein whose pathological aggregation and accumulation are central to the pathogenesis of Parkinson's disease and other synucleinopathies. By utilizing the RNA interference (RNAi) pathway, these double-stranded RNA molecules are incorporated into the RNA-induced silencing complex (RISC), where they guide the sequence-specific cleavage and degradation of SNCA messenger RNA (mRNA). This knockdown of SNCA mRNA subsequently lowers the levels of both soluble and aggregated alpha-synuclein protein in the brain. Various delivery systems, including chemical modifications, lipid nanoparticles, and intranasal, subcutaneous, or intrathecal administration, are being developed to optimize central nervous system distribution, cellular uptake, and duration of effect.
- Other names
- alpha-synuclein siRNAsiRNA targeting SNCAsiRNA targeting alpha-synucleinsiSNCAsmall interfering RNA targeting SNCA
02
Targets
AGO2 (Argonaute RISC catalytic component 2)Alpha-synuclein messenger RNASNCA (Alpha-synuclein)
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