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Sry antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- XYnapse Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intracerebral
01
Overview
Sry antisense oligonucleotide is an experimental RNA-based therapy designed to knock down the expression of the Y-chromosome gene *SRY* (Sex-determining region Y). Research indicates that *SRY* is expressed in male dopamine (DA) neurons in the substantia nigra and is significantly upregulated in Parkinson's disease (PD) models. By inhibiting *SRY* synthesis, this ASO aims to provide sex-specific neuroprotection for male dopamine neurons. Preclinical studies in toxin-induced rat models of PD have shown that reducing nigral *SRY* levels diminishes motor deficits, prevents dopamine cell loss, and reduces markers of DNA damage, mitochondrial degradation, and neuroinflammation. The therapy is being developed as a potential disease-modifying treatment specifically for men with Parkinson's disease, with humanized versions utilizing 2-O-Methoxyethyl (MOE) chemistry currently in development.
- Other names
- Sry ASOSRY antisense oligonucleotide
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