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A53T-specific antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Hellenic Pasteur Institute
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
A53T-specific antisense oligonucleotide is an allele-specific antisense oligonucleotide (ASO) designed to selectively target and downregulate the mutant SNCA p.A53T allele while preserving the expression of the wild-type (WT) alpha-synuclein allele. Developed by Vanda Pharmaceuticals in collaboration with the Hellenic Pasteur Institute, this 18-mer gapmer phosphorothioate methoxyethyl ASO is designed to have a higher affinity for the mutant allele. By selectively reducing the expression of the aberrant gain-of-function A53T alpha-synuclein, the therapy aims to restore synaptic function, improve neurite branching, and enhance neuronal viability, offering a potential disease-modifying treatment for early-onset Parkinson's disease without risking the neurodegeneration associated with broad alpha-synuclein depletion.
- Other names
- A53T-specific ASOA-53T-specific ASOA 53T-specific ASOallele-specific ASO targeting SNCA p.A53Tallele-specific antisense oligonucleotide targeting SNCA p.A53T
02
Targets
SNCA (Alpha-synuclein)
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