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ASO-CDNF
- Development stage
- Preclinical
- Lead developer
- Western University
- 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
- Implant
01
Overview
ASO-CDNF is an antisense oligonucleotide (ASO) designed to knockdown the expression of Cerebral Dopamine Neurotrophic Factor (CDNF). It is primarily utilized in preclinical research as a tool to induce phenotypes mimicking Parkinson's disease (PD), Parkinson's disease dementia (PDD), and Lewy body dementia (LBD) in rodent models. By inhibiting CDNF, which acts as a regulator of endoplasmic reticulum (ER) stress and the unfolded protein response (UPR), the ASO promotes alpha-synuclein aggregation and neurodegeneration. The specific sequence is typically phosphorothioate-modified to enhance stability against nuclease degradation. While the ASO itself is used to create disease models, the study of CDNF knockdown helps validate CDNF-based mimetics and neuroprotective strategies as potential disease-modifying therapies for synucleinopathies.
- Other names
- antisense oligonucleotide targeting CDNFCDNF knockdown ASO
02
Targets
Cerebral dopamine neurotrophic factor mRNA
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