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miR-183-5p antagomir
- Development stage
- Preclinical
- Lead developer
- Baylor College of Medicine
- 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
miR-183-5p antagomir is a chemically modified, single-stranded antisense oligonucleotide designed to specifically inhibit the activity of microRNA-183-5p (miR-183-5p). In preclinical models of amyotrophic lateral sclerosis (ALS), miR-183-5p is upregulated and promotes the pathological aggregation, mislocalization, and neurotoxicity of TAR DNA-binding protein 43 (TDP-43) by suppressing sequestosome 1 (SQSTM1/p62), a critical protein in autophagy and protein recycling. Treatment with the miR-183-5p antagomir restores SQSTM1/p62 expression, thereby reducing stress granule formation, decreasing aggregated TDP-43 levels, and protecting neuronal cells from cytotoxicity. It is widely used as a research tool to investigate the therapeutic potential of targeting the miR-183-5p-SQSTM1/p62 axis in neurodegenerative diseases.
- Other names
- antagomir-183-5pantagomir183-5pantagomir 183-5phsa-miR-183-5p antagomirhsa-miR183-5p antagomirhsa-miR 183-5p antagomirmiR-183-5p inhibitormiR183-5p inhibitormiR 183-5p inhibitormicroRNA-183-5p antagomirmicroRNA183-5p antagomirmicroRNA 183-5p antagomir
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