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antagomir against miR-451
- Development stage
- Unknown
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
- Administration
- Intravenous, Subcutaneous
01
Overview
Antagomir against miR-451 (also known as antagomir-451) is a chemically modified, single-stranded synthetic RNA oligonucleotide designed to silence endogenous microRNA-451 (miR-451). Antagomirs typically feature 2'-O-methyl modifications, phosphorothioate backbones, and a 3'-cholesterol conjugate to enhance stability and cellular uptake. In preclinical research, inhibition of miR-451 has been investigated for its therapeutic potential in various conditions, including beta-thalassemia, where silencing miR-451 derepresses CAB39 (MO25), thereby activating the LKB1/AMPK pathway, suppressing mTORC1, and promoting ULK1-mediated autophagy to clear toxic free alpha-globin precipitates. It has also been studied in models of myocardial ischemia, diabetic cardiomyopathy, and osteoblastogenesis.
- Other names
- antagomir-451antagomir451antagomir 451miR-451 antagomirmiR451 antagomirmiR 451 antagomirmiR-451 inhibitormiR451 inhibitormiR 451 inhibitormicroRNA-451 inhibitormicroRNA451 inhibitormicroRNA 451 inhibitor
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