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miR-134 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Neumirna Therapeutics
- 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
- Administration
- Intrathecal
01
Overview
miR-134 antisense oligonucleotides (also known as antimiR-134 or antagomir-134) are synthetic, chemically-modified RNA-targeted therapeutics designed to selectively inhibit microRNA-134 (miR-134). miR-134 is a brain-specific microRNA that is upregulated in patients with drug-resistant temporal lobe epilepsy and other neurological conditions, where it acts as a key regulator of neuronal microstructure and excitability by negatively regulating dendritic spine volume. By silencing miR-134, these antisense oligonucleotides (ASOs) promote neuroprotection, reduce hyperexcitability, and suppress spontaneous recurrent seizures. Neumirna Therapeutics is developing NMT.001, a proprietary design-optimized miR-134 ASO, as a disease-modifying therapy for drug-resistant epilepsy. Preclinical studies have demonstrated that NMT.001 achieves potent knockdown of miR-134, leading to significant seizure reduction and rescue of cognitive and behavioral comorbidities in animal models.
- Other names
- anti-miR-134anti-miR134anti-miR 134antagomir-134antagomir134antagomir 134antimiR-134antimiR134antimiR 134
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