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microRNA-193b-3p mimic

Development stage
Preclinical
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal, Intravenous
01

Overview

microRNA-193b-3p mimic is a synthetic, double-stranded RNA oligonucleotide designed to replicate the biological activity of the endogenous mature microRNA, miR-193b-3p. In the context of neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS), miR-193b-3p has been identified as a regulator of the TSC1/mTORC1 signaling axis. The mimic functions by binding to the 3' untranslated region (UTR) of the Tuberous Sclerosis 1 (TSC1) mRNA, leading to its degradation or translational repression. Since TSC1 is a critical negative regulator of the mechanistic target of rapamycin complex 1 (mTORC1), the administration of the miR-193b-3p mimic results in the activation of mTORC1 signaling. This activation subsequently inhibits autophagy and can promote cell death in motor neurons, as demonstrated in NSC-34 cell models. While its role in ALS suggests that its downregulation may be a protective mechanism to enhance autophagy, miR-193b-3p mimics are also investigated in oncology as potential tumor suppressors due to their ability to inhibit cell proliferation and invasion in various cancers.

Other names
miR-193b-3pmiR193b-3pmiR 193b-3pmicroRNA-193b-3pmicroRNA193b-3pmicroRNA 193b-3phsa-miR-193b-3p mimichsa-miR193b-3p mimichsa-miR 193b-3p mimic
02

Targets

TSC1 (TSC1 / Hamartin)

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