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Drug intelligence / Profile preview
antisense oligonucleotides targeting C19MC miRNAs
- Development stage
- Preclinical
- Lead developer
- Dana-Farber Cancer Institute
- 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
Antisense oligonucleotides targeting C19MC miRNAs are synthetic oligonucleotide therapeutics designed to silence members of the chromosome 19 microRNA cluster (C19MC). C19MC is a large microRNA gene cluster that is amplified and overexpressed in embryonal tumors with multilayered rosettes (ETMR), a highly aggressive pediatric brain tumor. By utilizing locked nucleic acid (LNA)-based or other short antisense oligonucleotides to target and silence specific oncogenic C19MC miRNA families, these agents aim to disrupt the transcriptional network governing stemness, self-renewal, and pluripotency in ETMR stem-like cells, thereby inhibiting tumor cell growth and inducing apoptosis.
- Other names
- LNA-based antisense oligonucleotides targeting C19MCantisense oligonucleotides targeting C19MCshort antisense oligonucleotides targeting C19MC
02
Targets
C19MC (Chromosome 19 microRNA cluster)
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