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THBS1-AS1 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Mayo Clinic
- 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
- Intravenous
01
Overview
THBS1-AS1 antisense oligonucleotide is an antisense oligonucleotide (ASO) designed to target and silence the long non-coding RNA (lncRNA) THBS1-AS1 (thrombospondin 1 antisense RNA 1). Developed by researchers at the Mayo Clinic, this therapeutic agent is being investigated for the treatment of progressive biliary diseases, such as primary sclerosing cholangitis (PSC), and cardiac fibrosis. THBS1-AS1 is selectively upregulated in diseased cholangiocytes and cardiac fibroblasts under TGF-β stimulation. In biliary disease, THBS1-AS1 interacts with the epigenetic silencer EZH2; silencing THBS1-AS1 prevents this interaction, reducing the expression of key fibrogenic genes (such as FN1, Serpine1, CTGF, and THBS1) and alleviating biliary fibrosis. In cardiac models, THBS1-AS1 acts as a molecular sponge for miR-221/222, regulating TGFBR1 expression to drive cardiac fibroblast activation.
- Other names
- THBS1-AS1 ASOTHBS-1-AS1 ASOTHBS 1-AS1 ASOTHBS1-AS1 antisense RNA 1 antisense oligonucleotideTHBS-1-AS1 antisense RNA 1 antisense oligonucleotideTHBS 1-AS1 antisense RNA 1 antisense oligonucleotide
02
Targets
THBS1-AS1 (Thrombospondin 1 antisense RNA 1)
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