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antisense oligonucleotides targeting circPVT1
- Development stage
- Preclinical
- Lead developer
- Istituto Romagnolo per lo Studio dei Tumori Dino Amadori
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
Antisense oligonucleotides (ASOs) targeting circPVT1 are experimental therapeutic agents designed to downregulate the circular RNA isoform of the Plasmacytoma Variant Translocation 1 (PVT1) gene. circPVT1, specifically the 410bp isoform hsa_circ_0001821, is frequently overexpressed in various cancers, including acute myeloid leukemia (AML), where it acts as an oncogene. By silencing circPVT1, these ASOs induce apoptosis in leukemic cells, reduce MYC protein expression, and modulate pathways related to DNA damage response (DDR) and immune signaling. Research indicates that circPVT1 knockdown can sensitize AML cells to ATM and ATR inhibitors, suggesting potential for combination therapies in hematological malignancies.
- Other names
- circPVT1 ASOscircPVT-1 ASOscircPVT 1 ASOsantisense oligonucleotides targeting hsa_circ_0001821
02
Targets
PVT1 (Plasmacytoma variant translocation 1)
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