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shRNA targeting RUNX1
- Development stage
- Preclinical
- Lead developer
- GeneChem Biotechnology
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
shRNA targeting RUNX1 (shRUNX1) is a research-stage RNA interference (RNAi) therapeutic modality designed to silence the expression of the Runt-related transcription factor 1 (RUNX1) gene. Developed as a research tool, notably by GeneChem (Shanghai), it utilizes short-hairpin RNA molecules to induce sequence-specific degradation of RUNX1 mRNA. RUNX1 is a master regulator of hematopoiesis, and its dysregulation or mutation is implicated in various malignancies, including acute myeloid leukemia (AML) and high-grade serous ovarian cancer (HGSOC). Preclinical studies have demonstrated that shRUNX1 can inhibit colony growth and induce apoptosis in AML cells harboring RUNX1 mutations, as well as suppress tumor growth in vivo. It has also been explored in strategies for HIV latency reactivation.
- Other names
- shRNA-mediated knockdown of RUNX1RUNX1 shRNARUNX-1 shRNARUNX 1 shRNA
02
Targets
RUNX1
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