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HMGA2 shRNA
- Development stage
- Preclinical
- Lead developer
- Johns Hopkins University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
HMGA2 shRNA is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of High Mobility Group AT-Hook 2 (HMGA2), a non-histone transcriptional modulator and epigenetic modifier. HMGA2 is frequently overexpressed in various cancers, including glioblastoma multiforme (GBM) and diffuse intrinsic pontine glioma (DIPG), where it promotes tumor invasion, stemness, and proliferation. By utilizing short hairpin RNA (shRNA) delivered via viral vectors (such as lentivirus), this agent triggers the degradation of HMGA2 mRNA, thereby reducing protein levels and inhibiting the oncogenic pathways driven by HMGA2. Research conducted at Johns Hopkins University has demonstrated that HMGA2 knockdown leads to decreased tumor growth and increased survival in orthotopic mouse models of high-grade gliomas.
- Other names
- HMGA2 short hairpin RNAHMGA-2 short hairpin RNAHMGA 2 short hairpin RNAshort hairpin RNA targeting HMGA2
02
Targets
HMGA2 (High mobility group AT-hook 2)
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