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AAV9-mCEROD shRNA
- Development stage
- Preclinical
- Lead developer
- The First Affiliated Hospital of USTC
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
AAV9-mCEROD shRNA is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy delivering a short hairpin RNA (shRNA) designed to knock down mouse Calcification-associated EIciRNA Regulator Of DHX36 (mCEROD), an exon-intron circular RNA (EIciRNA). In preclinical models of coronary artery calcification (CAC) and atherosclerosis, upregulation of CEROD promotes the ubiquitination and degradation of DHX36 via recruitment of the E3 ubiquitin ligase STUB1, which in turn stabilizes PAPPA2 mRNA and amplifies pro-calcific signaling. By knocking down mCEROD, AAV9-mCEROD shRNA restores DHX36 levels, downregulates PAPPA2, RUNX2, and BMP2, and reduces aortic plaque burden and calcification, improving vascular remodeling.
- Other names
- AAV9-mediated mCEROD shRNAAAV-9-mediated mCEROD shRNAAAV 9-mediated mCEROD shRNAAAV9-mCEROD-shRNAAAV-9-mCEROD-shRNAAAV 9-mCEROD-shRNA
02
Targets
BMP2 (Bone morphogenetic protein 2)DHX36 (ATP-dependent DNA/RNA helicase DHX36)PAPPA2 (Pregnancy-associated plasma protease A2)RUNX2STUB1 (STIP1 homology and U-box-containing protein 1)CEROD (Calcification-associated EIciRNA Regulator Of DHX36)
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