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AAV9-circAass
- Development stage
- Preclinical
- Modality
- Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-circAass is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed to deliver and restore the expression of the circular RNA circAass (circular aminoadipate-semialdehyde synthase). In the context of acute kidney injury (AKI), circAass levels are significantly reduced, leading to mitochondrial impairment and renal fibrosis. AAV9-circAass functions through a dual mechanism: in the cytoplasm, it acts as a competing endogenous RNA (ceRNA) by sequestering MIR324-3p, thereby upregulating PINK1 and promoting mitophagy; in the nucleus, it binds to the PGC-1α protein (PPARGC1A) to prevent its ubiquitin-mediated degradation, thus enhancing mitochondrial biogenesis. This restoration of mitochondrial homeostasis reduces tubular epithelial cell apoptosis and inflammation, ultimately mitigating renal fibrosis.
- Other names
- AAV9-circAASSAAV-9-circAASSAAV 9-circAASS
02
Targets
PINK1 (PTEN-induced kinase 1)PPARGC1A (Peroxisome proliferator–activated receptor gamma coactivator 1-alpha)
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