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AAV8-pGAFP-SIRT4
- Development stage
- Preclinical
- Lead developer
- Nanjing Drum-Tower Hospital
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-pGAFP-SIRT4 is an adeno-associated virus serotype 8 (AAV8) vector-based gene therapy designed for the targeted overexpression of sirtuin 4 (SIRT4) in hepatic stellate cells (HSCs). Developed by researchers at Nanjing Drum Tower Hospital and Nanjing University, the therapy is designed to treat liver fibrosis. Mechanistically, SIRT4 overexpression in HSCs activates the AMPKα-p53 signaling pathway, leading to the phosphorylation and nuclear translocation of p53. This upregulates the expression of NKG2D ligands (ULBP1 and ULBP2) on HSCs, which enhances their susceptibility to natural killer (NK) cell-mediated recognition and cytotoxicity, ultimately promoting the clearance of activated HSCs and reversing liver fibrosis.
- Other names
- AAV8-pGAFP-Sirt4AAV-8-pGAFP-Sirt4AAV 8-pGAFP-Sirt4HSC-targeted SIRT4 gene therapy
02
Targets
SIRT (Sirtuin family)
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