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AAV8-sh-SPP1
- Development stage
- Preclinical
- Lead developer
- Nanjing Medical University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-sh-SPP1 is an experimental adeno-associated virus serotype 8 (AAV8) vector-based gene therapy designed to deliver short hairpin RNA (shRNA) targeting secreted phosphoprotein 1 (SPP1, also known as osteopontin or OPN). Developed by academic and government research institutions including Capital Medical University, China Pharmaceutical University, the National Cancer Institute, and Nanjing Medical University, this construct is designed to knock down SPP1 expression in hepatocytes and cholangiocytes. In preclinical models, AAV8-sh-SPP1 has demonstrated efficacy in reducing hepatic SPP1 levels, thereby inhibiting hepatic stellate cell activation and attenuating liver fibrosis in models of congenital hepatic fibrosis (CHF) and metabolic dysfunction-associated steatohepatitis (MASH).
- Other names
- AAV8-Spp1-shRNAAAV-8-Spp1-shRNAAAV 8-Spp1-shRNAAAV8-sh-SPP1AAV-8-sh-SPP1AAV 8-sh-SPP1AAV8-shSpp1AAV-8-shSpp1AAV 8-shSpp1AAV8-U6-shSpp1AAV-8-U6-shSpp1AAV 8-U6-shSpp1scAAV8-U6-shSpp1scAAV-8-U6-shSpp1scAAV 8-U6-shSpp1
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