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AAV6-shMfap4
- Development stage
- Preclinical
- Lead developer
- Beijing Friendship Hospital
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV6-shMfap4 is a preclinical adeno-associated virus serotype 6 (AAV6) vector-based gene therapy designed to deliver short hairpin RNA (shRNA) targeting microfibrillar-associated protein 4 (MFAP4) [3352]. Developed by researchers at Beijing Friendship Hospital and Capital Medical University, the therapy is designed to selectively silence Mfap4 expression in activated hepatic stellate cells (HSCs) under the control of either a U6 or a glial fibrillary acidic protein (GFAP) promoter [3352]. By suppressing MFAP4, a key extracellular matrix protein upregulated in advanced liver fibrosis, AAV6-shMfap4 aims to attenuate liver fibrogenesis, reduce intrahepatic inflammation, and downregulate fibrosis-specific matrisome genes, offering a potential therapeutic strategy for liver fibrosis [3352].
- Other names
- GFAPsiMfap4GFAPsiMfap-4GFAPsiMfap 4U6siMfap4U-6siMfap4U 6siMfap4
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