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AAV9-TIMP-1
- Development stage
- Preclinical
- Lead developer
- Christian-Albrechts-Universität zu Kiel
- Modality
- Gene Therapies
- Administration
- Intraperitoneal, Intravenous
01
Overview
AAV9-TIMP-1 is an experimental adeno-associated virus serotype 9 (AAV9) gene therapy encoding tissue inhibitor of metalloproteinases-1 (TIMP-1). It is designed to overexpress TIMP-1 in vascular tissues, particularly endothelial cells, to restore extracellular matrix balance and inhibit matrix metalloproteinase (MMP) activity. In preclinical models of Marfan syndrome, AAV9-TIMP-1 (often coated with G2CNN nanoparticles for endothelial retargeting) has been shown to preserve elastin fiber architecture, reduce elastic fiber fragmentation, and attenuate aortic root dilatation, thereby preventing aortic aneurysm progression.
- Other names
- AAV-TIMP-1AAV-TIMP1AAV-TIMP 1AAV9-TIMP1AAV-9-TIMP1AAV 9-TIMP1G2CNN-AAV9-TIMP-1G-2CNN-AAV9-TIMP-1G 2CNN-AAV9-TIMP-1
02
Targets
MMP2 (Matrix metalloproteinase-2)MMP9 (Matrix metalloproteinase-9)TIMP1 (Metalloproteinase inhibitor 1)
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