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AAV9-VASH1
- Development stage
- Preclinical
- Lead developer
- Fukushima Medical University
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-VASH1 is a gene therapy construct utilizing an adeno-associated virus serotype 9 (AAV9) vector to deliver and overexpress the Vasohibin 1 (VASH1) gene. VASH1 is a tubulin carboxypeptidase that mediates the detyrosination of alpha-tubulin, a post-translational modification that is significantly elevated in failing cardiomyocytes. In preclinical research conducted at Fukushima Medical University, AAV9-VASH1 is used to induce cardiomyocyte-restricted overexpression of VASH1 in mouse hearts to study the pathological consequences of microtubule modification. Research indicates that VASH1-mediated alpha-tubulin detyrosination suppresses Parkin-mediated mitophagy and impairs mitochondrial function, leading to the development of heart failure with preserved ejection fraction (HFpEF). This construct serves as a critical tool for validating alpha-tubulin detyrosination as a potential therapeutic target for HFpEF and other cardiac conditions.
- Other names
- AAV9-Vasohibin 1AAV-9-Vasohibin 1AAV 9-Vasohibin 1
02
Targets
Tyr-tubulin (Alpha-tubulin C-terminal tyrosine)
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