AAV9-VEGF

AAV9-VEGF is a **gene therapy** that uses an **adeno-associated virus serotype 9 (AAV9) vector** to deliver and express the gene for **vascular endothelial growth factor (VEGF)** in target tissues. VEGF is a multifunctional protein primarily involved in promoting angiogenesis, endothelial cell proliferation, and tissue vascularization. AAV9-VEGF has been studied preclinically for a variety of conditions, including Duchenne muscular dystrophy (DMD), left ventricular hypertrophy (LVH), and ischemic cardiovascular diseases. - In muscular dystrophy models, AAV9-VEGF gene transfer resulted in increased VEGF levels, improved muscle strength, reduced inflammation, and lower serum creatine kinase, indicative of decreased muscle damage[2]. - In cardiac hypertrophy and failure models, AAV9-VEGF-B conferred **cardioprotection** by inducing angiogenesis, inhibiting apoptosis, reducing myocardial workload (as reflected by lower ANP expression), enhancing mitochondrial biogenesis, promoting fatty acid utilization, and promoting cardiomyocyte proliferation[1]. - The AAV9 capsid is **cardiotropic and myotropic**, allowing for efficient targeting of muscle and heart tissues[1][2]. - Potential risks include pathologic angiogenesis and rare vascular tumor formation, as reported in other AAV-VEGF vector studies (though this may depend on vector design and dosing)[5]. The therapy remains investigational and has not received regulatory approval.

Other names

adeno-associated virus serotype 9 vascular endothelial growth factorrecombinant AAV9-VEGFAAV9-VEGF gene therapyAAV-9-VEGF gene therapyAAV 9-VEGF gene therapy