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Drug intelligence / Profile preview
AAV9-YBX1
- Development stage
- Preclinical
- Lead developer
- National Institute of Diabetes and Digestive and Kidney Diseases
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-YBX1 is an adeno-associated virus serotype 9 (AAV9) gene therapy vector designed to overexpress Y-box binding protein 1 (YBX1). YBX1 is a multifunctional RNA/DNA-binding protein that acts as a 5-methylcytosine (m5C) and N6-methyladenosine (m6A) reader to stabilize mRNA and promote protein translation. In preclinical models of focal segmental glomerulosclerosis (FSGS), delivery of AAV9-YBX1 alleviated kidney injury, reduced proteinuria, and improved renal function by regulating synaptopodin mRNA and mitigating glomerulosclerosis. Additionally, AAV9-YBX1 has been investigated in preclinical models of myocardial infarction and diabetic cardiomyopathy, where it promotes angiogenesis, reduces fibrosis, and improves cardiac function.
- Other names
- AAV9-Ybx1AAV-9-Ybx1AAV 9-Ybx1adeno-associated virus 9-YBX1adeno-associated virus serotype 9-YBX1
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