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loop region mutant angiotensinogen adeno-associated virus
- Development stage
- Preclinical
- Lead developer
- University of Kentucky
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Loop region mutant angiotensinogen adeno-associated virus is an experimental gene therapy construct designed to express a modified version of the angiotensinogen (AGT) protein. The mutations are specifically located within a highly conserved loop region on the surface of the AGT molecule, which is spatially distal to the N-terminal site where renin cleaves AGT to produce angiotensin I. This AAV vector is primarily used in preclinical research to investigate the renin-independent functions of AGT, particularly its interaction with the endocytic receptor megalin (LRP2). Research suggests that the loop region, potentially acting in synergy with the AGT β-sheet region, regulates plasma AGT concentrations and influences Angiotensin II-mediated pathophysiological functions such as hypertension and atherosclerosis. The construct is typically delivered via hepatocyte-specific expression to study systemic effects in AGT-deficient models.
- Other names
- AAV-loop-mutant-AGTAAV-AGT-loop-mutantAAV encoding loop region mutant angiotensinogen
02
Targets
AGT (O6-methylguanine-DNA methyltransferase)
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