AAV9-HMGA1

AAV9-HMGA1 is an experimental gene therapy construct that utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver and overexpress the High Mobility Group AT-hook 1 (HMGA1) gene, specifically in cardiac tissue. HMGA1 is an architectural transcription factor that regulates gene expression by modifying chromatin structure. In preclinical research concerning sepsis-induced cardiomyopathy, AAV9-HMGA1 is used as a tool to study the pathological role of HMGA1. Findings indicate that overexpression of HMGA1 via this vector exacerbates myocardial inflammation and cardiomyocyte apoptosis by activating the mitochondrial-dependent apoptotic pathway. Consequently, while AAV9-HMGA1 is a critical research tool for elucidating disease mechanisms, HMGA1 itself is viewed as a therapeutic target for inhibition rather than a candidate for therapeutic overexpression in the context of sepsis.

Other names

Adeno-associated virus 9-HMGA1AAV9-High mobility group protein AT-hook 1AAV-9-High mobility group protein AT-hook 1AAV 9-High mobility group protein AT-hook 1