AAV9-MLP

AAV9-MLP is a preclinical gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver and overexpress Muscle LIM Protein (MLP, also known as CSRP3). MLP is a critical component of the Z-disc mechanosensory apparatus in cardiomyocytes, playing an essential role in maintaining cardiac structural integrity and mechanosignaling. In preclinical models of heart failure and myocardial infarction, AAV9-MLP-mediated re-expression of MLP restores the 'sandwich-like' MLP/α-actinin complex topology, leading to the regression of left ventricular mass, reduction of fibrosis, and partial recovery of cardiac function and geometry. This gene therapy is being investigated for its potential to promote cardiac reverse remodeling and enhance functional recovery in heart failure patients, particularly those undergoing mechanical unloading via left ventricular assist devices (LVADs).

Other names

AAV9-mediated CSRP3 gene therapyAAV-9-mediated CSRP3 gene therapyAAV 9-mediated CSRP3 gene therapyAAV9-mediated Muscle LIM Protein gene therapyAAV-9-mediated Muscle LIM Protein gene therapyAAV 9-mediated Muscle LIM Protein gene therapy