AAV9-tMCK-CN19o

AAV9-tMCK-CN19o is an experimental adeno-associated virus (AAV) gene therapy designed for the targeted inhibition of Calcium/calmodulin-dependent protein kinase II (CaMKII) in skeletal muscle. The construct utilizes an AAV9 capsid for systemic delivery and muscle tropism, with expression of the transgene controlled by the truncated muscle creatine kinase (tMCK) promoter to ensure muscle-specific activity. The transgene, CN19o, is an optimized peptide inhibitor derived from the natural CaM-KIIN protein, which specifically blocks CaMKII activity. Developed to address sarcopenia and age-related muscle dysfunction, the therapy aims to reverse the deleterious effects of chronic CaMKII activation, such as muscle atrophy, mitochondrial disorganization, and impaired contractility, thereby restoring a more youthful physiological and transcriptional profile to aged muscle.

Other names

AAV9-tMCK-CN19AAV-9-tMCK-CN19AAV 9-tMCK-CN19CaMKII inhibitor gene therapy