AAV-MCKhGAApA

AAV-MCKhGAApA is an **experimental gene therapy** utilizing adeno-associated virus (AAV) as a delivery vector to introduce the human GAA gene under the control of a muscle-specific MCK promoter into target tissues. The GAA gene encodes acid alpha-glucosidase, an enzyme required for glycogen breakdown in lysosomes. By delivering a functional copy of the GAA gene, AAV-MCKhGAApA aims to restore enzymatic activity in patients with Pompe disease (glycogen storage disease type II), thus reducing glycogen accumulation in muscles and improving motor function. The AAV vector achieves targeted long-term gene delivery and expression in muscle cells by leveraging the muscle-specific promoter MCK. This approach is characteristic of gene therapies targeting monogenic neuromuscular disorders.