AAV-PHP.B-GAA

AAV-PHP.B-GAA is a **gene therapy vector** that utilizes the adeno-associated virus variant **AAV-PHP.B** to deliver the human *acid alpha-glucosidase* (GAA) gene for the treatment of Pompe disease. The vector is engineered to achieve robust **systemic delivery and transduction** of both the **central nervous system (CNS)** and **skeletal muscle** following **intravenous injection**. In preclinical models (GAA-knockout mice), a single dose leads to widespread expression of GAA in affected tissues, resulting in normalization of glycogen levels in the brain and heart, and significant reduction in skeletal muscle, effectively preventing both muscular and neurological manifestations of Pompe disease. The GAA transgene in this vector is regulated by a CMV enhancer-chicken β-actin promoter, considering high efficiency and ubiquitous expression[1][2][3][4].

Other names

AAV-PHP.B-GAA