lentiviral vector encoding human GAA

A lentiviral vector encoding human GAA is a gene therapy designed to treat Pompe disease, a lysosomal storage disorder caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme. This therapeutic approach utilizes a lentiviral vector to deliver a functional copy of the human GAA gene into target cells, typically hematopoietic stem and progenitor cells (HSPCs). The aim is to enable these cells to produce the missing GAA enzyme, thereby reducing the pathological accumulation of glycogen in various tissues, including cardiac and skeletal muscle, and the central nervous system. The therapy also seeks to induce immune tolerance to the GAA protein, which can be a significant challenge with traditional enzyme replacement therapy (ERT). Some advanced versions of these vectors incorporate specific tags, such as IGF2 or GILT, to enhance the cellular uptake and overall efficacy of the expressed enzyme. AVROBIO is a company involved in the development of such a vector for Pompe disease.

Other names

HSPC-mediated lentiviral gene therapy with human GAA