lentiviral vector GAA

Lentiviral vector GAA is an investigational gene therapy modality that utilizes a genetically engineered lentiviral vector to deliver and express the human acid α-glucosidase (GAA) gene in hematopoietic stem and progenitor cells (HSPCs), primarily developed for the treatment of Pompe disease. Pompe disease is caused by a deficiency in the GAA enzyme, resulting in lysosomal glycogen accumulation and progressive myopathy. By introducing a functional copy of the GAA gene via ex vivo transduction of patient HSPCs, the therapy aims to restore endogenous GAA enzyme activity, thereby reducing pathological glycogen accumulation in various tissues including skeletal muscle and the central nervous system. Advanced lentiviral vectors may incorporate peptide tags or codon optimization to enhance lysosomal targeting and secretion, improving therapeutic outcomes. This modality is distinct from enzyme replacement therapies due to improved tissue biodistribution, potential for long-lasting correction, and reduced immunogenicity[3].

Other names

lentiviral GAAGAA lentiviral vectorlentiviral vector-mediated GAA