AAV9.CAG.BiP.vIGF2.hGAAco.rBG.KanR

AAV9.CAG.BiP.vIGF2.hGAAco.rBG.KanR is a recombinant gene therapy vector based on the adeno-associated virus serotype 9 (AAV9) platform. It is engineered to deliver a gene cassette encoding several elements: BiP (a molecular chaperone), a variant insulin-like growth factor 2 (vIGF2), a codon-optimized human acid alpha-glucosidase (hGAAco), and regulatory sequences (CAG promoter, rBG polyadenylation signal), with a Kanamycin resistance gene (KanR) for bacterial selection. The construct is designed for broad and efficient gene expression, likely targeting neurological and/or metabolic disorders where GAA enzyme deficiency is implicated, such as Pompe disease. The AAV9 vector enables widespread tissue transduction, including the central nervous system, due to its ability to cross the blood-brain barrier. The specific modality and arrangement suggest a gene replacement or enzyme augmentation approach, possibly combined with additional protective or supportive factors (BiP, vIGF2). No commercial products or late-stage trials for this exact construct are referenced in the current clinical literature[1][3][4].