METHODS AND COMPOSITIONS FOR TREATMENT OF POMPE DISEASE

摘要

Provided herein are chimeric polypeptides comprising a mature acid-alpha glucosidase (GAA) polypeptide and an internalizing moiety. An exemplary internalizing moiety of the application is the 3E10 antibody which is capable of transiting a cellular membrane via an equilibrative nucleoside transporter 2 (ENT2) and binding DNA. Further provided are methods and uses of the said chimeric polypetides for treating Pompe disease, increasing acid alpha-glucosidase enzyme activity in a cell and decreasing glycogen accumulation in the cytoplasm, lysosomes and autophagic vacuoles of cells.