Immunomodulating gene therapy

摘要

The present invention relates, in general, to Pompe disease and, in particular, to a methods of treating Pompe disease and to compounds/constructs suitable for use in such methods.

主权项

1. A method of enhancing the efficacy of human GAA in a Pompe disease patient comprising: (i) administering to said patient a therapeutic amount of human acid α-glucosidase (GAA) enzyme replacement therapy (ERT) produced in Chinese hamster ovary cells, and (ii) administering to said patient an immunosuppressive, subtherapeutic dose of an AAV type8 pseudotyped vector comprising a nucleic acid sequence encoding human GAA operably linked to a liver specific promoter comprising the sequence of SEQ ID NO:1, said vector being administered under conditions such that immune tolerance to said human GAA administered in step (i) does not induce an adverse immune response by said patient and the therapeutic efficacy of said human GAA administered in step (i) is thereby enhanced.