GAA and glycogen storage disease II: Considering that there is no long-term benefit from ERT in some Pompe disease patients, e.g., patients with high anti-GAA antibodies, and the potential immune responses against AAV9 could be controlled by immunosuppressive regimens which were widely used in gene therapy, the exclusion criteria of preexisting AAV9 NAb could be elevated to 1:200, even to 1:300.