Ontology highlight
ABSTRACT:
SUBMITTER: Han SO
PROVIDER: S-EPMC4303018 | biostudies-literature | 2015 Jan
REPOSITORIES: biostudies-literature
Han Sang-oh SO Li Songtao S Brooks Elizabeth D ED Masat Elisa E Leborgne Christian C Banugaria Suhrad S Bird Andrew A Mingozzi Federico F Waldmann Herman H Koeberl Dwight D
Human gene therapy 20150101 1
Enzyme replacement therapy (ERT) is the standard-of-care treatment of Pompe disease, a lysosomal storage disorder caused by deficiency of acid α-glucosidase (GAA). One limitation of ERT with recombinant human (rh) GAA is antibody formation against GAA. Similarly, in adeno-associated virus (AAV) vector-mediated gene transfer for Pompe disease, development of antibodies against the GAA transgene product and the AAV vector prevents therapeutic efficacy and vector readministration, respectively. Her ...[more]