Browsing by Author "0014640"
Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease. Bird, A; Kemper, Alex Randall; Koeberl, Dwight D; Li, S; Sun, Baodong; Thurberg, BL; Yi, Haiqing (J Gene Med, 2010-11)BACKGROUND: Lysosomal storage disorders such as Pompe disease can be more effectively treated, if immune tolerance to enzyme or gene replacement therapy can be achieved. Alternatively, immune responses against acid α-glucosidase ...
Discontinuation of thyroid hormone treatment among children in the United States with congenital hypothyroidism: findings from health insurance claims data Grosse, SD; Kemper, Alex Randall; Ouyang, L (BMC PEDIATRICS, 2010-02-15)
Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease. Clay, TM; Kemper, Alex Randall; Koeberl, Dwight D; Luo, X; Osada, Takuya; Rodriguiz, Ramona M; Sun, Baodong; ... (9 authors) (Hum Gene Ther, 2012-05)Pompe disease can be treated effectively, if immune tolerance to enzyme replacement therapy (ERT) with acid α-glucosidase (GAA) is present. An adeno-associated viral (AAV) vector carrying a liver-specific regulatory cassette ...