Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.
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Patients with progressive hepatic form of GSD IV often die of liver failure in early childhood. We tested the feasibility of using recombinant human acid-α glucosidase (rhGAA) for treating GSD IV. Weekly intravenously injection of rhGAA at 40 mg/kg for 4 weeks significantly reduced hepatic glycogen accumulation, lowered liver/body weight ratio, and reduced plasma ALP and ALT activities in GSD IV mice. Our data suggests that rhGAA is a potential therapy for GSD IV.
SubjectALT, alanine aminotransferase
AST, aspartate aminotransferase
ERT, enzyme replacement therapy
GAA, acid α-glucosidase
GBE, glycogen branching enzyme
GSD IV, glycogen storage disease type IV
Glycogen storage disease type IV
M6PR, mannose-6-phosphate receptor
Recombinant human acid-α glucosidase
Published Version (Please cite this version)10.1016/j.ymgmr.2016.09.008
Publication InfoAustin, S; Gao, Feng; Kishnani, Priya Sunil; Sun, Baodong; & Yi, Haiqing (2016). Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. Mol Genet Metab Rep, 9. pp. 31-33. 10.1016/j.ymgmr.2016.09.008. Retrieved from http://hdl.handle.net/10161/15077.
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Professor of Medicine
Dr. Feng Gao is Professor of Medicine at Duke University. The Gao laboratory has a long-standing interest in elucidating the origins and evolution of human and simian inmmunodeficiency viruses (HIV and SIV), and in studying HIV/SIV gene function and pathogenic mechanisms from the evolutionary perspective. These studies have led to new strategies to better understand HIV origins, biology, pathogenesis and drug resistance, and to design new AIDS vaccines.
Chen Family Professor of Pediatrics
RESEARCH INTERESTS A multidisciplinary approach to care of individuals with genetic disorders in conjunction with clinical and bench research that contributes to: 1) An understanding of the natural history and delineation of long term complications of genetic disorders 2) The development of new therapies for genetic disorders through translational research 3) The development and execution of large multicenter trials to confirm safety and efficacy of potential th
Associate Professor of Pediatrics
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and animal disease models. The main therapeutic approaches we are using in our pre-clinical studie
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