Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.
Publication
, Journal Article
Yi, H; Gao, F; Austin, S; Kishnani, PS; Sun, B
Published in: Mol Genet Metab Rep
December 2016
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.
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Published In
Mol Genet Metab Rep
DOI
ISSN
2214-4269
Publication Date
December 2016
Volume
9
Start / End Page
31 / 33
Location
United States
Related Subject Headings
- 3202 Clinical sciences
- 3105 Genetics
- 0604 Genetics
- 0601 Biochemistry and Cell Biology
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APA
Chicago
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Yi, H., Gao, F., Austin, S., Kishnani, P. S., & Sun, B. (2016). Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. Mol Genet Metab Rep, 9, 31–33. https://doi.org/10.1016/j.ymgmr.2016.09.008
Yi, Haiqing, Fengqin Gao, Stephanie Austin, Priya S. Kishnani, and Baodong Sun. “Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.” Mol Genet Metab Rep 9 (December 2016): 31–33. https://doi.org/10.1016/j.ymgmr.2016.09.008.
Yi H, Gao F, Austin S, Kishnani PS, Sun B. Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. Mol Genet Metab Rep. 2016 Dec;9:31–3.
Yi, Haiqing, et al. “Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.” Mol Genet Metab Rep, vol. 9, Dec. 2016, pp. 31–33. Pubmed, doi:10.1016/j.ymgmr.2016.09.008.
Yi H, Gao F, Austin S, Kishnani PS, Sun B. Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. Mol Genet Metab Rep. 2016 Dec;9:31–33.
Published In
Mol Genet Metab Rep
DOI
ISSN
2214-4269
Publication Date
December 2016
Volume
9
Start / End Page
31 / 33
Location
United States
Related Subject Headings
- 3202 Clinical sciences
- 3105 Genetics
- 0604 Genetics
- 0601 Biochemistry and Cell Biology