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G.P.8.05 The pharmacological chaperone AT2220 increases mutant acid alpha-glucosidase levels and reduces tissue glycogen in a mouse model of Pompe disease

R. Khanna,R. Soksa,J. Feng,Y. Lun,A.C. Powe,J. Flanagan,H. Do,D.J. Lockhart,K.J. Valenzano

NEUROMUSCULAR DISORDERS（2009）

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关键词
pharmacological chaperone at2220,tissue glycogen,alpha-glucosidase
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