Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease
Highly potent N-substituted δ-lactams have been rationally designed and synthesized by a concise route with a one-pot tandem reaction as key step. These iminosugars show weak inhibition of wildtype β-glucocerebrosidase but 3- to 6-fold increases in mutant
Wang, Guan-Nan,Reinkensmeier, Gabriele,Zhang, Si-Wei,Zhou, Jian,Zhang, Liang-Ren,Zhang, Li-He,Butters, Terry D.,Ye, Xin-Shan
supporting information; experimental part
p. 3146 - 3149
(2010/03/03)
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