Rational Design and Synthesis of Highly Potent Pharmacological Chaperones for Treatment of N370S Mutant Gaucher Disease
2009
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 wild-type β-glucocerebrosidase but 3- to 6-fold increases in mutant enzyme activity (N370S).
Keywords:
- Correction
- Source
- Cite
- Save
- Machine Reading By IdeaReader
30
References
62
Citations
NaN
KQI