Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.
Amino-myo-inositol derivatives have been found to be potent inhibitors of glucocerebrosidase (GCase), the β-glucosidase enzyme deficient in Gaucher disease (GD). When tested using lymphoblasts derived from patients with GD homozygous for N370S or L444P mutations, the compounds enhanced GCase activit...
Những tác giả chính: | Trapero, A, González-Bulnes, P, Butters, T, Llebaria, A |
---|---|
Định dạng: | Journal article |
Ngôn ngữ: | English |
Được phát hành: |
2012
|
Những quyển sách tương tự
-
Polyhydroxylated bicyclic isoureas and guanidines are potent glucocerebrosidase inhibitors and nanomolar enzyme activity enhancers in Gaucher cells.
Bằng: Trapero, A, et al.
Được phát hành: (2011) -
Chaperoning glucocerebrosidase: a therapeutic strategy for both Gaucher disease and Parkinsonism
Bằng: Benjamin McMahon, et al.
Được phát hành: (2016-01-01) -
Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease.
Bằng: Wang, G, et al.
Được phát hành: (2009) -
Synthesis of N-substituted ε-hexonolactams as pharmacological chaperones for the treatment of N370S mutant Gaucher disease.
Bằng: Wang, G, et al.
Được phát hành: (2012) -
Progranulin Recruits HSP70 to β-Glucocerebrosidase and Is Therapeutic Against Gaucher Disease
Bằng: Jinlong Jian, et al.
Được phát hành: (2016-11-01)