| Id |
Subject |
Object |
Predicate |
Lexical cue |
| T1 |
0-74 |
Sentence |
denotes |
Structural basis of pharmacological chaperoning for human β-galactosidase. |
| T2 |
75-305 |
Sentence |
denotes |
GM1 gangliosidosis and Morquio B disease are autosomal recessive diseases caused by the defect in the lysosomal β-galactosidase (β-Gal), frequently related to misfolding and subsequent endoplasmic reticulum-associated degradation. |
| T3 |
306-592 |
Sentence |
denotes |
Pharmacological chaperone (PC) therapy is a newly developed molecular therapeutic approach by using small molecule ligands of the mutant enzyme that are able to promote the correct folding and prevent endoplasmic reticulum-associated degradation and promote trafficking to the lysosome. |
| T4 |
593-793 |
Sentence |
denotes |
In this report, we describe the enzymological properties of purified recombinant human β-Gal(WT) and two representative mutations in GM1 gangliosidosis Japanese patients, β-Gal(R201C) and β-Gal(I51T). |
| T5 |
794-870 |
Sentence |
denotes |
We have also evaluated the PC effect of two competitive inhibitors of β-Gal. |
| T6 |
871-1017 |
Sentence |
denotes |
Moreover, we provide a detailed atomic view of the recognition mechanism of these compounds in comparison with two structurally related analogues. |
| T7 |
1018-1143 |
Sentence |
denotes |
All compounds bind to the active site of β-Gal with the sugar-mimicking moiety making hydrogen bonds to active site residues. |
| T8 |
1144-1366 |
Sentence |
denotes |
Moreover, the binding affinity, the enzyme selectivity, and the PC potential are strongly affected by the mono- or bicyclic structure of the core as well as the orientation, nature, and length of the exocyclic substituent. |
| T9 |
1367-1493 |
Sentence |
denotes |
These results provide understanding on the mechanism of action of β-Gal selective chaperoning by newly developed PC compounds. |
