Journal:Acta Cryst D:S205979832000501X
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<b>Molecular Tour</b><br> | <b>Molecular Tour</b><br> | ||
| - | + | β-glucocerebrosidase (GBA) is a lysosomal enzyme which catalyses the hydrolysis of glycosphingolipids. Inherited deficiencies in GBA cause an accumulation of glycolipids in various cells throughout the body, subsequently leading to the most common lysosomal storage disorder Gaucher Disease (GD). Lysosomal storage disorders are a group of 50 rare inherited metabolic disorders which result from abnormal lysosomal function. GD affects multiple organs, such as the heart, kidneys, brain and liver, resulting in debilitating symptoms which severely impact an individuals quality of life and reduces their life expectancy. | |
Due to its involvement in GD, GBA is of considerable medical interest with continuous advances in the development of inhibitors, chaperones and activity-based probes for therapeutic and diagnostic applications. The development of novel GBA inhibitors requires a source of active protein, however, the majority of structural and mechanistic studies on this enzyme rely on expired clinical formulations which are incredibly costly and difficult to obtain in adequate supply.This problem is further compounded by a lack on consensus on a reliable platform for GBA production for research purposes.There is thus a need for an alternative source of GBA to meet research demands and reduce the reliance upon clinical formulations. As part of our long-standing interest in the development of activity-based probes to study GBA and other glycosidases, we established a baculovirus-insect cell expression system for the production of GBA. | Due to its involvement in GD, GBA is of considerable medical interest with continuous advances in the development of inhibitors, chaperones and activity-based probes for therapeutic and diagnostic applications. The development of novel GBA inhibitors requires a source of active protein, however, the majority of structural and mechanistic studies on this enzyme rely on expired clinical formulations which are incredibly costly and difficult to obtain in adequate supply.This problem is further compounded by a lack on consensus on a reliable platform for GBA production for research purposes.There is thus a need for an alternative source of GBA to meet research demands and reduce the reliance upon clinical formulations. As part of our long-standing interest in the development of activity-based probes to study GBA and other glycosidases, we established a baculovirus-insect cell expression system for the production of GBA. | ||
Revision as of 11:21, 22 April 2020
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