BioCentury | Sep 25, 2014
Distillery Therapeutics

Indication: Endocrine/metabolic disease

...DnaJ homolog subfamily B member 11 (DNAJB11; ERdj3); glucocerebrosidase (GBA; GCase) Mouse studies suggest inhibiting ERdj3...
...a lysosomal storage disorder caused by loss-of-function mutations in GCase. In patient-derived fibroblasts, siRNA against ERdj3...
...of disease-associated mutant GCase and slowed its degradation compared with control siRNA. In patient-derived fibroblasts, ERdj3...
Items per page:
1 - 1 of 1