Article
Potential Binding Sites of Pharmacological Chaperone NCGC00241607 on Mutant β-Glucocerebrosidase and Its Efficacy on Patient-Derived Cell Cultures in Gaucher and Parkinson's Disease.
International journal of molecular sciences - 22 May 2023
Kopytova Alena E, Rychkov George N, Cheblokov Alexander A, Grigor'eva Elena V, Nikolaev Mikhail A, Yarkova Elena S, Sorogina Diana A, Ibatullin Farid M, Baydakova Galina V, Izyumchenko Artem D, Bogdanova Daria A, Boitsov Vitali M, Rybakov Akim V, Miliukhina Irina V, Bezrukikh Vadim A, Salogub Galina N, Zakharova Ekaterina Y, Pchelina Sofya N, Emelyanov Anton K
Abstract excerpt
Mutations in the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase), cause Gaucher disease (GD) and are the most common genetic risk factor for Parkinson's disease (PD). Pharmacological chaperones (PCs) are being developed as an alternative treatment approach for GD and PD. To date, NCGC00241607 (NCGC607) is one of the most promising PCs. Using molecular docking and molecular dynamics simulation...
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