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

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,...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10219579/
https://www.ncbi.nlm.nih.gov/pubmed/37240451
http://dx.doi.org/10.3390/ijms24109105

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