Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism-MedSci.cn

Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism

Kopytova, AE; Rychkov, GN; Nikolaev, MA; Baydakova, GV; Cheblokov, AA; Senkevich, KA; Bogdanova, DA; Bolshakova, OI; Miliukhina, IV; Bezrukikh, VA; Salogub, GN; Sarantseva, SV; Usenko, TC; Zakharova, EY; Emelyanov, AK; Pchelina, SN

Kopytova, AE (corresponding author), Natl Res Ctr, Kurchatov Inst, Petersburg Nucl Phys Inst Named BP Konstantinov, Gatchina, Russia.

Abstract

Mutations in the glucocerebrosidase gene (GBA) encoding the lysosomal enzyme glucocerebrosidase (GCase) cause Gaucher disease (GD) and are the most co......

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Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism

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