Multivalent GCase Enhancers: Synthesis and Evaluation of Glyco-Gold Nanoparticles Decorated with Trihydroxypiperidine Iminosugars DOI
Francesca Buco, Francesca Clemente, Amelia Morrone

и другие.

Bioconjugate Chemistry, Год журнала: 2024, Номер unknown

Опубликована: Дек. 19, 2024

The present study reports the preparation of first multivalent iminosugars built onto a glyco-gold nanoparticle core (glyco-AuNPs) capable stabilizing or enhancing activity lysosomal enzyme GCase, which is defective in Gaucher disease. An N-nonyltrihydroxypiperidine was selected as bioactive iminosugar unit and further functionalized, via copper-catalyzed alkyne–azide cycloaddition, with thiol-ending linker that allowed conjugation to gold core. These ligands were obtained either linear monomeric dendritic trimeric arrangement iminosugar. concentration on surface modulated different amounts glucoside bearing short spacer inner ligand. new mixed-ligand coated glyco-AuNPs fully characterized, those highest colloidal stability aqueous medium subjected biological evaluation. Glyco-AuNPs units showed ability stabilize recombinant GCase thermal denaturation assay, while able enhance mutant patient's fibroblasts by 1.9-fold at 2.2 μM.

Язык: Английский

Assessing the Potential of N-Butyl-l-deoxynojirimycin (l-NBDNJ) in Models of Cystic Fibrosis as a Promising Antibacterial Agent DOI Creative Commons
Anna Esposito, Alice S. Rossi,

Maria Rosaria Stabile

и другие.

ACS Pharmacology & Translational Science, Год журнала: 2024, Номер 7(6), С. 1807 - 1822

Опубликована: Май 10, 2024

Over the past few years, l-iminosugars have revealed attractive pharmacological properties for managing rare diseases including Cystic Fibrosis (CF). The iminosugar

Язык: Английский

Процитировано

1
Multivalent GCase Enhancers: Synthesis and Evaluation of Glyco-Gold Nanoparticles Decorated with Trihydroxypiperidine Iminosugars DOI
Francesca Buco, Francesca Clemente, Amelia Morrone

и другие.

Bioconjugate Chemistry, Год журнала: 2024, Номер unknown

Опубликована: Дек. 19, 2024

The present study reports the preparation of first multivalent iminosugars built onto a glyco-gold nanoparticle core (glyco-AuNPs) capable stabilizing or enhancing activity lysosomal enzyme GCase, which is defective in Gaucher disease. An N-nonyltrihydroxypiperidine was selected as bioactive iminosugar unit and further functionalized, via copper-catalyzed alkyne–azide cycloaddition, with thiol-ending linker that allowed conjugation to gold core. These ligands were obtained either linear monomeric dendritic trimeric arrangement iminosugar. concentration on surface modulated different amounts glucoside bearing short spacer inner ligand. new mixed-ligand coated glyco-AuNPs fully characterized, those highest colloidal stability aqueous medium subjected biological evaluation. Glyco-AuNPs units showed ability stabilize recombinant GCase thermal denaturation assay, while able enhance mutant patient's fibroblasts by 1.9-fold at 2.2 μM.

Язык: Английский

Процитировано

1