Fluorous iminoalditols act as effective pharmacological chaperones against gene products from GLB1 alleles causing GM1-gangliosidosis and Morquio B disease

Fluorous Iminoalditols: A New Family of Glycosidase Inhibitors and Pharmacological Chaperones

(2010) Georg Schitter et al.   CHEMBIOCHEM

Phenotype determining alleles in GM1 gangliosidosis patients bearing novel GLB1 mutations

(2010) D Hofer et al.   CLINICAL GENETICS

DLHex-DGJ, a novel derivative of 1-deoxygalactonojirimycin with pharmacological chaperone activity in human GM1-gangliosidosis fibroblasts

(2010) Katrin Fantur et al.   MOLECULAR GENETICS AND METABOLISM

Hydrophobic tail length, degree of fluorination and headgroup stereochemistry are determinants of the biocompatibility of (fluorinated) carbohydrate surfactants

(2009) Xueshu Li et al.   COLLOIDS AND SURFACES B-BIOINTERFACES

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

(2009) Giancarlo Parenti   EMBO Molecular Medicine

GM1 gangliosidosis and Morquio B disease: expression analysis of missense mutations affecting the catalytic site of acid β-galactosidase

(2009) Doris Hofer et al.   HUMAN MUTATION

The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines

(2009) E. R. Benjamin et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

(2009) Richie Khanna et al.   MOLECULAR THERAPY

1-Deoxygalactonojirimycin-lysine hybrids as potent d-galactosidase inhibitors

(2008) Andreas J. Steiner et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Chemical chaperone therapy for GM1-gangliosidosis

(2008) Y. Suzuki   CELLULAR AND MOLECULAR LIFE SCIENCES

Lysosomal Storage Diseases and the Blood-Brain Barrier

(2008) David Begley et al.   CURRENT PHARMACEUTICAL DESIGN

The potential action of galactose as a “chemical chaperone”: Increase of beta galactosidase activity in fibroblasts from an adult GM1-gangliosidosis patient

(2008) Anna Caciotti et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

GM1 gangliosidosis: Review of clinical, molecular, and therapeutic aspects

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR GENETICS AND METABOLISM

Synthesis and biocompatibility evaluation of fluorinated, single-tailed glucopyranoside surfactants

(2008) Xueshu Li et al.   NEW JOURNAL OF CHEMISTRY

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity

(2007) Jian-Qiang Fan   BIOLOGICAL CHEMISTRY