Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2013

Takai Tomoko, Higaki Katsumi, Aguilar-Moncayo Matilde, Mena-Barragán Teresa, Hirano Yuki, Yura Kei, Yu Liang, Ninomiya Haruaki, García-Moreno M Isabel, Sakakibara Yasubumi, Ohno Kousaku, Nanba Eiji, Ortiz Mellet Carmen, García Fernández José M, Suzuki Yoshiyuki

Abstract excerpt

Lysosomal β-galactosidase (β-Gal) deficiency causes a group of disorders that include neuronopathic GM1 gangliosidosis and non-neuronopathic Morquio B disease. We have previously proposed the use of small molecule ligands of β-Gal as pharmacological chaperones (PCs) for the treatment of GM1 gangliosidosis brain pathology. Although it is still under development, PC therapy has yielded promising preclinical results...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Mucopolysaccharidosis IV
Share to 1-Deoxynojirimycin

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Administration, Oral

Share to beta-Galactosidase

Share to Bridged Bicyclo Compounds, Heterocyclic

Share to Gangliosidosis, GM1

Share to Lysosomes

Share to Molecular Chaperones

Source

Open in PubMed Open full text

Identifiers

PMID: 23337983
DOI: 10.1038/mt.2012.263
PMCID: PMC3589148

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.PMID 23337983Read ↗

Structural basis of pharmacological chaperoning for human β-galactosidase.Semantic proximity Read ↗

The pharmacological chaperone N-n-butyl-deoxygalactonojirimycin enhances β-galactosidase processing and activity in fibroblasts of a patient with infantile GM1-gangliosidosis.Semantic proximity Read ↗

Chaperone activity of bicyclic nojirimycin analogues for Gaucher mutations in comparison with N-(n-nonyl)deoxynojirimycin.Semantic proximity Read ↗

Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.Semantic proximity Read ↗

Molecular basis of 1-deoxygalactonojirimycin arylthiourea binding to human α-galactosidase a: pharmacological chaperoning efficacy on Fabry disease mutants.Semantic proximity Read ↗

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

Synthesis of a New β-Galactosidase Inhibitor Displaying Pharmacological Chaperone Properties for GM1 Gangliosidosis.Semantic proximity Read ↗

Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis.Semantic proximity Read ↗

Probing the Inhibitor versus Chaperone Properties of sp²-Iminosugars towards Human β-Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease.Semantic proximity Read ↗

Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.Semantic proximity Read ↗

New insights into the pharmacological chaperone activity of c2-substituted glucoimidazoles for the treatment of Gaucher disease.Semantic proximity Read ↗

Identification of pyrimidinyl piperazines as non-iminosugar glucocerebrosidase (GCase) pharmacological chaperones.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.