(5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B-Reference-Cited by-同舟云学术

(5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B

Published:2017-01 Issue: Volume:126 Page:160-170
ISSN:0223-5234
Container-title:European Journal of Medicinal Chemistry
language:en
Short-container-title:European Journal of Medicinal Chemistry

Author:

Front Sophie,Biela-Banaś Anna,Burda Patricie,Ballhausen Diana,Higaki Katsumi,Caciotti Anna,Morrone Amelia,Charollais-Thoenig Julie,Gallienne Estelle,Demotz Stéphane,Martin Olivier R.

Funder

Swiss Sanfilippo Foundation

Publisher

Elsevier BV

Subject

Organic Chemistry,Drug Discovery,Pharmacology,General Medicine

Reference35 articles.

1. GM1 gangliosidosis: review of clinical, molecular, and therapeutic aspects;Brunetti-Pierri;Mol. Genet. Metab.,2008

2. GM1 gangliosidosis and Morquio B disease: an update on genetic alterations and clinical findings;Caciotti;Biochim. Biophys. Acta,2011

3. Population analysis of the GLB1 gene in South Brazil;Baiotto;Genet. Mol. Biol.,2011

4. Therapy for lysosomal storage disorders;Beck;IUBMB Life,2010

5. Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders;Parenti;Mol. Ther.,2015

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