β-半乳糖苷酶相关 G-神经节苷脂贮积症和莫尔奎氏 B 型黏多糖贮积症的药物伴侣：最新进展。
Pharmacological Chaperones for β-Galactosidase Related to G -Gangliosidosis and Morquio B: Recent Advances.
机构信息
Glycogroup, Institute of Chemistry and Technology of Biobased Systems, Graz University of Technology, Stremayrgasse 9, A-8010, Graz, Austria.
出版信息
Chem Rec. 2021 Nov;21(11):2980-2989. doi: 10.1002/tcr.202100269. Epub 2021 Nov 23.
PMID:34816592
Abstract
A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for G -gangliosidosis and Morquio B associated mutants of human lysosomal β-galactosidase is provided highlighting recent developments in this particular area of lysosomal storage disorders and orphan diseases.
摘要
提供了一份关于选定的β-半乳糖苷酶抑制剂作为潜在的药理学伴侣用于 G-神经节苷脂贮积症和 Morquio B 相关的人溶酶体β-半乳糖苷酶突变体的简短调查，突出了溶酶体贮积症和孤儿病这一特定领域的最新进展。
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