Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

著录项

• 期刊名称 The Journal of Biological Chemistry
• 作者

  Joseph C. Chen; Amanda R. Luu; Nathan Wise; Rolando De Angelis; Vishal Agrawal; Linley Mangini; Jon Vincelette; Britta Handyside; Harry Sterling; Melanie J. Lo; Hio Wong; Nicole Galicia; Glenn Pacheco; Jeremy Van Vleet; Alexander Giaramita; Sylvia Fong; Sushmita M. Roy; Chuck Hague; Roger Lawrence; Sherry Bullens; Terri M. Christianson; Alessandra dAzzo; Brett E. Crawford; Stuart Bunting; Jonathan H. LeBowitz; Gouri Yogalingam;

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• 作者单位

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• 年(卷),期 2020(295),39
• 年度 2020
• 页码 13532–13555
• 总页数 24
• 原文格式 PDF
• 正文语种
• 中图分类 分子生物学;
• 关键词

  lysosomal storage disease; gene therapy; unfolded protein response (UPR); toxicity; biophysics; beta-galactosidase; cation-independent mannose-6-phosphate receptor; enzyme replacement therapy (ERT); lysosome; neurodegeneration; endoplasmic reticulum stress; safety; GM1 gangliosidosis;

  机译：溶酶体储存疾病;基因治疗;展开蛋白质反应（UPR）;毒性;生物物理学;β-半乳糖苷酶;阳离子无关的甘露糖-6-磷酸受体;酶替代疗法（ERT）;溶酶体;神经变性;内质网应力;安全;GM1甘草化症;