Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion
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- 作者：Jose Condoria ; 1 ; 2 ; Walter Acostaa ; 1 ; Jorge Ayalaa ; 1 ; Varun Kattaa ; Ashley Florya ; Reid Martina ; b ; Jonathan Radina ; Carole L. Cramera ; b ; cramer@biostrategies-lc.com" class="auth_mail" title="E-mail the corresponding author ; David N. Radina
- 关键词：GM1 ; GM1-gangliosidosis ; β-gal ; acid β-galactosidase ; LSD ; lysosomal storage disease ; ERT ; enzyme replacement therapy ; RTB ; B-subunit of ricin ; BBB ; blood brain barrier ; CNS ; central nervous system ; PPCA ; protective protein/cathepsin A ; Neu1 ; neuraminidase
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：117
- 期：2
- 页码：199-209
- 全文大小：1377 K
文摘
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The RTB plant lectin is proposed as a carrier for lysosomal replacement enzymes.
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Plant-made β-gal:RTB fusions retain galactosidase and lectin-binding activities.
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β-gal:RTB reduces lysosomal GM1 ganglioside levels in GM1 patient fibroblasts.
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β-gal:RTB undergoes lysosomal β-gal proteolytic activation in GM1 fibroblasts.
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Dynamics of GM1 correction of β-gal:RTB differs from mammalian cell-derived β-gal.