Abstract：
:Enzyme replacement therapies have revolutionized patient treatment for multiple rare lysosomal storage diseases but show limited effectiveness for addressing pathologies in "hard-to-treat" organs and tissues including brain and bone. Here we investigate the plant lectin RTB as a novel carrier for human lysosomal enzymes. RTB enters mammalian cells by multiple mechanisms including both adsorptive-mediated and receptor-mediated endocytosis, and thus provides access to a broader array of organs and cells. Fusion proteins comprised of RTB and human α-L-iduronidase, the corrective enzyme for Mucopolysaccharidosis type I, were produced using a tobacco-based expression system. Fusion products retained both lectin selectivity and enzyme activity, were efficiently endocytosed into human fibroblasts, and corrected the disease phenotype of mucopolysaccharidosis patient fibroblasts in vitro. RTB-mediated delivery was independent of high-mannose and mannose-6-phosphate receptors, which are exploited for delivery of currently approved lysosomal enzyme therapeutics. Thus, the RTB carrier may support distinct in vivo pharmacodynamics with potential to address hard-to-treat tissues.
journal_name
Sci Repjournal_title
Scientific reportsauthors
Acosta W,Ayala J,Dolan MC,Cramer CLdoi
10.1038/srep14144subject
Has Abstractpub_date
2015-09-18 00:00:00pages
14144issn
2045-2322pii
srep14144journal_volume
5pub_type
杂志文章abstract：:Carbonatites, usually occurring within intra-continental rift-related settings, have strong light rare earth element (LREE) enrichment; they rarely contain economic heavy REE (HREE). Here, we report the identification of Late Triassic HREE-Mo-rich carbonatites in the northernmost Qinling orogen. The rocks contain abun...
journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/srep37377
更新日期：2016-11-18 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2015-06-09 00:00:00
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journal_title：Scientific reports
pub_type： 已发布勘误
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pub_type： 杂志文章
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更新日期：2019-02-14 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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pub_type： 杂志文章
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pub_type： 杂志文章
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2016-08-17 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2016-02-05 00:00:00
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pub_type： 杂志文章
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pub_type： 杂志文章
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更新日期：2018-07-19 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2017-08-11 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-020-80005-w
更新日期：2021-01-13 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2017-11-06 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-019-48284-0
更新日期：2019-08-12 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-020-72108-1
更新日期：2020-09-18 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-017-11599-x
更新日期：2017-09-21 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-017-12668-x
更新日期：2017-09-29 00:00:00
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pub_type： 杂志文章
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2015-11-02 00:00:00
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pub_type： 杂志文章
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更新日期：2016-02-23 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章,meta分析
doi：10.1038/srep08510
更新日期：2015-03-02 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-019-46370-x
更新日期：2019-07-08 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/srep05703
更新日期：2014-07-16 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2017-02-17 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/srep01774
更新日期：2013-01-01 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
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更新日期：2016-05-25 00:00:00
abstract：:A correction to this article has been published and is linked from the HTML and PDF versions of this paper. The error has not been fixed in the paper. ...
journal_title：Scientific reports
pub_type： 已发布勘误
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更新日期：2018-08-10 00:00:00
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journal_title：Scientific reports
pub_type： 杂志文章
doi：10.1038/s41598-019-47126-3
更新日期：2019-07-22 00:00:00
