Article
N-glycan structures and downstream mannose-phosphorylation of plant recombinant human alpha-L-iduronidase: toward development of enzyme replacement therapy for mucopolysaccharidosis I.
Plant molecular biology - 1 Dec 2017
Pierce Owen M, McNair Grant R, He Xu, Kajiura Hiroyuki, Fujiyama Kazuhito, Kermode Allison R
Abstract excerpt
KEY MESSAGE: Arabidopsis N-glycan processing mutants provide the basis for tailoring recombinant enzymes for use as replacement therapeutics to treat lysosomal storage diseases, including N-glycan mannose phosphorylation to ensure lysosomal trafficking and efficacy. Functional recombinant human alpha-L-iduronidase (IDUA; EC 3.2.1.76) enzymes were generated in seeds of the Arabidopsis thaliana...
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