ArticleNature communications2012
Production of α-L-iduronidase in maize for the potential treatment of a human lysosomal storage disease.
Article in Nature communications, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 28 citations in OpenAlex.
- Plant molecular farming: a promising frontier for orphan drug production.Biotechnology letters · 2025Review
- Expression of Tailored α-N-Acetylglucosaminidase inBioengineering (Basel, Switzerland) · 2025Article
- Improving yield of a recombinant biologic in a Brassica hairy root manufacturing process.Biotechnology and bioengineering · 2022Article
- Contemporary therapeutics and new drug developments for treatment of Fabry disease: a narrative review.Cardiovascular diagnosis and therapy · 2021Review
- Glyco-Engineering of Plant-Based Expression Systems.Advances in biochemical engineering/biotechnology · 2021Article
- Toward Engineering the Mannose 6-Phosphate Elaboration Pathway in Plants for Enzyme Replacement Therapy of Lysosomal Storage Disorders.Journal of clinical medicine · 2019Article
- Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.Cellular and molecular life sciences : CMLS · 2019Review
- Brassica rapa hairy root based expression system leads to the production of highly homogenous and reproducible profiles of recombinant human alpha-L-iduronidase.Plant biotechnology journal · 2019Article
- 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 · 2017Article
- Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Advanced drug delivery reviews · 2017Review
- Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice.Journal of inherited metabolic disease · 2016Article
- RTB Lectin: a novel receptor-independent delivery system for lysosomal enzyme replacement therapies.Scientific reports · 2015Article
- The dynamic behavior of storage organelles in developing cereal seeds and its impact on the production of recombinant proteins.Frontiers in plant science · 2014Review
- Characterization and downstream mannose phosphorylation of human recombinant α-L-iduronidase produced in Arabidopsis complex glycan-deficient (cgl) seeds.Plant biotechnology journal · 2013Article
- Insights into mucopolysaccharidosis I from the structure and action of α-L-iduronidase.Nature chemical biology · 2013Article
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Authors and funding
10 authors at 6 institutions in 4 countries.
Funding
Abstract
Lysosomal storage diseases are a class of over 70 rare genetic diseases that are amenable to enzyme replacement therapy. Towards developing a plant-based enzyme replacement therapeutic for the lysosomal storage disease mucopolysaccharidosis I, here we expressed α-L-iduronidase in the endosperm of maize seeds by a previously uncharacterized mRNA-targeting-based mechanism. Immunolocalization, cellular fractionation and in situ RT-PCR demonstrate that the α-L-iduronidase protein and mRNA are targeted to endoplasmic reticulum (ER)-derived protein bodies and to protein body-ER regions, respectively, using regulatory (5'- and 3'-UTR) and signal-peptide coding sequences from the γ-zein gene. The maize α-L-iduronidase exhibits high activity, contains high-mannose N-glycans and is amenable to in vitro phosphorylation. This mRNA-based strategy is of widespread importance as plant N-glycan maturation is controlled and the therapeutic protein is generated in a native form. For our target enzyme, the N-glycan structures are appropriate for downstream processing, a prerequisite for its potential as a therapeutic protein.
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