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- W2789917827 abstract "Lysosomal storage diseases (LSDs) are a broad class of over 70 genetic diseases that are caused by mutations in proteins critical for lysosomal function; collectively these diseases have an estimated prevalence of 1 in 5000 live births. LSDs can be treated by enzyme replacement therapy (ERT) in which the purified recombinant enzyme is delivered intravenously to patients by weekly infusions. Plant-based platforms for ERT drug production are being pursued; these may enable more economical and safe treatments than traditional production platforms. Yet the use of plant platforms poses significant technical challenges – one of which is associated with generating a therapeutically efficacious product. The administered recombinant enzyme must have suitable targeting signals for endocytosis into patient cells and for intracellular delivery to the lysosome. For many enzymes this requires the mannose-6-phosphate (M6P) tag on the replacement protein. Elaboration of this trafficking motif requires post-translational enzymatic machinery that is not present in plant cells. We discuss approaches to effect M6P elaboration onto plant-made recombinant lysosomal hydrolases, as well as emerging alternative modifications for improved biodistribution of ERT." @default.
- W2789917827 created "2018-03-29" @default.
- W2789917827 creator A5028534140 @default.
- W2789917827 creator A5065806505 @default.
- W2789917827 creator A5087597856 @default.
- W2789917827 date "2018-03-09" @default.
- W2789917827 modified "2023-09-26" @default.
- W2789917827 title "Plant Recombinant Lysosomal Enzymes as Replacement Therapeutics for Lysosomal Storage Diseases" @default.
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