RTB Lectin: a novel receptor-independent delivery system for lysosomal enzyme replacement therapies

Sci Rep. 2015 Sep 18:5:14144. doi: 10.1038/srep14144.

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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Drug Carriers*
  • Drug Delivery Systems*
  • Enzyme Replacement Therapy
  • Fibroblasts / drug effects
  • Fibroblasts / metabolism
  • Gene Expression
  • Glycosaminoglycans / metabolism
  • Humans
  • Iduronidase / administration & dosage
  • Iduronidase / genetics
  • Iduronidase / metabolism
  • Lectins, C-Type / metabolism
  • Lysosomal Storage Diseases / therapy
  • Mannose Receptor
  • Mannose-Binding Lectins / metabolism
  • Nicotiana / chemistry
  • Phenotype
  • Plant Leaves / genetics
  • Plant Leaves / metabolism
  • Receptor, IGF Type 2 / metabolism
  • Receptors, Cell Surface / metabolism
  • Recombinant Fusion Proteins
  • Ricin* / genetics
  • Ricin* / metabolism

Substances

  • Drug Carriers
  • Glycosaminoglycans
  • Lectins, C-Type
  • Mannose Receptor
  • Mannose-Binding Lectins
  • Receptor, IGF Type 2
  • Receptors, Cell Surface
  • Recombinant Fusion Proteins
  • Ricin
  • Iduronidase