title: Erythrocyte-mediated delivery of recombinant enzymes.
abstract: The possibility to clone, express and purify recombinant enzymes have originated the opportunity to dispose of a virtually infinite array of proteins that could be used in the clinics to treat several inherited and acquired pathological conditions. However, the direct administration of these recombinant proteins faces some intrinsic difficulties, such as degradation by circulating proteases and/or inactivation by the patient immune system. The use of drug delivery systems may overcome these limitations. Concerning recombinant enzyme therapy, the present review will mainly focus on the exploitation of erythrocytes as a carrier system for enzymes removing potentially noxious metabolites from the circulation, either as limiting treatment strategy for auxotrophic tumours or as a detoxing approach for some intoxication type inherited metabolic disorders. Moreover, the possibility of using RBCs as a potential delivering system addressing the enzymes to the monocyte-macrophages of reticular endothelial system for the treatment of diseases associated with this cell lineage, e.g. lysosome storage diseases, will be briefly discussed.
authors.affiliation: Department of Child and Adolescent Neuropsychiatry, SAPIENZA University of Rome, Via deiSabelli 108, 00185, Rome, Italy. vincenzo.leuzzi@uniroma1.it.
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Leuzzi
authors.affiliation: Department of Biomolecular Science, University of Urbino, Via Saffi 2, 61029, Urbino, PU, Italy.
authors.forename: Luigia
authors.initials: L
authors.lastname: Rossi
authors.affiliation: Department of Biomolecular Science, University of Urbino, Via Saffi 2, 61029, Urbino, PU, Italy.
authors.forename: Claudia
authors.initials: C
authors.lastname: Gabucci
authors.affiliation: Department of Child and Adolescent Neuropsychiatry, SAPIENZA University of Rome, Via deiSabelli 108, 00185, Rome, Italy.|Department of Physiology and Pharmacology, SAPIENZA University of Rome, Piazzale Aldo Moro 5, 00185, Rome, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Nardecchia
authors.affiliation: Department of Biomolecular Science, University of Urbino, Via Saffi 2, 61029, Urbino, PU, Italy.
authors.forename: Mauro
authors.initials: M
authors.lastname: Magnani
mesh_terms: D000818:Animals; D004337:Drug Carriers; D016503:Drug Delivery Systems; D056947:Enzyme Replacement Therapy / Q000379:methods*; D004798:Enzymes / Q000008:administration & dosage*; D004912:Erythrocytes / Q000502:physiology*; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy; D011994:Recombinant Proteins / Q000008:administration & dosage*
chemical_list: D004337:Drug Carriers; D004798:Enzymes; D011994:Recombinant Proteins