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.

Erythrocyte-mediated delivery of recombinant enzymes / Leuzzi, Vincenzo; Rossi, Luigia; Gabucci, Claudia; Nardecchia, Francesca; Magnani, Mauro. - In: JOURNAL OF INHERITED METABOLIC DISEASE. - ISSN 0141-8955. - STAMPA. - (2016). [10.1007/s10545-016-9926-0]

Erythrocyte-mediated delivery of recombinant enzymes

LEUZZI, Vincenzo
Primo
;
ROSSI, Luigia
Secondo
;
NARDECCHIA, FRANCESCA
Penultimo
;
2016

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.
2016
genetics; metabolic disease; therapeutical approach
01 Pubblicazione su rivista::01a Articolo in rivista
Erythrocyte-mediated delivery of recombinant enzymes / Leuzzi, Vincenzo; Rossi, Luigia; Gabucci, Claudia; Nardecchia, Francesca; Magnani, Mauro. - In: JOURNAL OF INHERITED METABOLIC DISEASE. - ISSN 0141-8955. - STAMPA. - (2016). [10.1007/s10545-016-9926-0]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/869257
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