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Journal of Inherited Metabolic Disease

Erschienen in:

30.03.2016 | SSIEM 2015

Erythrocyte-mediated delivery of recombinant enzymes

verfasst von: Vincenzo Leuzzi, Luigia Rossi, Claudia Gabucci, Francesca Nardecchia, Mauro Magnani

Erschienen in: Journal of Inherited Metabolic Disease | Ausgabe 4/2016

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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.

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Titel: Erythrocyte-mediated delivery of recombinant enzymes
verfasst von: Vincenzo Leuzzi
Luigia Rossi
Claudia Gabucci
Francesca Nardecchia
Mauro Magnani
Publikationsdatum: 30.03.2016
Verlag: Springer Netherlands
Erschienen in: Journal of Inherited Metabolic Disease / Ausgabe 4/2016
Print ISSN: 0141-8955
Elektronische ISSN: 1573-2665
DOI: https://doi.org/10.1007/s10545-016-9926-0

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