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Carbon nanotubes as nanovectors for intracellular delivery of laronidase in Mucopolysaccaridosis type I

Abstract

The immobilization of proteins on carbon nanotubes (CNTs) has been widely reported mainly for the preparation of sensors while the conjugation of enzymes for therapeutic purposes has been scarcely considered. Herein we report, at the best of our knowledge, the first example of intracellular delivery of a therapeutic enzyme by means of CNTs, retaining its activity. Mucopolisaccharodosis I is a rare genetic disease characterized by a deficiency or absence of activity of the α-L-iduronidase (IDUA) enzyme. We evaluated the capacity of the recombinant form of human IDUA enzyme, laronidase (Aldurazyme®), conjugated with CNTs to be internalized by fibroblasts from subjects affected with Mucopolisaccaridosis type I and the capacity of the enzyme to keep its activity after internalization. The enzyme was successfully delivered into the lysosomal space and the enzymatic activity of the conjugate was preserved after internalization up to 48 hours. This paves the way towards the use of such kind of constructs for therapeutic applications.

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Publication details

The article was received on 04 Oct 2017, accepted on 24 Nov 2017 and first published on 29 Nov 2017


Article type: Paper
DOI: 10.1039/C7NR07393C
Citation: Nanoscale, 2017, Accepted Manuscript
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    Carbon nanotubes as nanovectors for intracellular delivery of laronidase in Mucopolysaccaridosis type I

    T. Da Ros, A. Ostric, F. Andreola, M. Filocamo, M. Pietrogrande, F. Corsolini, M. Stroppiano, S. Bruni, A. Serafino and S. Fiorito, Nanoscale, 2017, Accepted Manuscript , DOI: 10.1039/C7NR07393C

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