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2016-09-09

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Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/116163

Highly Versatile Polyelectrolyte Complexes for Improving the Enzyme Replacement Therapy of Lysosomal Storage Disorders

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Recurs relacionat

https://doi.org/10.1021/acsami.6b08356

Resum

Lysosomal storage disorders are currently treated by enzyme replacement therapy (ERT) through the direct administration of the unprotected recombinant protein to the patients. Herein we present an ionically cross-linked polyelectrolyte complex (PEC) composed of trimethyl chitosan (TMC) and α-galactosidase A (GLA), the defective enzyme in Fabry disease, with the capability of directly targeting endothelial cells by incorporating peptide ligands containing the RGD sequence. We assessed the physicochemical properties, cytotoxicity, and hemocompatibility of RGD-targeted and untargeted PECs, the uptake by endothelial cells and the intracellular activity of PECs in cell culture models of Fabry disease. Moreover, we also explored the effect of different freeze-drying procedures in the overall activity of the PECs. Our results indicate that the use of integrin-binding RGD moiety within the PEC increases their uptake and the efficacy of the GLA enzyme, while the freeze-drying allows the activity of the therapeutic protein to remain intact. Overall, these results highlight the potential of TMC-based PECs as a highly versatile and feasible drug delivery system for improving the ERT of lysosomal storage disorders.

Matèries

Errors congènits del metabolisme, Lisosomes, Nanomedicina

Matèries (anglès)

Inborn errors of metabolism, Lysosomes, Nanomedicine

Citació

Col·leccions

Articles publicats en revistes (Ciència dels Materials i Química Física)
Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)
Articles publicats en revistes (Institut de Bioenginyeria de Catalunya (IBEC))

Citació

GIANNOTTI, Marina Inés, et al. Highly Versatile Polyelectrolyte Complexes for Improving the Enzyme Replacement Therapy of Lysosomal Storage Disorders. ACS Applied Materials & Interfaces. 2016. Vol. 8, num. 39, pags. 25741-25752. ISSN 1944-8244. [consulted: 3 of August of 2026]. Available at: https://hdl.handle.net/2445/116163

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