Impact of Chemical Composition on the Nanostructure and Biological Activity of α-Galactosidase-Loaded Nanovesicles for Fabry Disease Treatment.

Autor:	Tomsen-Melero J; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Nanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Passemard S; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., García-Aranda N; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Drug Delivery and Targeting, and Functional Validation and Preclinical Research (FVPR), CIBBIM-Nanomedicine, Vall d'Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain., Díaz-Riascos ZV; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Drug Delivery and Targeting, and Functional Validation and Preclinical Research (FVPR), CIBBIM-Nanomedicine, Vall d'Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain., González-Rioja R; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Nedergaard Pedersen J; Interdisciplinary Nanoscience Center (iNANO), Aarhus University, DK-8000 Aarhus C, Denmark.; Department of Chemistry, Aarhus University, Langelandsgade 140, DK-8000 Aarhus C, Denmark., Lyngsø J; Interdisciplinary Nanoscience Center (iNANO), Aarhus University, DK-8000 Aarhus C, Denmark.; Department of Chemistry, Aarhus University, Langelandsgade 140, DK-8000 Aarhus C, Denmark., Merlo-Mas J; Nanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain., Cristóbal-Lecina E; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Corchero JL; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Departament de Genètica i de Microbiologia, Institut de Biotecnologia i de Biomedicina (IBB), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain., Pulido D; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Cámara-Sánchez P; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Drug Delivery and Targeting, and Functional Validation and Preclinical Research (FVPR), CIBBIM-Nanomedicine, Vall d'Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain., Portnaya I; CryoEM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel., Ionita I; CryoEM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel., Schwartz S Jr; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Drug Delivery and Targeting, CIBBIM-Nanomedicine, Vall d'Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain., Veciana J; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Sala S; Nanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain., Royo M; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Córdoba A; Nanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain., Danino D; CryoEM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel.; Faculty of Biotechnology and Food Engineering, Guangdong Technion-Israel Institute of Technology, Daxue Road, Shantou 515063, Guangdong Province, China., Pedersen JS; Interdisciplinary Nanoscience Center (iNANO), Aarhus University, DK-8000 Aarhus C, Denmark.; Department of Chemistry, Aarhus University, Langelandsgade 140, DK-8000 Aarhus C, Denmark., González-Mira E; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Abasolo I; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Drug Delivery and Targeting, and Functional Validation and Preclinical Research (FVPR), CIBBIM-Nanomedicine, Vall d'Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain., Ventosa N; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, Campus UAB, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red-Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.
Jazyk:	angličtina
Zdroj:	ACS applied materials & interfaces [ACS Appl Mater Interfaces] 2021 Feb 24; Vol. 13 (7), pp. 7825-7838. Date of Electronic Publication: 2021 Feb 14.
DOI:	10.1021/acsami.0c16871
Abstrakt:	Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to overcome these problems is the use of nanocarriers for encapsulating the enzyme. Nanoliposomes functionalized with RGD peptide have already emerged as a good platform to protect and deliver GLA to endothelial cells. However, low colloidal stability and limited enzyme entrapment efficiency could hinder the further pharmaceutical development and the clinical translation of these nanoformulations. Herein, the incorporation of the cationic miristalkonium chloride (MKC) surfactant to RGD nanovesicles is explored, comparing two different nanosystems-quatsomes and hybrid liposomes. In both systems, the positive surface charge introduced by MKC promotes electrostatic interactions between the enzyme and the nanovesicles, improving the loading capacity and colloidal stability. The presence of high MKC content in quatsomes practically abolishes GLA enzymatic activity, while low concentrations of the surfactant in hybrid liposomes stabilize the enzyme without compromising its activity. Moreover, hybrid liposomes show improved efficacy in cell cultures and a good in vitro/in vivo safety profile, ensuring their future preclinical and clinical development.
Databáze:	MEDLINE
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