DataVXACS Applied Materials & InterfacesPaper
journal article·2021-02-24

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

Judit Tomsen-Melero, Solène Passemard, Natalia García-Aranda, Zamira Vanessa Díaz-Riascos, Ramon González-Rioja, Jannik Nedergaard Pedersen, Jeppe Lyngsø, Josep Merlo-Mas, Edgar Cristóbal-Lecina, José Luis Corchero, Daniel Pulido, Patricia Cámara-Sánchez, Irina Portnaya, Inbal Ionita, Simó Schwartz, Jaume Veciana, Santi Sala, Miriam Royo, Alba Córdoba, Dganit Danino, Jan Skov Pedersen, Elisabet González-Mira, Ibane Abasolo, Nora Ventosa

ACS Applied Materials & Interfaces· Volume 13· Issue 7
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Source abstract

Abstract

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.
Source: openalex_inverted_index · Quality: baseline

Research context

Nanomaterials

OpenAlex topics: Lysosomal Storage Disorders Research · Carbohydrate Chemistry and Synthesis · Biomedical Research and Pathophysiology

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

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

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

Interdisciplinary Nanoscience Center (iNANO), Aarhus University, DK-8000 Aarhus C, Denmark

Department of Chemistry, Aarhus University, Langelandsgade 140, DK-8000 Aarhus C, Denmark

Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain

Departament de Genètica i de Microbiologia, Institut de Biotecnologia i de Biomedicina (IBB), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain

CryoEM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel

Drug Delivery and Targeting, CIBBIM-Nanomedicine, Vall d’Hebron Institut de Recerca (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain

Faculty of Biotechnology and Food Engineering, Guangdong Technion-Israel Institute of Technology, Daxue Road, Shantou 515063, Guangdong Province, China

Funding

Centro de Investigaci??n Biom??dica en Red en Bioingenier??a, Biomateriales y Nanomedicina · European Commission · Generalitat de Catalunya · Ministerio de Econom??a y Competitividad · Ministerio de Ciencia e Innovaci??n

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