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“a-Galactosidase A Loaded Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration”

Unit 1 of NANBIOSIS, Protein Production Platform (PPP), Unit 3, Synthesis of Peptides, Unit 20, In Vivo Experimental Platform and Unit 6, Biomaterial Processing and Nanostructuring Unit, have jointly developed the research conducted in relation with a CO2-based methodology for the one-step production of protein-nanoliposome conjugates as bio-active nanomaterials with therapeutic interest. The results have been published in Advanced Healthcare Materials: http://www.ncbi.nlm.nih.gov/pubmed/26890358

“a-Galactosidase A Loaded Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration I. Cabrera, I. Abasolo, J. L. Corchero, E. Elizondo,  P. Rivera, E. Moreno, J. Faraudo, S. Sala, D. Bueno, E. González-Mira, M. Rivas, M. Melgarejo, D. Pulido, F. Albericio, M. Royo, A. Villaverde, M. F. García-Parajo, S. Schwartz Jr., N. Ventosa,*, and J. Veciana,*

Lysosomal storage disorders (LSD) are caused by lysosomal dysfunction usually as a consequence of deficiency of a single enzyme required for the metabolism of macromolecules such as lipids, glycoproteins and mucopolysaccharides. For instance, the lack of alpha-Galactosidase A (GLA) activity in Fabry disease patients causes the accumulation of glycosphingolipids in the vasculature leading to multiple organ pathology.

Enzyme replacement therapy (ERT), which is the most common treatment of LSD, exhibits several drawbacks mainly related to the instability and low efficacy of the exogenously administered therapeutic enzyme. In this work, the unprecedented increased enzymatic activity and intracellular penetration achieved by the association of a human recombinant GLA to nanoliposomes functionalized with RGD peptides is reported. Moreover, these new GLA loaded nanoliposomes lead to a higher efficacy in the reduction of the GLA substrate named globotriasylceramide (Gb3) in a cellular model of Fabry disease, than that achieved by the same concentration of the free enzyme. The preparation of these new liposomal formulations by DELOS-SUSP, based on the Depressurization of a CO2-Expanded Liquid Organic Solution, shows the great potential of this CO2-based methodology for the one-step production of protein-nanoliposome conjugates as bioactive nanomaterials with therapeutic interest.

“a-Galactosidase A Loaded Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration”
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Unit 1 of NANBIOSIS certified with ISO 9001: 2008

Unit 1 of NANBIOSIS, Production Platform Protein (PPP) at the Autonomous University of Barcelona, ​​directed by Dra. Neus Ferrer and coordinated by Professor Antonio Villaverde, provides the service of production and purification of recombinant proteins in different systems expression, microbial and non-microbial. This service was audited last November, based on the UNE EN ISO 9001: 2008 AENOR, who has issued a favourable certification report.

The requirements of this standard cover all aspects of management. This certification will expand and consolidate the service  that the Unit 1 of NANBIOSIS is giving to companies and public sector researchers and facilitate their integration in cooperative international projects.

AENOR is a Spanish company, renowned in national and international levels, in the development of standardization and certification activities. It was, in 1996 the first Spanish entity accredited by the National Accreditation Body (ENAC)

Nanbiosis_Unit 1 of NANBIOSIS certified with ISO 9001- 2008
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Researchers from the Folding and Conformational Diseases Group at the Institute of Biotechnology and Biomedicine Group are part of the European project Neuromed to develop molecules and detect biomarkers of this disease before the signs of neurodegeneration become evident.

The Neuromed project is led by the University of Zaragoza and aims to design molecules, drugs and new diagnostic tools early for three neurodegenerative diseases in which defective proteins are involved: Parkinson’s disease and two rare diseases, phenylketonuria and protein TTR Amyloidosis.

The coordination of the Parkinson’s research line will be directed, from the IBB, by Salvador Ventura, principal researcher and director the  Protein Folding and Conformational Diseases group and Professor at the Department of Biochemistry and Molecular Biology at the UAB. Researchers in his group are also involved in the project.

The goal of Professor Ventura and his collaborators is focused on developing a diagnostic kit that will allow early and sensitive detection of the presence of Parkinson’s biomarkers in blood and cerebrospinal fluid, in such a way  that the treatment of the disease can begin before the signs of neurodegeneration are obvious.

Neuromed strikes a common element of the three diseases to be investigated: their conformational defects. The research will look for new molecules that can even recover the activity of defective proteins. The combination of computational and biophysical techniques to identify and develop compounds that are tested in cell and animal models would allow the development of drugs active on the three diseases, and will contribute to the early diagnosis of Parkinson.

The Consortium Neuromed involves six partners from Spain, Portugal and France. The research groups, Including the one of professor Ventura, have consolidated expertise in the approach to diagnose and treat these diseases. The project will run for 14 months and has a total cost exceeding EUR 1 million.

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