Five projects by the Center for Neurosciences and Cell Biology of the University of Coimbra (CNC-UC) will receive funding of around 750,000 euros from the Management Authority of the Center for Regional Coordination and Development (CCDRC), through the CENTRO2020 Operational Program. The global amount will contribute to research related to non-alcoholic fatty liver disease, Machado-Joseph disease and the use of exosomes (small vesicles) for therapeutic purposes.

The project related to non-alcoholic fatty liver disease will be led by Paulo Oliveira, a researcher at CNC-UC. It is called “MitoBOOST v2.0 - Proof of Concept for the Use of Mitochondria Targeted Polyphenols to Treat Non-Alcoholic Fatty Liver Disease” and it intends to carry out a proof of concept concerning the use of a new molecule, AntiOXBEN2, still in pre-clinical phase, for the treatment of this disease, which has been growing in recent decades and affects about a quarter of the world's population. The project is the result of a consortium between CNC-UC, the company Mitotag and the Faculty of Sciences of the University of Porto, which developed the molecule in question.

For the study of exosomes, Lino Ferreira, researcher at CNC-UC, will coordinate the project “BIO-MED: Biomolecular Engineering of Extracellular Vesicles for Regenerative Medicine” which focuses on the use of these small vesicles, naturally existing in the body, as drug delivery systems for therapeutic purposes.

The remaining three projects have a common focus, Machado-Joseph disease, a rare neurodegenerative disease, with prevalence in Portugal, without a cure. Luís Pereira de Almeida, professor at the Faculty of Pharmacy of the University of Coimbra and president of the CNC-UC, coordinates the project “MJDedit - Gene editing systems for the ATXN3 gene: A therapy for Machado-Joseph disease”, which aims to find a therapy for this disease, through gene editing tools. Effective strategies for this disease are potentially applicable to other diseases of the brain.

The project “BDforMJD - Development of a biomarker for Machado-Joseph Disease”, led by CNC-UC researcher Magda Santana, aims to develop a biomarker (biological identifier) ​​to monitor the progression of Machado-Joseph disease and to evaluate the response to therapies in interventional studies.

The project “ModelPolyQ 2.0 - Advanced animal models for polyglutamine diseases”, is coordinated by Rui Jorge Nobre, also a researcher at CNC-UC, and its purpose is to develop cellular and animal models for polyglutamine diseases, such as Machado-Joseph disease, using a kit of viral vectors created at CNC-UC.

The projects are winners of the contest "Notice 01/SAICT/2021 - Reinforce research, technological development and innovation - Proof of Concept Projects (Pdc)" which aims to enhance scientific and technological development activities promoting the translation of science and technology for the society.

Each of the five winning projects lasts approximately one year and can be consulted here.

Carolina Caetano & Catarina Ribeiro