Therapy1 | Structure

From 2015 to 2020 the group of vectors and Gene therapy will further develop molecular tools, such as viral and non-viral vectors, with a multidisciplinary expertise aiming at investigating ways to create new therapies for neurodegenerative and cancer diseases that can be moved from the bench top to the bedside.

This large group will incorporate 4 new integrated members with complementary expertise, in this way reaching 22 PhD-holding integrated members of which 6 faculty members and a total of 66 members. This considerable critical mass is expected to further increase the impact of the group’s contributions, which over the last 5 years has allowed its members, to identify novel targets and generate advanced therapies for neurodegenerative and cancer disease. These span from alleviation of Machado-Joseph disease and glioblastoma upon gene silencing, microRNA and proteolysis inhibition, or activation of autophagy in the brain, to efficient targeting of tumor cells and tumor regression as well as effective vaccination with nanoformulations. These contributions resulted in 108 publications, of which 62 are in the first quartil, 6 in the top 3% of their area and 15 with impact factor over 7. Moreover, 8 patents, 4 of these licensed, were generated and a spin-off company TREAT U SA was created. Further investment in high impact and open access publications will be fostered and systematic patent submission will be implemented.

To reach its objectives the group will mantain its organization in two main scientific areas: (i) Neurodegenerative diseases and (ii) Cancer diseases, and two main technological approaches: a) Viral vectors and, b) Non-viral vectors/Nanotechnologies. In addition, a structural division into smaller teams, focusing on specific problems (see webpage), will help achieving research milestones, while keeping a broad multidisciplinary expertise within the larger group: a) Mechanisms of membrane fusion, b) targeted lipid-based formulations c) new MicroRNA-based biomarkers and therapeutic targets in AD and cancer d) Nanoformulated vaccines, d) Gene and stem-cell therapy of Machado-Joseph disease. Moreover the group will further reinforce collaboration with the Coimbra University Hospital (CHUC), as well as the Portuguese Institute of Oncology (IPO), in the period 2015-2020 by further attracting hospital members into our team and involving the clinical staff in the research.

The internationalization of the group will be fostered by further investing in new and in the present international networks on which the group is integrated, namely E-rare ERA-net, Marie-Curie iTN networks, as well as the MIT-Portugal Program and collaboration with leading groups abroad either in Europe or USA, such as the University of California, Harvard Medical School, Broad Institute in Cambridge, MIT or MD Anderson Cancer Center.
The past productivity has only been possible with significant funding that the investigators have secured from several international and national agencies such as the Portuguese Foundation for Science and Technology, Marie Curie People Program, National Ataxia Foundation, Association Française de Myopathies, Portuguese Ministry of Economy (QREN) among others, in a total of over 4 million euros. The group expects to continue to secure funding in similar or higher level by further applying to international funding, with a special emphasis on the Horizon 2020 program.