Project Profile

The objective of the project is the enhancement of advanced technological platforms for the development of genetic and pharmacological therapies for genetic and degenerative diseases. These platforms integrate high-content "screening" skills, computational biology, molecular, cellular, genetic and clinical biology. The enhancement work concerns three different components which are directly derived from the three TIGEM research programmes (Cellular Biology, Gene Therapy and Computational Biology) and which integrate and reciprocally enhance:

A) High Content Screening (HCS) Platforms on cellular models for the identification of pharmacological targets and active principles, for the identification of chemical compositions which improve the effectiveness of transduction and tissue specificity of viral vectors, and also for the characterisation of the biological activity compositions of synthetic or natural origin, not necessarily of a pharmacological nature.

B) Computational biology approach both to identify pharmacological therapies based on small molecules and to support the HCS in the discovery of new drugs and pharmacological targets, and in the identification of active compositions which optimise the transduction of viral vectors.

C) Platform for the production of adeno-associated viruses (AAV) to use both for human gene therapy and within the scope of preclinical research (Research Grade, AAV-RG) and validation of viruses produced in animal models.

A point of reference at national and international level for research on genetic diseases, TIGEM currently includes a body of 180 people, including several Italian and foreign researchers who have obtained more than 700 international scientific publications, identifying 25 genes responsible for rare genetic diseases. For Andrea Ballabio at the head of Tigem, there are three pillars on which the research centre is based: "passion, professionalism and merit".

The training project tied to enhancement foresees the objective of developing didactic and high-level training of professional figures highly specialised in the field of human gene therapy, and to spread the scientific culture also through agreements with Universities and other training organisations. On 31.12.2012 TIGEM was able to increase the existing endowment of tools for the components indicated, and the training courses of the 9 selected trainees were launched.

In recent years TIGEM has played a first-level role in research funded by the EU and has been part of thirteen consortia, six of which were coordinated and managed by TIGEM. Furthermore, thanks to the centre's capacity to attract funding and researchers even from abroad, TIGEM represents a significant opportunity for development and growth for Italy and the south.