Objectives and scientific workpackages
The PIPgen research program is organized in 5 complementary Scientific Work Packages (WPs 5-9) based on defined molecular processes. WP5-6 are focused on monogenic rare diseases with PI3K/PTEN alterations. WP7-8 stem from the study of cancer. WP9 is a transversal package that will produce innovative tools to be applied in WP5-8.
The scientific objectives:
- Growth (WP5) the study of the molecular mechanisms that cause developmental overgrowth disorders (PIK3CA-related overgrowth disorders (PROS) and PTEN-hamartoma tumour syndrome (PHTS)).
- Immunodeficiency (WP6) the study of the impact of PI3K mutations on immunity.
- Cancer-Intrinsic (WP7) to understand the molecular and therapeutic relevance of PI3K pathway alterations in cancer cells.
- Cancer -Extrinsic (WP8) to investigate the impact of PI3K pathway alterations in the non-cancerous cells inhabiting the tumour.
- Tools (WP9) to identify candidate processes to be studied in the other WPs and to generate small molecule modulators to stimulate translational research and drug development.