ABOUT THE PROJECT
This project aims to evaluate the use of precision medicine to target three pathways altered due to the loss of tumor suppressor genes (TSGs) recurrently mutated in MPNSTs (NF1, CDKN2A/B, PRC2) bridging basic, pre- clinical and clinical research. For the development of the three subprojects we will use: genomic and bioinformatic analysis of MPNSTs; CRISPR editing of TSGs in an induced pluripotent stem cell (iPSC)-based tumor model; drug co-treatment for TSG loss in an MPNST patient-derived orthotopic xenograft platform; pilot individual co-clinical assays using avatars; liquid biopsy for MPNST monitoring.
Present work will clarify the contribution of the three most recurrently lost TSGs (NF1, CDKN2A/B, PRC2) to MPNST tumorigenicity. In addition, we will obtain pre-clinical information on the pharmacological targeting of altered pathways upon their loss. Different co-clinical individual assays performed in parallel in human and mouse avatars will provide clinical-utility information on the use of drugs directed against the loss of these TSGs. We will evaluate the possibility of using liquid biopsy to detect and monitor MPNSTs. Finally, a repository of clinical, genomic, tumorigenic properties and treatment response will be built to gain predictive capacity for the treatment of new developing MPNSTs.
Funding
La Marató
Contact
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