Safety of gene therapy and insertional mutagenesis research
Safety of gene therapy and insertional mutagenesis research unit The mission of the safety of gene therapy and insertional mutagenesis research unit is to unravel the basic rules that regulate the genotoxic potential of gene therapy vectors in vivo and to study the interactions between integrated vector sequences and the host genome and its impact on gene expression and mRNA structure. Moreover, taking advantage of the wide tissue tropism, broad integration pattern of HIV-derived lentiviral vectors (LV) we develop a new type of replication-defective insertional mutagen based on LV constructs specifically tailored to efficiently deregulate genes upon genomic integration. These new tools allow forward genetics screenings to identify the molecular culprits of cell transformation by mapping the genomic integration sites in cancer cells. The acquired knowledge will be important to further improve the safety profile of gene therapy vectors and to unravel basic aspects of gene regulation, cell transformation and to identify novel cancer genes. This lab provides a top ranking research training experience to undergraduate fellows, PhD Students and Postdoctoral Fellows.