Summary of the technology
Genetic and Epigenetic Editing for Brain Tumor Control
Project ID : 6-2017-4496
Description of the technology
- Glioblastoma multiforme (GBM) is a common, very aggressive brain cancer with a dismal prognosis, affecting young and middle-age adults with no effective treatment.
- Former efforts to diagnose glioblastoma tumors and tailor personal treatments were so far limited to the coding sequences of the cancer genes, but it is lately become clear that the cancer is due to mutation of gene regulation rather than of gene structure
- The effect of transcriptional enhancers remains unclear due to the lack of an effective way to link enhancers with their controlled genes.
A novel method to control the activity level of key GBM genes and attack the cancer through its regulatory networks (a new class of genomic targets).
Using a novel mapping technology, a list of critical genomic targets (enhancer sites) for GBM driver genes (unique to our lab and not available for others) were identified in human tumors.
The ability to control glioblastoma proliferation by modifying the regulatory targets via Cas-CRISPR editing was demonstrated.
The activity of the targeted genes was tuned by epigenetic editing of the critical regulatory sites without introducing irreversible genetic modifications.
- Better targets – Provide a new world of regulatory targets
- Safer treatment - Expression-related epigenetic modifications are tunable and reversible, therefore side effects are better controlled, and the risk of unwanted permanent effects is much lower.
- Better control - The ability to tune activity level of genes (vs. current gene KO approaches) is revolutionary more effective and provide significantly improved ability for personalized fitting.
- Diagnostic and prognosis kits for brain cancer subtypes.
- Monitoring cancerous tumors during treatment.
- Biochemical measuring tools for molecular diagnostics.
- Genetic and epigenetic therapy tools.
- Indicate existing drugs for additional types of patients.