Gene therapies I want to see developed
Gene therapies I'd love to see developed/tested in my lifetime:
1. daf-2 - Downregulation increases lifespan in c.elegans
Next steps: RNAi and/or Crispr to cause daf-2 homolog (FOXO3) downregulation in mice + tests on resulting phenotype.
2. PEPCK-C - Beta-actin promoter linked to pepck gene shown to improve longevity, intelligence metabolism in mice.
Next steps: Lentiviral transfection vector as a longevity vaccine (develop in mouse ageing model with human gene)
3. m-CAT - Mitochondrial targeted catalase : upregulation shown to improve cancer resistance and longevity
Next steps: Develop transfection vector as a cancer vaccine (develop in mouse ageing model with human gene)
4. HAS2 - Molecular weight and production of hyaluron decreases over time. Organisms with large amounts of HMWHA live longer and resist cancer.
Next steps: Test regular intravenous injection of HMWHA in rat models of cancer and aging. Develop gene therapy intervention to increase HAS2 expression.
5. TERT - TERT+ mice live longer, but also produce more tumors. HAS2, m-CAT and PEPCK-C all interfere with tumorgenesis. The upregulation of TERT while simultaneously increasing m-CAT and HAS2 expression should have a strong synergistic effect.
Next steps: Develop gene therapy intervention to increase TERT expression, matching prior experiments, but combine with other interventions.