Gene therapies I want to see developed
Posted 10:58 AM

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.
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​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)
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​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)
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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.

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