Gene Therapy

Harvard study advances gene therapy in fighting age-related diseases

Posted on 29 November 2019

We’re focusing on the reversal of age-related diseases so we’ll be more healthy and youthful later in life.

exclaims Dr. George Church, a notable Harvard professor and leading figure in aging research. 

Dr. Church’s lab has been trialling a ‘one-off’ injection of three genes including; fibroblast growth factor (FGF21), ɑklotho and a soluble form of mouse transforming growth factor-β receptor 2 (sTGFβR2). These have all previously shown association with longevity to reduce the effects of age-related diseases in mice. The addition of these genes amplify levels of proteins in the body that are found to be diminished with age.

The therapy was found to significantly improve kidney and heart function in mice, completely reversing failure of these organs in a proportion of the population. 

A similar therapy was able to treat heart failure, type 2 diabetes, obesity and renal failure, demonstrating the power of gene therapy to treat a multitude of conditions in one fell swoop.

Phenotype exhibited by mice on an HFD with control vector (Right) and AAV:FGF21 vector (Left)

The next research stage is trials of the combination therapy in dogs, which will take roughly two years, after which human clinical trials can begin. Even If everything goes smoothly, Church says the product could take over a decade to get to market. The silver lining being that by this time, hopefully, the price of gene therapy will have dramatically fallen from ~$1 million per patient which it sits at now, to something a little bit more reasonable. 


References

  1. Noah Davidsohn, Matthew Pezzone et al. A single combination gene therapy treats multiple age-related diseases. Proceedings of the National Academy of Sciences of the United States of America, 116, 47, 2019. DOI: 10.1073/pnas.1910073116
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