Combined Gene Therapy for the Treatment of Age-related Diseases

New research conducted jointly by the Wyss Institute and Harvard Medical School suggests that multiple age-related diseases can be treated with just therapy.

In this study, a gene therapy based on an adeno-associated virus (AAV) consisting of three “longevity genes” was used in mice, significantly improving or completely eliminating mice from multiple age-related diseases.

Noah Davidson and George Church together with their colleagues focused on three genes associated with increased life expectancy and healthy longevity in mice (FGF21, sTGFβR2, and αKlotho), and modified them to increase their expression. They suggested that introducing these extra gene copies to unmodified mice would have a positive effect on their health.

The team created a separate drug for each gene, using AAV8 as a delivery mechanism and injected it into mice with obesity, type 2 diabetes, heart failure, and kidney failure. They were used individually as well as in combination with others to find out if there is a synergistic effect.

A single dose of FGF21 completely reversed obesity and type 2 diabetes in mice, and its combination with sTGFβR2 reduced renal atrophy by 75 percent in mice with renal fibrosis. Cardiac function in mice with heart failure improved by 58 percent with sTGFβR2 alone or in combination with one of the other genes. Using all three at the same time led to slightly lesser results, most likely due to the interaction of FGF21 and αKlotho, which has not yet been fully studied.

But what is important: these genes did not modify the DNA of the mice!

How cool is that?