New Combination Gene Therapy Technique
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New Combination Gene Therapy Technique

As humans age, they develop diseases such as heart failure, kidney failure, diabetes, and obesity, and the presence of any one disease increases the risk of developing others. Conventional drug treatments, however, target one condition. That means patients have to take multiple drugs, increasing both the risk of adverse side effects and the likelihood of forgetting to take the medications on time.

New findings from the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School suggested that it may be possible to tend to multiple ailments with one treatment.

New Combination Gene Therapy Technique

In the Wyss institute research, a single administration of an adeno-associated virus (AAV)-based gene therapy technique, which delivered combinations of three longevity-associated genes to mice, surprisingly improved multiple age-related diseases, suggesting that a systems-level approach to treating such conditions could improve overall health and lifespan.

The research took place in the lab of Wyss core scientist George Church as part of Davidsohn’s postdoctoral research into the genetics of aging. Davidsohn, Church, and team homed in on three genes that demonstrated to confer increased health and lifespan benefits in mice that were genetically engineered to overexpress them, namely- FGF21, sTGFβR2, and αKlotho. The Scientists hypothesized that providing extra copies of those genes to non-engineered mice using the gene therapy technology would combat age-related diseases and bring health benefits.

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The research team created separate gene therapy constructs for each gene using the AAV8 serotype as a delivery vehicle. They injected them into mouse models for obesity, Type 2 diabetes, including heart failure, and renal failure. This was done in combination with the other genes to see whether there was a positive synergistic effect.

FGF21 caused a complete reversal of weight gain and Type 2 diabetes in obese, diabetic mice following a single gene therapy administration. Its combination with the sTGFβR2 gene reduced kidney atrophy by 75% in mice suffering from renal fibrosis. Heart function in mice with heart failure improved by 58% when they were given the sTGFβR2 gene individually or in combination with either of the other two genes, showing that a combined gene therapy treatment of FGF21 and sTGFβR2 could successfully treat all four age-related conditions.

Interestingly, the injected genes remained separate from the animals’ genetic makeup. It did not modify their DNA, and could not pass to the future generations.

Wyss Founding Director Donald Ingber, who is the Judah Folkman Professor of Vascular Biology at HMS and the Vascular Biology Program at Boston Children’s Hospital, said that the ability to treat several age-related diseases at once using gene therapy offers a potential pathway to make aging a more manageable process.

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Rahul Mishra is a Science enthusiast and eager to learn something new each day. He has a degree in Microbiology and has joined forces with Biotecnika in 2019 due to his passion for writing and science.

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