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Gene Therapy Combo Treats Multiple Age-Related Diseases

Adeno-associated virus

Adeno-associated virus for delivering gene therapies (Jazzlw, Wikimedia Commons)

5 Nov. 2019. A bio-engineering team shows a single experimental gene therapy cocktail can reduce or reverse four age-related diseases in lab mice. Researchers from Harvard University and spin-off company Rejuvenate Bio describe the treatment in yesterday’s Proceedings of the National Academy of Sciences.

A team from the lab of genetics professor George Church at the Wyss Institute for Biologically Inspired Engineering and Harvard Medical School are seeking to solve a continuing problem in the health of older people, the simultaneous occurrence of several serious diseases associated with aging. In most cases, disorders like heart and kidney failure are treated individually, and not always coordinated with type 2 diabetes and obesity treatments. The simultaneous occurrence of these diseases in many older people suggests the need for a simultaneous, coordinated treatment.

The researchers led by Noah Davidsohn, a former research scientist at the Wyss Institute and now chief technology officer at Rejuvenate Bio, are focusing their coordinated treatment on three genes: fibroblast growth factor 21 or FGF21, alpha-Klotho, and a soluble form of transforming growth factor-beta receptor 2 or sTGF-betaR2. These genes are associated with age-related diseases, and previous studies show changing the expression of these genes offers health benefits and longer life spans to genetically engineered mice.

In their study, the team tested gene therapies in lab mice induced with four age-related diseases: obesity, type 2 diabetes, heart failure, and kidney failure. The mice were treated with gene therapies that added extra copies of suspect genes alone or in combination, but the animals were otherwise not genetically engineered. The gene therapies were delivered with adeno-associated virusesbenign and naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, other than at most mild reactions. The researchers point out that the mice genomes were not changed by the treatments, nor are they transferable to future generations.

The results show, for the most part, single-dose therapies transferring one or more of the three genes reduce or reverse the age-related diseases in lab mice. For example, increasing the FGF21 gene alone caused a reversal of type 2 diabetes and weight gain, and with extra sTGF-betaR2 genes reduced kidney damage from fibrosis by 75 percent. More of the sTGF-betaR2 genes also improved heart functioning by 58 percent. A single dose of of FGF21 combined with sTGF-betaR2 genes can simultaneously treat all four diseases. However, a combination therapy that increases the number of all three genes results in somewhat worse outcomes, due to adverse interactions between FGF21 and alpha-Klotho.

“The results we saw were stunning,” says Davidsohn in a Wyss Institute statement, “and suggest that holistically addressing aging via gene therapy could be more effective than the piecemeal approach that currently exists.” Church adds, “This research marks a milestone in being able to effectively treat the many diseases associated with aging, and perhaps could lead to a means of addressing aging itself.”

Davidsohn, Church, and co-author Daniel Oliver founded Rejuvenate Bio last year. The company is developing treatments for age-related diseases, such as heart failure, in dogs, not humans. As noted as recently as September in Science & Enterprise, Church is a serial entrepreneur, founding or licensing discoveries from his labs to dozens of start-up and spin-off enterprises, including Rejuvenate Bio.

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