The Belmonte lab’s advanced in vivo Cas9-based epigenetic gene activation system in mice

Salk scientists have created a new version technique of the CRISPR/Cas9 genome editing technology that allows them to activate genes without creating breaks in the DNA, potentially circumventing a major hurdle to using gene-editing technologies to treat human diseases (read more HERE).

To test the method, the researchers used mouse models of , type 1 diabetes and a form of muscular dystrophy. In each case, they engineered their CRISPR/Cas9 system to boost the expression of an endogenous gene that could potentially reverse disease symptoms. In the case of kidney disease, they activated two genes known to be involved in kidney function and observed not only increased levels of the proteins associated with those genes but improved kidney function following an acute injury. For type 1 diabetes, they aimed to boost the activity of genes that could generate insulin-producing cells. Once again, the treatment worked, lowering blood glucose levels in a  of diabetes. For , the researchers expressed genes that have been previously shown to reverse disease symptoms, including one particularly large gene that cannot easily be delivered via traditional virus-mediated gene therapies.

“We were very excited when we saw the results in mice,” adds Fumiyuki Hatanaka, a research associate in the lab and co-first author of the paper. “We can induce gene activation and at the same time see physiological changes.”

Izpisua Belmonte’s team is now working to improve the specificity of their system and to apply it to more cell types and organs to treat a wider range of human diseases, as well as to rejuvenate specific organs and to reverse the ageing process and age-related conditions such as hearing loss and macular degeneration. More safety tests will be needed before human trials, they say (Turning Up Gene Expression to Treat Disease / Cell, December 7, 2017 Vol. 171, Issue 7).

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