Developing Epigenetic Drugs for Genetic and Regenerative Medicine
Gene editing currently makes long-term changes to DNA sequences as a way of turning genes on or off. For many, however, the potential for permanent genetic changes is concerning. In contrast, a new approach known as “genetic tuning” manipulates the epigenetics of a cell and, in so doing, enables gene expression to be turned up or down.
“Genetic tuning can achieve a much greater dynamic range of effects … not turning a gene on or off, but turning it up or down a little,” Derek Jantz, PhD, chief scientific officer, Tune Therapeutics, tells GEN. “By tuning the epigenome, we can make precisely controlled changes to gene expression in much the same way that our cells do every day. Our cells don’t change their genome. Our cells change their epigenome.”
Basically, our epigenome editing platform, TEMPO, can change the way cells in the body interpret their underlying DNA sequence,” Jantz explains. “That allows us, potentially, to address some very common and very complex diseases that are caused by dysregulation of gene expression.
First data, shared last May at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), showed that in nonhuman primates, TEMPO downregulated the PCSK9 gene. The PCSK9 protein is important in regulating lipid homeostasis. “We now have a group of animals that have been treated and have significantly reduced cholesterol as a result,”
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