The prevalence of [Hutchinson-Gilford progeria syndrome, or] HGPS is approximately 1 in 20 million, so at any given time, there are approximately 400 children living with progeria worldwide… Now, scientists have identified a potential new treatment approach in mice for treating the rare genetic condition.
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The researchers used mice with HGPS that produced progerin and inactivated the [isoprenylcysteine carboxymethyltransferase, or] ICMT gene, to observe how their health was affected. They found the mice that lacked ICMT survived significantly longer, and had higher body weights, compared to unmodified mice with progeria. They also had larger skeletal muscle fibers, that resembled those of healthy mice. Their findings are significant as cardiovascular problems are the main cause of mortality in children with HGPS.
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“Our study has taken important steps in validating ICMT as a potential drug target that could provide advantages over existing treatments for children with this fatal condition,” concluded senior author Martin Bergo, professor at the department of biosciences and nutrition, Karolinska Institute.
Further studies are now needed to find compounds that can target ICMT in living organisms, not just in cells, the researchers said. These findings may lead to the development of new effective treatments.
