A new CRISPR/Cas9 therapy can suppress aging

Medical Xpress | February 19, 2019

Aging is a leading risk factor for a number of debilitating conditions, including heart disease, cancer, and Alzheimer's disease, to name a few. This makes the need for anti-aging therapies all the more urgent. Now, Salk Institute researchers have developed a new gene therapy to help decelerate the aging process. The findings, published on February 18, 2019, in the journal Nature Medicine, highlight a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated aging observed in mice with Hutchinson-Gilford progeria syndrome, a rare genetic disorder that also afflicts humans. This treatment provides important insight into the molecular pathways involved in accelerated aging, as well as how to reduce toxic proteins via gene therapy. "Aging is a complex process in which cells start to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of aging," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and senior author of the paper. "Progeria is an ideal aging model because it allows us to devise an intervention, refine it and test it again quickly."

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A biomarker is “a characteristic that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention”[1]. Biomarkers are commonly used in the pharmaceutical industry to evaluate the safety and/or effectiveness of drugs. For example, they can be used to assess the binding of drugs to their target, which can be critical.

Spotlight

A biomarker is “a characteristic that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention”[1]. Biomarkers are commonly used in the pharmaceutical industry to evaluate the safety and/or effectiveness of drugs. For example, they can be used to assess the binding of drugs to their target, which can be critical.

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