The revolutionary gene-editing technique, CRISPR, seems to be curing a patient of a deadly inherited disease, showing promise in additionally curing many other common illnesses.
“This is a major milestone for patients,” says Jennifer Doudna of the University of California, Berkeley, who shared a Nobel Prize for her work in helping develop CRISPR. “While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place.”
Through the CRISPR gene-editing technique, researchers were able to make very precise changes to the DNA. They then infused the gene-editor in the patient, suffering from transthyretin amyloidosis, and let it find its way to the right gene in the right cell. Transthyretin amyloidosis is an inherited disease characterized by a misshapen protein that builds up and destroys important tissues such as nerves. The first-of-its-kind experimental treatment proved to have worked as levels of the destructive protein dropped. This technique could become vital for illnesses such as heart disease, muscular dystrophy, and brain diseases like Alzheimer’s.