Many of the diseases caused by point mutations have no established therapeutic approaches. Prof. Tsukahara and colleagues (Japan Advanced Institute of Science and Technology) are studying a therapeutic method using artificial RNA editing. Artificial site-directed RNA editing is an important technique for modifying genes and ultimately regulating protein function. We are trying to modify the […]
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In a first, researchers use base editing to correct recessive genetic deafness and restore partial hearing to mice
When Wei Hsi (Ariel) Yeh was a young undergraduate student, one of her close friends went from normal hearing to complete deafness in the span of one month. He was 29 years old. No one knew why he lost his hearing; doctors still don’t know. Frustrated and fearful for her friend, Yeh, who graduated last […]
Genome editing helps decipher a congenital liver disease
Congenital hepatic fibrosis (CHF) is a rare genetic disease that causes malformation and fibrosis (scarring) of the liver. Occurring in roughly one out of every 20,000 births, CHF can lead to an enlarged liver, impaired blood flow to the intestines (portal hypertension), infection of the bile ducts and liver failure. In severe cases, a liver […]
A case against a moratorium on germline gene editing
Should researchers put the brakes on genetically engineering babies? Leading scientists and ethicists recently called for a moratorium on clinical applications of germline gene editing: inheritable alterations to the DNA of embryos to improve kids’ health or other features – or just “gene editing,” for short. This declaration was prompted in part by the birth […]
Preventing sudden cardiac death with genome editing
Each year, at least 3 million people worldwide die of sudden cardiac death. In the U.S., this number reaches up to 450,000 people. Although sudden cardiac death is more common in older adults, younger people also are significantly affected. In the 1 to 40 age group, up to 9 per 100,000 people are affected each […]