Base editing approach reverses pathogenic SCN8A mutation in mice, with reduced seizure burden, improved survival and restored neuronal function pointing towards future gene therapies for severe ...

A new study compares the accumulation of mutations across many animal species and has shed new light on decades-old questions about the role of these genetic changes in ageing and cancer. Researchers ...

Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard showed how prime editing can correct the mutation that causes sickle cell disease in a potentially ...

The first study to compare the accumulation of mutations across many animal species has shed new light on decades-old questions about the role of these genetic changes in aging and cancer. Researchers ...

In a step toward treating mitochondrial diseases, researchers in the Netherlands have successfully edited harmful mutations in mitochondrial DNA using a genetic tool known as a base editor. The ...

University of Virginia School of Medicine scientists have used a next-generation form of gene editing to fix the underlying cause of a severe form of epilepsy in lab mice.

Fanconi anemia is a rare genetic disease in which essential DNA repair pathway genes are mutated, disrupting the DNA damage response. Patients with Fanconi anemia experience hematological ...

In the children’s game telephone, a whispered phrase like “I ate a pear” can quickly become “I hate bears” as it moves down a line of players. As genes are passed down from parents to offspring, they ...