Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders ...

A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing mutations. The technology could advance treatments for cystic fibrosis and ...

A new genome editing tool—minimal versatile genetic perturbation technology (mvGPT)—can achieve simultaneous and orthogonal gene editing and gene regulation in human cells. The coming together of ...

For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these mutations cause serious diseases that pass down only through mothers. They ...

For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...

Sherry Gao, Tyler Daniel (pictured) and their coauthors developed a new tool that can simultaneously and independently edit multiple genes and regulate their expression. Influential inventions often ...

When it comes to challenges in the genome editing space, all eyes are on delivery. Efficient editing of cells directly in an organism (rather than ex vivo) and targeting of relevant stem cells is ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of "genetic surgery" is performed on DNA, for ...