Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized gene-editing tool,

Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer Jennifer Doudna wants to build an entire ecosystem to bring these treatments mainstream.

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more reliable therapies for a wide range of genetic diseases,

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand design and clinical use of a personalized gene editor for ...

Gene editing has enormous potential to help feed the world’s growing population, but it’s currently difficult, time-consuming, and only works in some plant species. A big part of the problem is CRISPR/Cas9’s size: it’s too large to be delivered into plant cells.

In this webinar, researchers discuss using and improving next-generation gene editing approaches for targeted, accurate, and precise DNA modification.

Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol. It’s very early stage research, tried in only a few dozen people so far.