A schematic showing the cytosine base editing intermediate and the various outcomes that can occur when the UNG protein is active. You may have seen it in the news recently: a baby in Pennsylvania ...

Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene ...

Re “Custom Gene-Editing Treatment Helps Baby in World’s First Case” (front page, May 16): Your article highlighting the remarkable work of Dr. Rebecca Ahrens-Nicklas in developing a bespoke ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...

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 ...

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 ...

A single infusion of an experimental gene-editing drug appears safe and effective for cutting cholesterol, possibly for life, according to a small early study released Saturday. The study, which ...

It may sound unreal, but by just altering one gene could one day help many people to permanently lower their dangerously high cholesterol and blood fat levels. This is the biggest takeaway from the ...

C&EN’s award-winning podcast Inflection Point leans on our 100-year archive to trace headline topics in science today back to their disparate and surprising roots. In each episode, we explore three ...