This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...

Biotech startup CrisprBits uses CRISPR-based techniques to change the shape of diagnostics by decoding genetic sequencing ...

A rare disease mom and scientist shares on the "First Opinion Podcast" how she serves as a translator between her two ...

For years, Yale researchers David Breslow and Mustafa Khokha have worked together with a similar challenge in their ...

The tests have not been approved by federal regulators, but that hasn’t stopped patients from wanting them — and doctors from worrying.

To compete at iGEM, a sort of science Olympics, teens at a Georgia high school set their sights on finding a better way to ...

Drug resistance has long turned some of the most advanced lung cancer therapies into temporary victories, with tumors learning to shrug off chemotherapy that once held them in check. A new wave of ...

An experimental gene-editing treatment shows promise for permanently lowering levels of cholesterol and triglycerides, possibly helping cut the... CRISPR gene-editing works to reduce high cholesterol ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...