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

Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...

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

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

To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

America's next wave of scientific talent may come from Lambert High School, where students used CRISPR to develop a promising ...

Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable ...

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

Lucy Landman was born with a very rare genetic disorder that causes severe intellectual disability, weak muscles and seizures, among other symptoms. "She is expected to very much never be able to live ...

Philadelphia, October 28, 2024 – CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy. However, until now, the ...

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 ...

Discover why Crispr Therapeutics AG is a strong buy with FDA-approved Casgevy, a promising pipeline, and major growth ...