N of 1 therapy uniquely developed, on demand, for infant with life-threatening rare metabolic disorder in six months Results published in The New England Journal of Medicine and showcased at American ...

It has been described as a revolutionary technology — and won its inventors a Nobel Prize. CRISPR gene editing, often simply dubbed CRISPR, is a tool that allows scientists to precisely target and ...

In a major breakthrough, a patient has been treated with a personalized CRISPR therapeutic that aims to cure their rare genetic disease. This individual, who is known as KJ, was diagnosed with severe ...

A big breakthrough is being hailed in the world of gene editing, with the first custom-designed treatment using CRISPR technology (NASDAQ:CRSP). The therapy was developed for an infant named KJ ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A group of scientists successfully made a bespoke gene ...

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging. In the latest case, the scientists developed a CRISPR ...

CRISPR is a gene-editing tool that acts like “molecular scissors,” but using it on cancer is complex. The technology’s biggest impact so far is in research labs, helping scientists understand how ...

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

The Influenza A virus (IAV) has been the cause of six major flu pandemics, responsible for 50 to 100 million deaths globally. In the U.S. alone, it is estimated that, despite seasonally updated ...