The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...

Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...

There are 20 cell and gene therapies on Europe's market versus 43 in the U.S. Find out more about this ecosystem.

NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and ...

Similar targeted genetic therapy approaches have been evaluated ... with predicted biological effect within tumor cells, for example cytotoxic-induced tumor cell death due to the activated ...