Peking University scientists have developed a cancer therapy that could make life-saving treatment accessible to any patient, ...

At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved ...

Researchers used evolutionary genetics and CRISPR gene editing tech to develop an innovative treatment for gout. The approach ...

Despite volatility and regulatory headwinds, AMT-130's efficacy and safety profile could position it as a first-in-class, ...

The era of gene therapy is here, at least for a Folly Beach infant. Born with a devastating muscle disease, he got gene ...

Edits create cells that don’t trigger an immune response, allowing implant recipient to forego immune-suppressing drugs.

However, more than a third of melanoma patients given checkpoint inhibitors develop side effects so severe that they compromise their quality of life and ability to continue therapy.

Three changes in total to the genetic code were needed to ensure the transplanted donor cells didn't trigger an immune ...

Side effects most often involve some form of inflammation, a sign of an overactive immune response. Patients can experience severe skin rashes, diarrhea, or hyperthyroidism.

Many gene therapy developers use viral vectors that exhibit specific tissue tropism as delivery vehicles, meaning they preferentially infect certain tissues to deliver the gene therapy cargo.

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development and manufacturing.

A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β-thalassemia.