Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

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

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 million people worldwide.

Scientists in the polo-obsessed nation have produced world's first genetically edited horses, designed to outrun champion ...

A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 gene helps CAR-T cells ...

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

Martin Friedlander, MD, PhD, discusses phase 3 macular telangiectasia findings, highlighting the potential for a decade-long ...

That’s important because thousands of mutations across more than 50 genes have been associated with RP, so a gene therapy that targets just one mutation would only work in a subset of patients.

Scientists have developed an innovative method for growing brain tumors of individual patients in the laboratory that mimic the original structure and the molecular property of the parental tumor as ...