This trial marks the world's first clinical study of an in vivo CRISPR-Cas ribonucleoprotein (RNP)-based genome editing investigational therapy for TGFBI corneal dystrophy.

About TGFBI Corneal Dystrophy . TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of cornea.

About GEB-101. GEB-101, a wholly owned program of GenEditBio, is a genome editing drug candidate designed as a once-and-done treatment for TGFBI corneal dystrophy.GEB-101 is based on the CRISPR-Cas ...

"Today, on National Sight Day, we are proud and honored to announce that we have initiated the world's first clinical study of an in vivo genome editing investigational therapy for corneal dystrophy.

Chronic reflex sympathetic dystrophy (also called the complex regional pain syndrome) is a painful, disabling disorder for which there is no proven treatment. In observational studies, spinal cord ...

Unbeknown to him, his unusual gait was a symptom of muscular dystrophy, a condition in which muscles weaken over time. Mr Eng, now 37, recalls: “I fell easily and frequently.

The teenager recently ran the Manchester marathon in aid of charity Muscular Dystrophy. The event day was the first time she ran on roads, having trained on the farm, fields and surrounding fells.