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.

Measurements of the curvature of the cornea of the eye can be significantly affected by the status of dry eye in a patient, according to a study published in Frontiers in Medicine. 1 Different ...

Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants ...

Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene ...

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 ...