Light activated gene therapy shows early promise for inherited blindness
Retinitis pigmentosa is an inherited disease that slowly takes away vision. Because it can be caused by changes in more than 100 different genes, traditional gene therapies only help a small share of patients, and other treatments do not exist. Optogenetics takes a different route by making remaining cells in the eye respond to light, regardless of which gene caused the disease. A research highlight in Nature Medicine describes a small study in patients who were blind from advanced disease. The therapy appeared safe, and early signals of effectiveness were promising, while also pointing to technical improvements still to make. It is an early but hopeful sign for a condition with very few options.