Gene Therapy Can Reverse Congenital Blindness
According to Scientific American, scientists have demonstrated that by using gene therapy, congenital blindness, which affects thousands of people worldwide, can be cured.
Congenital blindness is an inherited retinal disease where people are born with bad vision and then over time, slowly lose their sight. Sometimes they can only see the shape of an object, and even then only in bright sunlight.
Jean Bennett and Katherine A. High are using gene therapy to use a normal copy of the disease-inducing gene, RPE65, to help fix vision. The delicate procedure involves injecting the vector into cells on the back of the retina. They also created a mobility test to help measure changes in vision. The FDA has since approved the therapy, and Bennett and High also received approval from the European Commission in 2018.
The journey to help those living with the RPE65 mutation is far from over. Now, High and Bennett are looking for a way to standardize the practice of genetic screening for retinal diseases, as many families struggle to diagnose their children with the disease.