By the time Toby Willis arrived at Children's Hospital Los Angeles in March 2018 to receive a first-of-its-kind gene therapy treatment, he had lost most of his eyesight to the inherited eye disease retinitis pigmentosa. Willis, 44, a software engineer for Expedia in Seattle, could only see shapes and shadows. He'd given up driving 20 years earlier, and while he could get along on foot with a cane and his seeing-eye dog, his remaining eyesight was deteriorating.

Then Willis learned from a geneticist that his disease was caused by a rare mutation in the gene RPE65 and that it could be treated with a new gene therapy surgically delivered into each eye. The therapy, called Luxturna, involves inserting a functional copy of RPE65 that takes over for the faulty gene, producing a protein vital for proper vision. While he wouldn't regain all his sight, doctors told him he might recoup enough to significantly improve his quality of life. He signed up right away for the new therapy at Children's Hospital LA – one of the first hospitals equipped to provide it.

Read more at US News.