Health
Gene-Editing Experiment Let Patients See Color Again
Carlene Knight’s vision was so bad that she couldn’t even maneuver around the call center where she works using her cane.
“I was bumping into the cubicles and really scaring people that were sitting at them,” says Knight, who was born with a rare genetic eye disease.
But that’s changed as a result of volunteering for a landmark medical experiment. Her vision has improved enough for her to make out doorways, navigate hallways, spot objects and even see colors.
“It’s nice. I don’t scare people and I don’t have as many bruises on my body,” Knight says, laughing.
Knight is one of seven patients with a rare eye disease who volunteered to let doctors modify their DNA by injecting the revolutionary gene-editing tool CRISPR directly into cells that are still in their bodies. Knight and one other study volunteer gave NPR exclusive interviews about their experience.
This is the first time researchers worked with CRISPR this way. Earlier experiments had removed cells from patients’ bodies, edited them in the lab and then infused the modified cells back into the patients.
On Wednesday, researchers revealed the first evidence that the approach appears to be working — improving vision for at least some patients with the condition, known as Leber congenital amaurosis, or LCA, a severe form of vision impairment.
“It’s a really amazing technology and very powerful,” says Dr. Mark Pennesi, professor of ophthalmology at the Casey Eye Institute at the Oregon Health & Science University. He presented the results at an International Symposium on Retinal Degeneration in Nashville, Tenn.