Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness.
People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight.
The experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot. It’s intended as a onetime treatment that permanently alters the person’s native DNA.
My daughter can see only light an can identify colours after she was.three years.she is now 26 yrs old.
Please advise how/ where recruitment is. Our son was diagnosed with Lebers Amaurosis at 4 months old. He is now 35 years old, lives and works in Los Angeles. Thank you, Teresa Marositz- parent