Eye Gene Therapy
There are more than 300 genes that cause hereditary blinding diseases of the retina. Our expert ophthalmologists and researchers at Children’s Hospital Los Angeles are working to understand those genes. We are improving the eyesight of children with inherited retinal diseases—and we’re doing it with gene therapy.
The Vision Center at CHLA is the first site on the West Coast authorized to deliver the FDA-approved retinal gene therapy, Luxturna (voretigene neparvovec-rzyl) to children. Our team has the unique expertise needed to perform complex gene therapy surgery. When appropriate, we also offer gene therapy through clinical trials.
What Is Gene Therapy for Eye Diseases?
Underlying genetic problems are the cause of many pediatric eye diseases. Faulty or missing genes cannot do their job. When those genes are not corrected, they can lead to blindness.
Gene therapy offers a way to correct genetic mutations. There are many ways gene therapy can correct gene mutations, such as:
- Adding a healthy copy of the gene to replace or help the faulty gene (gene replacement therapy)
- Fixing the defective gene (gene editing)
- Turning off a mutated gene that is not functioning as it should (gene silencing)
How We Use Gene Therapy for Blindness
Our specialists use an FDA-approved gene therapy to treat children who have hereditary retinal disease and a mutation of the RPE65 gene. Our pediatric gene therapy surgeons add a healthy gene to your child’s eye to function in place of the faulty gene. This gene therapy involves two surgeries—one for each eye—performed about a week apart.
Gene therapy surgery requires advanced expertise. Your child’s surgical team includes a:
- pediatric gene therapy surgeon with a unique understanding of retinal disease in children
- pediatric anesthesiologist who understands the effects of anesthesia on children and can manage sedation during complex surgeries
- pharmacist who can prepare and handle the delicate treatments with precision
Who Can Receive FDA-Approved Gene Therapy for Retinal Disease?
Children with inherited retinal disease need an accurate genetic diagnosis to determine whether gene therapy is an appropriate treatment option. The Center for Personalized Medicine at Children’s Hospital Los Angeles performs the genetic testing necessary to identify gene therapy patients quickly and accurately.
If there is no existing gene therapy for your child’s genetic diagnosis, we’ll invite you to participate in our research registry. As new gene therapy treatments become available, we match them to the registry. If your child’s diagnosis matches the treatment, we’ll offer your child the opportunity to participate in clinical trials or receive treatment.
Leading the Way in Ophthalmology Research
Our robust research program is dedicated to curing childhood blindness. We understand the role of gene therapy and how it can benefit children with inherited retinal diseases. Learn more about ophthalmology research at CHLA.
Contact us
To schedule an appointment at the Vision Center, call 323-361-2347.
