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Gene and cell therapy
New science for rare eye diseases.
A small number of inherited and rare eye conditions can now be treated with gene or cell-based therapy given by retina surgeons. An eye exam and genetic testing are the first steps toward finding out whether one might apply to you.
The first approval came in 2017.
The FDA approved the first gene therapy for an inherited eye disease in December 2017, for people with a specific gene change that causes retinal degeneration.
Signs worth mentioning to an eye doctor.
Trouble in the dark
Night vision that is poor or worsening.
Shrinking side vision
Bumping into things at the edges.
Family history
Relatives with inherited vision loss.
Childhood vision loss
Poor vision noticed early in life.
Central blur in midlife
Changes in reading vision or distortion.
Slow dark adaptation
Taking long to adjust to dim rooms.
Genetic testing comes first.
Gene therapy only works for the exact gene change it was designed for, so a confirmed genetic diagnosis is needed before treatment is considered.
A cell-based option arrived in 2025.
An implant containing cells that release a protective protein was approved in 2025 for adults with a rare macular condition called macular telangiectasia type 2.
How treatment is given
Surgery at specialized centers.
These treatments are placed inside the eye by retina surgeons, usually one eye at a time, followed by close monitoring.
Your eye doctor stays involved.
Your optometrist can recognize early signs, arrange testing and imaging, and help with low vision support before and after treatment.
Before your visit.
Gather family history
Who in the family has had vision loss, and when.
Bring past records
Earlier eye exams, imaging or genetic results.
Note daily challenges
Night driving, reading or moving in dim light.
How the path usually unfolds.
Imaging tells the story.
Retinal scans, photos and visual field tests show how the retina is working and help guide testing and referral.
Keep exploring.
Fast answers.
Is gene therapy available for every inherited eye disease?
No. Approved treatments target specific gene changes or conditions. Research continues for others.
How do I find out if I qualify?
Start with a dilated eye exam. Your eye doctor can arrange imaging and genetic testing, then refer you.
Is it a one-time treatment?
The approved therapies are given once per eye, with follow-up visits afterward.
Who performs the treatment?
Retina surgeons at specialized centers.
Start with a thorough eye exam.
Find an eye doctor near you with the Vision Rescue doctor locator.
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Sources and notes
- U.S. FDA: first gene therapy for an inherited eye disease
- National Eye Institute: Retinitis pigmentosa
- Exam-room and showroom photography are owned Eye Care Advisors assets approved for use by Vision Rescue.
- Full source list: Vision Rescue sources
This page is for general education and is not medical advice. Only an eye care professional who has examined you can recommend what is right for your eyes.
