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.
Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!
GET STARTEDWhat is it?
STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.
What's included?
- Daily reporting and analysis
- The most comprehensive industry coverage from a powerhouse team of reporters
- Subscriber-only newsletters
- Daily newsletters to brief you on the most important industry news of the day
- STAT+ Conversations
- Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
- Exclusive industry events
- Premium access to subscriber-only networking events around the country
- The best reporters in the industry
- The most trusted and well-connected newsroom in the health care industry
- And much more
- Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
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