Gene and Cell Therapy Group
- Public Engagement
- Our research programme
- Jobs and Studentships
- Our research in the media
- Contact Us
- Find Us
Phase I/II trial to assess safety and efficacy of viral RPE65 gene therapy
Phase I/II trial to assess safety and efficacy of viral RPE65 gene therapy for Leber congenital amaurosis
We began the world's first gene therapy trial in the eye in 2007.
The trial is to test the safety and efficacy of gene therapy using an adeno-associated virus (AAV) to deliver the RPE65 gene to retinal cells in patients who have Leber congenital amaurosis (LCA).
Further patients have been treated, and the latest arm of the study includes younger patients.
The encouraging results from this trial suggest that gene therapy can be an effective treatment for inherited retinal degeneration, and extensive evidence from laboratory research shows that other diseases that lead to blindness may also be treatable using gene therapy.
Each of these therapies requires the development of a new gene therapy vector and independent safety testing
Visit the EyeTherapy information hub for more details on:
Pipeline of gene therapy treatments for sight loss
The results of this trial indicate that gene therapy in the eye is safe and can be effective at treating sight loss.
We are developing a pipeline of gene therapy strategies for a range of other conditions that cause sight loss.
Page last modified on 15 jul 14 12:50