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Our amazing volunteers are diverse in age, background and ethnicity. The one thing they have in common is that they are all living with, or directly affected by, an inherited sight loss condition.

• Case study - community
• Written

The project aims to develop a cost effective method of examining the entire ABCA4 gene to look for variations that might cause disease.

• Case study - research
• Written

Luxturna, a gene therapy, is only for the treatment of Leber congenital amaurosis type 2 (LCA2) and severe early-onset RP caused by mutations in a specific gene called RPE65.

• FAQ
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The following projects were being funded by Retina UK and have recently come to an end.

Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.

• News
• Usher syndrome

When we talk about gene therapy we are usually referring to use of a harmless virus, called a vector, to deliver a normal copy of a defective gene into the cells of the eye.

Do you want to win up to £25,000 and feel good about supporting people with inherited progressive sight loss?!

Biotechnology company ProQR has announced encouraging results from its early analysis of the phase 1/2 trial of QR-421a, an innovative approach to treating sight loss caused by mutations in a particular section of the USH2A gene.

• News
• Usher syndrome

Bhavini was diagnosed with RP in 1997 aged 17. The consultant who confirmed her diagnosis told her that “there was no cure or treatment and she should prepare to go blind".

• Case study - community
• Written