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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.
Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients.
The aim of the site is to increase the level of awareness and understanding of genetic testing and genetic counselling among people living with inherited retinal dystrophies, empowering them to make fully informed decisions about their lives, healthcare and family planning.
Progress towards treatments for inherited retinal conditions continues to gather pace and there’s been lots going on in the last few months, with more and more approaches being explored. This round-up gives a flavour of the variety of developments, including plenty that are not specific to a particular genetic fault.
Next month, Anisha and Sheena will take on The Thames Path Challenge. Together, they explain why they decided to join #TeamRetinaUK.
Our monthly e-Newsletter featuring the latest updates from Retina UK.
In January 23-year-old Jake Ternent became the first person with an inherited sight loss condition to be treated in the UK with Luxturna (voretigene neparvovec) for Leber congenital amaurosis (LCA).
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.
Saturday 12 October, 10.30am - 3.30pm - In-person - Come and join the Hampshire group for a visit to the Tudor House and Garden in historic Southampton, followed by a pub lunch.
CRISPR gene editing leads to improvements in vision for people with inherited blindness, clinical trial shows.
