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Biopharmaceutical company ProQR is conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat Leber's Congenital Amourosis (LCA) 10.
Elena Piotter, a PhD student working in Robert MacLaren’s group in the Nuffield Laboratory of Ophthalmology at the University of Oxford, is currently researching DNA and RNA base editing tools aiming to correct pathogenic mutations in ABCA4.
Genes exist inside all the cells that make up your body. They contain genetic code (DNA) and provide instructions for constructing proteins, which are the building blocks of our bodies and perform a huge variety of roles.
Your doctor or genetic counsellor will spend some time asking about other people in your family to try to work out the way in which your particular faulty gene has been passed down through the generations – this is referred to as the inheritance pattern.
Biotechnology company MeiraGTx has announced encouraging news from its phase 1/2 clinical trial of botaretigene sparoparvovec (previously known as AAV-RPGR), a gene therapy aimed at X-linked retinitis pigmentosa caused by faults in the RPGR gene.
The following questions are often asked by people contacting Retina UK.
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Fancy fundraising for Retina UK? Here’s a few pointers to help get you started.
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.
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