Search Results for “Case study - Ambassador” - Page 13

A DNA spiral with a piece removed being held by tweezers

Genetics and gene therapy

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.

A magnifier illustration highlighting test tubes in a rack

Genetic testing

Genetic testing, usually via a blood sample, is used to try and identify which gene contains the fault that is causing an individual’s sight loss.

Retina UK awards new research funding

Retina UK is delighted to have awarded a new three-year research grant to Prof Majlinda Lako at Newcastle University for a project entitled: To establish AAV.PRPF31 gene augmentation in PRPF31-deficient RPE and photoreceptor cells and assess its efficacy in restoring RPE and photoreceptor function.

ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10

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.