IOO Genetics

We have started a clinical trial to assess the safety of injecting retinal pigment epithelium (RPE) cells, grown from embryonic stem (ES) cells, into the eyes of patients with Stargardt macular dystrophy. This is the first clinical trial in Europe to use cells derived from ES cells, and follows an ongoing study in the USA where two patients received similar cells without any complications to date.

In an important study, the Gene and Cell Therapy Group have showed for the first time that transplanting light-sensitive photoreceptors into the eyes of visually impaired mice can restore their vision.

The results, published in Nature, suggest that transplanting photoreceptors – light-sensitive nerve cells that line the back of the eye – could form the basis of a new treatment to restore sight in people with degenerative eye diseases.

Dr. Rachael Pearson injected cells from young healthy mice directly into the retinas of adult mice that lacked functional rod photoreceptor cells - rod cells are vital for seeing in the dark as they are extremely sensitive to even low levels of light. Loss of photoreceptors is the cause of blindness in many human eye diseases including age-related macular degeneration, retinitis pigmentosa and diabetes-related blindness. 

After four to six weeks, the transplanted cells appeared to be functioning almost as well as normal rod photoreceptor cells and had formed the connections needed to transmit visual information to the brain. 

We also tested the vision of the treated mice in a dimly lit maze. Those mice with newly transplanted rod cells were able to use a visual cue to quickly find a hidden platform in the maze whereas untreated mice were able to find the hidden platform only by chance after extensive exploration of the maze.

Patients with retinal degeneration and their families gathered in London on April 21^st to attend the first UCL/Moorfields Biomedical Research Centre for Ophthalmology “Retina Patient Day”. Over 250 attendees had the opportunity to interact with more than 40 clinicians and scientists from Moorfields Eye Hospital and UCL Institute of Ophthalmology Department of Genetics, who provided updates on their research into developing effective treatments for blinding retinal conditions.