Gene Therapy Cures Eye disease

Scientists have brought back the sight to a 58-year-old man with the inherited Eye Disease retinitis pigmentosa by injecting genetically engineered viruses into his eye. The man who was blind for decades was able to see small objects like a staple box, a tumbler or a notebook when wearing a specialized pair of goggles. This is described in the paper published in the journal Nature Medicine on Monday.

This relies on optogenetics, a fledgling area of biological research aimed at controlling nerve cells via light. Raymond Wong, a stem cell biologist at the University of Melbourne developing treatments for Eye DiseaseEye Disease who were not affiliated with the study said this is exciting news.

Most people are reading this article on your mobile device or your computer screen because of the complex decoding performed by your retina. Botond Roska, a biomedical researcher at the University of Basel and author of the new study said that the retina is like a biological computer at the back of your eye.

The biological computer has layers. Light filters in through the eye and interacts with specialized photoreceptor cells at the bottom layer of the computer, known as rods and cones. These pass signals on to another specialized cell known as a retinal ganglion cell, which sits at the top layer of this computer.

Genetic mutations cause the rods and cones to function incorrectly or die off. There are dozens of different mutations which can lead to retinitis pigmentosa. According to the National Institutes of Health, the disease affects up to 1 in 4,000 people worldwide. In many cases, this results in a patient with tunnel vision and eventually, most patients lose their sight.