Optogenetic gene therapy restores some vision in small trial
- Six participants with advanced retinitis pigmentosa experienced clinically meaningful improvements in light sensitivity following a single gene therapy injection, according to research published in the New England Journal...
- The experimental approach uses optogenetic therapy, a technique that turns nerve cells on and off using light.
- What we can say for sure is that the concept of optogenetics is valid for vision restoration, said Prof.
Six participants with advanced retinitis pigmentosa experienced clinically meaningful improvements in light sensitivity following a single gene therapy injection, according to research published in the New England Journal of Medicine and covered by STAT and The Guardian. While the study followed participants for up to five years, researchers noted that only one severe eye-related side effect occurred, which resolved within minutes.
Optogenetic Treatment Restores Basic Vision in Clinical Trial
The experimental approach uses optogenetic therapy, a technique that turns nerve cells on and off using light. The procedure involves a single injection into the eye, targeting ganglion cells that send visual information to the brain but deteriorate less rapidly than light-sensitive photoreceptor cells destroyed by retinitis pigmentosa.
What we can say for sure is that the concept of optogenetics is valid for vision restoration,
said Prof. Botond Roska, director of the Institute of Molecular and Clinical Ophthalmology Basel and a lead author of the study, as quoted by The Guardian.
Goggles Translate Light Into Monochrome Images
Patients wear specialized light-stimulating goggles that capture images of the surrounding environment and convert them into single-wavelength pulses of light. These light pulses activate the ganglion cells that have been modified to become light-sensitive, allowing the wearer to perceive a monochrome image.
Out of the ten trial participants who received the treatment in their worse-seeing eye, six showed clinically meaningful improvements in light sensitivity. Some participants also demonstrated better performance in a range of tasks when wearing the light-stimulating goggles after receiving the gene therapy.
Future Goals Aim Toward High-Resolution Vision
Researchers emphasized that the current functional gains are initial steps. The primary aim for the research team is now working toward restoring high-resolution vision, with an estimated goal of achieving that milestone within five to ten years.
Gene Therapy Repairs Damaged Retinas in Animal Studies
One can essentially discuss the mutations in the retinal gene as a typo in a blueprint that makes the instructions incomprehensible to the system, resulting in a faulty design and subsequent vision loss,
study lead Billie Beckwith-Cohen said in a press release. Not only were new components added, but pre-existing abnormalities were repaired.