A blind 58-year-old man has partially regained his vision thanks to the scientific breakthrough of Hungarian biomedical researcher Botond Roska. Roska and his research team have shown that optogenetic therapy can help individuals with retinitis pigmentosa partially regain their sight. This is a big step for gene therapy and could lead to technology that restores vision.
Nature Medicine reported on the findings of an international research team led by José-Alain Sahel and Botond Roska which included members from Institut de la Vision and Hôpital National des Quinze-Vingts, Paris, the University of Pittsburgh, the Institute of Molecular and Clinical Ophthalmology Basel (IOB), StreetLab, and GenSight Biologics.
On the Road to Restoring People’s Vision
Roska, Founding Director of IOB and Professor at the University of Basel, said that “the findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible.”
The doctor explained that all optic misfunctions originate in the eye or retina, which acts like a biological computer, recording video, then creating 30 representations of that video and forwarding it to the brain. The brain then predicts what it sees based off the information sent from the eyes. Vision impairment occurs when part of that connection is interrupted.
The research was focused on retinitis pigmentosa, a neurodegenerative eye disease responsible for 20 percent of all blindness. This disease causes the light-sensing layer of the eye to be desensitized to light, thus the goal of Roska’s team is to return that sensitivity.
Optogenetics, the therapy used by Roska’s team, involves genetically altering cells to produce light-sensitive proteins called channelrhodopsins, that restore sensitivity to the photoreceptors of the eye. It took his team, in collaboration with that of José-Alain Sahel from the University of Pittsburgh School of Medicine, 13 years of research to reach the findings they have today.
Optogenetics Proven to Fix Blindness
The therapy was initially used on mice and monkeys before clinical trials were launched in 2018-2019. The French man who took part in their clinical trials began going blind 40 years ago. Due to the pandemic, he is currently the only patient of the research team.
In the beginning of the trails, the patient’s worse eye was injected with a harmless virus carrying the gene for an opsin protein that would return sensitivity to his photoreceptors.
Researchers then waited 4.5 months for the patient’s eye to produce the new protein. Then, with the use of special goggles that amplify incoming light, he began being able to see objects.
Thanks to this method, the entirely blind patient started to see objects. It is the first step in the process of restoring vision, and we hope that the events will pick up pace from now on.”
Roska believes there to be two directions from these results. Either a new science of visual rehabilitation has been created, or a door has been opened to new optogenetic strategies which might present therapies that are even better than the ones his team discovered.
A Potential Nobel Prize for Botond Roska
In an interview with Válasz Online, Roska said that the therapy used to cure the patient’s sight is similar to the Covid-19 vaccines.
Gene therapies use in large part adeno-associated viruses as vectors. This is completely safe, and today we are already able to rewrite the DNA of the virus as we see fit to the sequences which require the specified gene therapy.”
He added that his institute cooperates with three Hungarian research institutions and has Hungarians present in key positions. Roska works with Hungarian scientists such as Balázs Rózsa of Femtonics Ltd., Arnold Szabó, and Zoltán Nagy of Semmelweis University.
The work of Roska and his team will no doubt bring on even more success, perhaps even a Nobel Prize.
Featured photo illustration via Semmelweis University’s Youtube channel