Vision of a man who lost sight because of Retinitis pigmentosa (RP) was partially recovered by optogenetic treatment in the first reported case of functional recovery in this new post-treatment neurodegenerative disease.
“Witnessing the initial recovery of visual function in visually impaired patients was breathtaking,” Botond Roska, MD, and PhD said in a news release.
“We’ve been working on optogenetic therapies in the lab for 16 years, and now it’s a unique experience to see a proof of concept for a patient,” said the founding of the Institute for Molecular Clinical Ophthalmology in Basel, Switzerland. Director Roska added. -Founder of GenSight Biologics, a company that develops treatments.
The case is Report online May 24 Nature Medicine.
Important milestones
Blindness in a 58-year-old man was caused by retinitis pigmentosa (RP), a neurodegenerative eye disease in which loss of photoreceptors can lead to complete blindness.
As part of the Phase 1/2a PIONEER study, patients received an intraocular injection of an adeno-associated virus vector encoding the optogenetic sensor Chrimson R in one blind direction. The team also designed light-stimulated goggles equipped with a camera that captures visual images and projects them onto the retina.
Training with goggles began about 5 months after injection, which gave Chrimson R time to stabilize in ganglion cells. Seven months later, the patient began reporting signs of improved vision.
“In this kind of study, we really learn from the patients themselves. They’re like experimenters telling us what we’re looking at with restored vision,” Pennsylvania said. Dr. Jose Alan Sahel, MD, Principal Investigator, University of Pittsburgh Medical Center, Pittsburgh, said. Said at a press conference.
The team reported that the patient was able to recognize, locate, count, and touch a variety of objects while wearing goggles, using only vector-processed eyes. Patients were unable to visually detect objects before or after injection with or without goggles.
Patients also reported a significant improvement in their ability to perform daily activities, such as navigating in outdoor and indoor environments.
During vision, multi-channel EEG recordings showed object-related activity above the visual cortex.
“Therefore, we were able to show that visual behavior correlates with the activation of the brain, which corresponds to visual function, in this patient,” Rosco said.
Video of the patient performing the test, submitted as a supplement Nature medicineCan be seen at www.gensight-biologics.com..
“This is an important milestone, and undoubtedly further improvements will make optogenetic therapy a viable option for many patients in the future,” said Robert MacLaren, MBChB, Professor of Ophthalmology, Oxford University, Oxford University, UK. DPhil says. Non-profit British Science Media Center.
“This exciting new technology may help people with very serious vision problems,” said James Bainbridge, MBChB, and PhD, professor of retina research at United Kind Rubber, University College London, London. This is a quality study. It is carefully carried out and managed.
“The findings are based on laboratory tests in just one individual. More work is needed to see if this technology can be expected to provide a useful vision,” said Bainbridge. Stated.
This study was funded by GenSight. Several authors have disclosed their financial relationship with the company. McLaren is NICE’s scientific advisor on retinal gene therapy. Bainbridge does not disclose any relevant financial relationships.
Nut Med.. Published online on May 24, 2021. Full text
