Irina Zakharova, a Candidate of Biological Sciences and Head of the Scientific and Educational Laboratory of Regenerative Biotechnologies and Tissue Engineering at the National Research Nuclear University MEPhi, has stated that optogenetics may become the foundation for new treatments to restore vision in patients with blindness and other conditions.
Optogenetics enables control of cell activity using light. According to Zakharova, this technique involves injecting a gene for a photosensitive protein known as opsin into cells. When exposed to light, this protein opens ion channels in the cell membrane, triggering electrical pulses that restore function.
“Optogenetics allows you to selectively activate only specific types of cells without affecting neighboring ones,” Zakharova explained.
The approach has been instrumental in identifying neural circuits linked to memory, fear, hunger, thirst, and social connections. In medical applications, it could potentially treat blindness and deafness, combat neurodegenerative diseases, and correct heart rhythm disorders.
Specifically, the MCO-010 therapy from Nanoscope Therapeutics is nearing completion of clinical trials for patients with severe stages of retinitis pigmentosa or Stargardt’s disease. This treatment uses an adeno-associated virus to deliver a multifunctional opsin gene to bipolar retinal cells.
Zakharova noted that light-insensitive cells initially gain the ability to respond to ordinary daylight, and in some patients, vision recovery has been maintained for almost three years.
