Healing Blindness in Mice

Transforming sight: Delivering a light-sensitive protein (highlighted in green) to specific cells in the retina (highlighted in red) of a blind mouse makes those cells sensitive to light, restoring some vision. (Other retinal cells appear in blue.)

Viruses can deliver light-sensitive proteins to specific cells in the retinas of blind mice, allowing rudimentary vision, according to new research. Although previous studies have shown that the light-sensitive proteins can be beneficial, the delivery methods were not practical for humans. The viral-delivery method is similar to ones already used in human gene therapy.

The new light-sensitive proteins were active for the length of the study, about 10 months, suggesting the treatment would work long-term. In addition, the therapy appeared safe; the proteins, which were derived from algae, remained within the eye, and they did not trigger inflammation.

“In my opinion, the biggest step forward in this paper is the use of viral delivery techniques, the same delivery techniques that would have to be used should the technique move on into human treatment,” says Thomas Münch, a researcher at the University of Tübingen, who was not involved in the study but has done similar research. Recent gene-therapy studies, which used similar viruses to deliver different proteins, have shown preliminary success in treating a rare genetic form of blindness in patients.But the current approach could be applied to a much broader group of people because it could restore light-sensitivity to the retina regardless of the cause of degeneration.

To restore vision, Alan Horsager, a researcher at the University of Southern California, and collaborators capitalized on optogenetics, a type of genetic engineering that makes neurons sensitive to light. They used a specially designed virus to deliver numerous copies of the gene that makes a protein called channelrhodopsin to the eye. The protein forms a channel that sits on a cell’s membrane and opens when exposed to light. Positively charged ions then rush into the cell, triggering an electrical message that is transferred to other cells in the retina.

via Healing Blindness in Mice – Technology Review.

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