Scientists have restored sight to blind mice using gene therapy
Thanks to a new gene therapy, scientists have succeeded in restoring blind mice’s eyesight to a degree that allowed them to move efficiently between obstacles. Researchers want to test their method on humans soon.
Researchers at the University of California, Berkeley, using an inactive virus, have placed a gene encoding a green light receptor in the eyes of blind mice. After a month, it was observed that the until recently blind mice avoided obstacles roas easily as mice without problemsow with vision. Scientists say that treated rodents are able to perceive movement, changes in light brightness, and even someore small details on the iPad screen, whichowhich would be enough to restoreoletter sensations.
As the authors of the study admitted, the technique they used is surprisingly simple, and the results in mice are so good that they would like to spruce up a similar gene therapyobe in clinical trials involving humans within three years. This treatment could helpoc people whoore have lost their vision due to macular degenerationolt or suffering from retinal pigmentary degenerationowki, giving them sufficient visual ability to move efficiently. Perhaps it would be possible roalso restoreoto give them the ability to read or watch TV.
The findings were published in the journal „Nature Communications”.
About 170 millionoin people around the world are living with age-related macular degenerationoltej. The condition affects one in ten individualsob over 55 years of age. In contrast, for the most common form of hereditary blindness, retinal pigmentary degenerationowki, who suffers nearly two million osob.
Ehud Isacoff, a professor of molecular biology and comohe UC Berkeley researcher admitted that there is a good chance that after injecting a crafted virus into a patient’s eye, the patient would be able to see something after a few months. – In the case of diseasesob degenerative retinasowki research is focused on stopping or slowing further changes. Therapy, whichora could bringoto improve vision in a matter of months would be something amazing – said.
Correction of the genetic defect responsible for retinal degenerationowki is not simple. There are more than 250 roof genetic mutations responsible for retinal pigmentary degenerationowki. About 90 percent of them kill the comore photoreceptor cellsocells – the rods, responsible for perceiving shapeow and movement, as well as cones, enabling color visionow. But retinal degenerationowki usually spares other layers of the comoretinal ganglion cellsowki, including comork bipolar and ganglionic retinitis pigmentosaowki, whichore can remain healthy, although they are not light-sensitive. In tests on mice, scientists have shown that it is possible to make up to 90 percent of the. comoganglion cells.
To reverseocess to blindness in mice, the researchers engineered a virus targeting comoretinal ganglion cellsowks and applied the gene encoding the green light-sensitive protein to it – opsin. Opsins are found in cones and are responsible for their sensitivity to light. The virus carried the gene to the comoganglion cells, whichore normally insensitive to light, making them sensitive to light and able to send signalsow into the mozgu, ktore were interpreted as vision.
WspoThe author of the study, John Flannery, acknowledged that once the gene was applied, it was difficult to regrowoThe difference between healthy mice and mice thatore have undergone therapy. – We will see if it actually works if we can get a similar effect in humans – said Flannery.
In mice, the researchers were able to deliver opsin to most of the comorek ganglionic reticulumowki. However, to treat humans, they would have to inject many more virus particles, since the human eye contains thousands of times more cellsoganglion cells than the mouse eye. However, the complexoł from UC Berkeley have developed a wayob to overcome this obstacle and achieve a similarly high percentage of sensitization of the comoganglion records, which in rodents.
– We are really satisfied that our way ofob works. For this it is very simple. Ironically, we could have done this 20 years ago – said Isacoff. Researchers are currently raising funds for a clinical trial involving human. They hope these will begin within three years.