Using experimental gene therapy in mice, University of Florida scientists turned off a gene playing an essential role in retinitis pigmentosa, a major cause of inherited blindness.
This therapy includes injecting the eye with genetic material known as interfering RNA, which puts out of action the targeted gene, called rhodopsin, the investigators explained. In general, this gene is necessary for healthy eyesight. But mutated versions of the gene may lead to retinitis pigmentosa.
Mutated forms of rhodopsin generate a toxic protein in the retina that is responsible for killing cells that receive light. The initial symptoms normally occur between the ages of 10 and 30, and the majority of people are blind by the age of 40. Retinitis pigmentosa strikes around 1.5 million people all over the world.
The UF group of scientists engineered the interfering RNA into a virus and injected it into the retinas of over a dozen normal mice.
According to the research, the treatment decreased the amount of rhodopsin by around 60 per cent.
May finally work in people
The results of the study are expected to be published in an upcoming issue of the journal Vision Research. This study indicates a potential method of treating patients experiencing retinitis pigmentosa.
"If we decrease the amount of protein molded by mutated rhodopsin, that may be enough to sustain vision in patients who are affected by retinitis pigmentosa," Marina Gorbatyuk, assistant professor of molecular genetics and microbiology in the UF College of Medicine, said in a prepared statement.
The UF scientists are now planning to learn whether introducing standard rhodopsin to the retinas of mice will bring back vision.
