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In a recent study to confirm a new surgical procedure’s safety in treating a type of blinding disease, patients reported quick and lasting vision improvement.
Gene therapy has a partially successful record in treating immune diseases and blood cancers, but research is ongoing with small studies and preliminary results. In a recent study to confirm a new surgical procedure’s safety in treating a type of blinding disease, patients reported quick and lasting vision improvement.
In gene therapy, doctors inject new genes so that cells hopefully will use them to prevent further degeneration caused by mutated genes.
For this study, all six patients age 35 to 63 have a genetic disease called “choroideremia,” which gradually blinds them from childhood. Doctors injected a working gene just under the eye’s retina via a virus.
Not only was there no eye damage, but all six patients reported improvements six months later. Four of them had increased light sensitivity while two saw significant changes in reading, adding 2 to 4 lines on an eye chart.
Some months after the procedure, 43-year-old Wayne Thompson said he saw stars for the first time in years.
While no one knows how long the surprising effects will last, one patient has had his improvements holding strong for 2 years. Doctors are optimistic about further research into higher-dose therapies and the potential to prevent blindness due to choroideremia and other retinal diseases.