Researchers at Oxford University have reported successful findings from their Phase 1/2 clinical testing of the CHM gene for treatment of choroideremia. Choroideremia is a rare disease affecting mostly males that leads to blindness.
In the multicentre clinical trial, six male patients (aged 35—63 years) all showed improvement after replacement of the malfunctioning gene. The mean gain in acuity was 3.8 letters on the chart. The researchers concluded that their findings lend support to further assessment of gene therapy in the treatment of choroideremia and other diseases, such as age-related macular degeneration.