Choroideremia Gene Therapy Showing Promise

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 [Read More]