Some very exciting news for patients who experience site loss as a result of retinal dystrophy. In particular, this treatment is prescribed for patients whose conditions is a direct result of inheriting a flawed RPE65 gene from both parents. RPE65 is imperative for providing the pigment that light sensitive cells require to properly absorb light. As the article states, the condition begins by only impacting a person’s ability to see at night, but as the condition progresses and cells die it can lead to total blindness.
At this point, the treatment has limited implications; it is only appropriate for patients with some remaining vision and the timeline of benefits is not for-certain (it could be up to several decades). It remains an exciting advancement in the treatment of blindness or near-blindness and is said to bring the largest benefits to children with this condition, possibly pausing sight loss before permanent damage can be done.
