Dr. Robert Koenekoop of Montreal Children’s Hospital calls it “one of the most significant advances in human history in the field of medicine.” Using gene therapy, scientists have succeeded in providing partial vision to patients who were almost blind due to a condition known as Leber congenital amaurosis, which is characterized by blindness from birth. It means scientists might finally “be able to treat a complicated retinal disease that we thought only ten years ago was untreatable,” Koenekoop said on CTV’s Canada AM.
Three patients (two women, aged 19 and 26, and a man, 26) were involved in the study. Researchers injected millions of copies of a working gene under the retina in the back of their eyes, treating only one eye on each (the worst), to compare against the unaffected eye. All three of them (who apparently had “severely abnormal vision” before the study began) can now read several lines on an eye chart, and can see better in dark spaces (results were published in The New England Journal of Medicine). No serious side effects were reported, although one patient did develop a hole in his retina—but it didn’t affect his eyesight.
As trials continue, nine individuals aged 8 to 27 will be enrolled; researchers believe the treatment could potentially give near-normal vision to children born blind.