From Shadows to Sight

A revolutionary gene therapy has given blind children with a rare eye disorder the ability to see

- by IAN RANDALL

LITTLE HARVEY HAINES HAS BEEN GIVEN A new lease of life. Born with a rare eye condition severely affecting his sight, he struggled to communicate with children his own age, shied away from environments that he wasn't confident in and even found it difficult to play with his siblings. But all that has changed for him and a group of other children following a revolutionary new gene therapy breakthrough.

Researchers from London's Moorfields Eye Hospital, biotech firm MeiraGTx and University College London have demonstrated that their therapy is both safe and effective in improving the vision of and slowing retinal deterioration in young patients born with LCA-AIPL1.

This previously untreatable genetic disorder, which affects some two to three of every 10 million newborn babies, leads to profound visual impairments and legal blindness. In turn, this causes affected children to typically experience delayed and disrupted development across many areas including behavior, communication and mobility.

After trials of the new procedure, however, children that before could only play with toys by feeling them are now able to safely run about, identify pictures and even drive go-karts.

"It's an absolutely transformational improvement," paper author and Moorfields ophthalmologist Michel Michaelides told Newsweek.

LCA, or Leber congenital amaurosis, is the name given to a family of inherited eye disorders that affect the retina—the layer at the back of the eyeball containing light-sensitive "photoreceptor" cells.

This category of disorders is seen in roughly two to three out of every 100,000 births. There are many types of LCA and these vary depending on which of the genes involved in the development and function of the retina are affected.