‘Life-changing’ gene therapy for children born blind

Submission Statement:

An experimental trial of gene therapy has helped four toddlers – born with one of the most severe forms of childhood blindness – gain “life-changing improvements” to their sight, according to doctors at Moorfields Eye Hospital in London.

The rare genetic condition means the babies’ vision deteriorated very rapidly from birth.

Before the therapy, they were registered legally blind and only just able to distinguish between dark and light. After the infusion, all parents reported improvements – with some of their young children now able to begin to draw and write.

Further work is being done to confirm the early study, which appears in the Lancet medical journal.

The new work builds on that success by injecting healthy copies of a defective gene into the back of a child’s eye, very early in life, to treat a severe form of the condition.