Gene therapy improves vision in Leber congenital amaurosis patients

Clinical trial involving 12 adults and two children with LCA sees eyesight improve in 79% of participants

An animation of three light blue DNA double helix strands are displayed on a dark blue background
Pixabay/Arek Socha

A new study published in New England Journal of Medicine has described the effect of gene editing in a group of patients with Leber congenital amaurosis (LCA).

The clinical trial involved two children and 12 adults with the genetic eye condition. Participants received an experimental gene editing therapy called Edit-101.

Outcomes used to evaluate the clinical trial were: visual acuity; full-field test results; the ability of participants to navigate a research maze containing obstacles and varied lighting levels; and self-reported improvements in quality of life.

The researchers found that 11 of the participants (79%) showed improvements in at least one of the four outcome measures, while 6 participants (43%) showed improvement in two or more outcomes. Four patients (29%) experienced improvements in visual acuity.

The scientists reported that there were no serious adverse effects linked to the treatment.

Dr Mark Pennesi, of Oregon Health & Science University, shared how rewarding it was to hear about vision improvements among patients who took part in the trial.

“One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights,” he said.

“While these types of tasks might seem trivial to those who are normally sighted, such improvements can have a huge impact on quality of life for those with low vision,” he added.