Study: Children Benefit Most From Gene Therapy For Treating Leber Congenital Amaurosis

Children inflicted with Leber Congenital Amaurosis (LCA) have great chances of improving their condition with gene therapy. According to a new study, children with LCA benefit most from gene therapy.

What is LCA?

According to AAPOS, LCA is an inherited condition caused by a defect in the cells that detect light in the retina, as a result it causes poor vision. Most findings are diagnosed after 2-3 months of age. LCA is a rare condition, only three in 100,000 births have LCA. Unfortunately, this condition is tough. About 10 to 18 percent of congenital blindness or severely reduced vision in children are due to LCA.

Gene Therapy Efficient Treatment For LCA And SECORD

To help children with LCA, the scientists at Oregon Health & Science University Casey Eye Institute and Baylor College of Medicine's Cullen Eye Institute published their findings from a two-year Phase I clinical trial in the Ophthalmology journal. The study showed promising results.

According to Science Daily, children benefit the most from gene therapy for LCA treatment or Severely Early Childhood Onset Retinal Degeneration (SECORD). LCA and SECORD are related in inherited retinal degenerative diseases that trigger severe loss of vision in infancy. Per the study, nine of the 12 participants experienced better visual functions after gene therapy.

"While other studies have shown similar results, an important finding from this study is that the young patients saw the greatest benefit in treatment," said Timothy Stout, senior author of the study. "In addition, this study for the first time demonstrates improvement in visual field by using visual field modeling and assessment."

The study involved eight adults and four children ages 6 to 39. The Phase 1 clinical trials revealed that nine out of 12 patients experienced better visions. Gene therapy is a new approach to treating rare genetic eye diseases due to mutation like LCA and it has proven efficient.

"I am very optimistic about these study findings," said Richard Weber, senior author and professor of ophthalmology in oHSU's Casey Eye Institute. "Gene replacement therapy has proven to be the most promising method to halt the progression of childhood blindness due to single gene defects. The eye is a perfect platform for gene therapy because we can treat one eye to see how it responds compared to the other eye."

Do you agree that gene therapy is promising for treating genetic diseases? Are you hopeful of this new approach? Share your thoughts below.