India, May 6 -- A recent clinical trial has shown promising results for a CRISPR/Cas9 gene editing therapy that could potentially restore vision in individuals with a rare type of inherited or congenital blindness.
The trial, called BRILLIANCE, involved 14 participants - 12 adults and 2 children - who received treatment with EDIT-101 to address Leber Congenital Amaurosis (LCA) caused by mutations in the CEP290 gene. The trial is seen as a landmark achievement in the treatment of genetic blindness, offering an alternative treatment option when traditional forms of gene therapy are not viable.
During the trial, each participant received an injection of EDIT-101 in one eye through a surgical procedure. The participants were divided into thre...