A clinical trial utilizing CRISPR-based gene editing demonstrated promising results, with approximately 79% of participants showing improvement in a rare form of blindness caused by a mutation in the CEP290 gene. The study, published in the, marks a significant step in using gene editing to treat inherited retinal diseases. Credit: SciTechDaily.com
About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published today in the“This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration,” said Mark Pennesi, M.D., Ph.D.
, or LCA, Type 10, for which there is no treatment available. LCA’s various types occur in a total of about 2 or 3 out of 100,000 newborns.in early 2020. That procedure also marked the first time that CRISPR had been used to edit genes within the human body, which is also calledToday’s paper describes the study’s findings through February 2023, and details how the trial’s 14 participants –12 adults of whom were adults, while two were children – responded to receiving EDIT-101 in one eye.
“This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials,” said Mass Eye & Ear ophthalmologist Eric Pierce, M.D., Ph.D., who is also a corresponding author. “While more research is needed to determine who may benefit most, we consider the early results promising. To hear from several participants how thrilled they were that they could finally see the food on their plates – that is a big deal.