Participants in Pioneering CRISPR Gene Editing Trial See Vision Improve

“This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials.” – Corresponding author

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 on May 6 in the New England Journal of Medicine. The article is titled “Gene Editing for CEP290-Associated Retinal Degeneration.” “This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration,” said Mark Pennesi, MD, PhD, a corresponding author on the paper, an ophthalmologist and Oregon Health & Science University’s (OHSU’s) lead scientist for the Phase 1/2 BRILLIANCE trial. “There is nothing more rewarding to a physician than hearing a patient describe how their vision has improved after a treatment. 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.” “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.”
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