In a medical milestone, researchers have treated the first patient with inherited vision loss using an innovative CRISPR-based therapy designed to restore sight.
A groundbreaking clinical trial, detailed in a March 4, 2020, press release from Editas Medicine, aims to restore vision to those affected by genetic blindness. This U.S.-based genome-editing leader has collaborated with Oregon Health & Science University and Allergan plc. The first patient was dosed in the phase 1/2 BRILLIANCE trial.
The patient's vision loss stems from a hereditary mutation in the CEP290 gene, causing Leber congenital amaurosis 10 (LCA10), a form of childhood blindness. The experimental therapy, AGN-151587 (EDIT-101), is delivered via subretinal injection to precisely edit the faulty gene using CRISPR technology.
This marks the first human trial of an in vivo CRISPR genome-editing therapy, administered directly within the body. The phase will evaluate the treatment across 18 patients, focusing on safety, tolerability, and efficacy.
Dr. Mark Pennesi, who treated the initial patient, emphasized the trial's significance in advancing promising new treatments for genetic vision disorders. Full results are pending as the study continues.
Previously, U.S. researchers at the University of Georgia used CRISPR-Cas9 to engineer albino lizards, aiding studies in oocyte fertilization for egg-laying species.
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