CRISPR Partially Restores Vision to Blind Patients During Trial
Results from a Phase 1/2 clinical trial indicate that CRISPR gene-editing has enhanced the vision of individuals with inherited blindness, offering promising prospects for patients and suggesting broader applications of CRISPR in human therapeutics.
Leber Congenital Amaurosis (LCA) is a rare disorder affecting approximately one in every 40,000 newborns. Individuals with LCA carry a genetic mutation that severely impairs vision, leading to complete blindness in about one-third of cases. Presently, there are no FDA-approved treatments available.
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