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CRISPR Gene Editing Shows Promise: Early Trial Improves Vision in Blind Children

Gene therapy has shown promise in improving eyesight in congenitally blind children with Leber Congenital Amaurosis (LCA), a rare disease.

United statesQuincy jonBaisong meiAdam cukerTomas alemanHemophilia thrombosis programEar ocular genomics instituteTech timesNew england journalMass eyeLeber congenital amaurosisStar local mediaEric pierceEditas medicine chief medical officerGene therapy gets greenlightPenn medicine

Gene Therapy Improves Vision in People With Inherited Blindness

Key Takeaways

United statesUnited kingdomTomas alemanMark pennesiBaisong meiBascom palmer eye institute in miamiWk kellogg eye centerEar ocular genomics instituteScheie eye instituteOregon health science universityHealthday newsLeber congenital amaurosisNew england journalEric pierceMass eyeBerman gund laboratory

Clinical trial shows measurable improvement in inherited blindness after gene therapy

An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.

United statesUnited kingdomMark pennesiTomas alemanBaisong meiEar ocular genomics instituteBascom palmer eye institute in miamiWk kellogg eye centerScheie eye instituteLeber congenital amaurosisNew england journalEric pierceMass eyeBerman gund laboratoryRetinal degenerationsEditas medicine

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