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Gene therapy injection tested to halt rare form of blindness

NCT ID NCT02652780

Summary

This study tested whether a single gene therapy injection (called GS010) could improve vision in people with a rare inherited eye disease called Leber Hereditary Optic Neuropathy (LHON). The trial involved 37 participants who had experienced vision loss for 6 to 12 months. Researchers compared the effects of the real injection to a sham (placebo) procedure to see if the therapy could help restore some sight.

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Contacts and locations

Locations

  • Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt

    Paris, Paris, France

  • Department of Neurology, University of Munich, Friedrich-Baur-Institute

    Munich, 80336, Germany

  • Department of Ophthalmology, Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Doheny Eye Center, University of California, Los Angeles

    Los Angeles, California, United States

  • IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

    Bologna, Bologna, Italy

  • Moorfields Eye Hospital NHS Foundation Trust

    London, EC1V 2PD, United Kingdom

  • Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

Conditions

Explore the condition pages connected to this study.