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Gene therapy offers hope for rare blindness condition

NCT ID NCT02652780

First seen Mar 04, 2026 · Last updated May 06, 2026 · Updated 11 times

Summary

This study tested a gene therapy called GS010 for people with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that leads to vision loss. The trial included 37 participants who had vision loss for 6 to 12 months. They received a single injection of GS010 or a sham treatment to see if it could improve their eyesight.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.