Gene therapy shows promise for rare eye disease
NCT ID NCT04671433
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 35 times
Summary
This study tested a gene therapy (AAV5-RPGR) for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. 105 participants received the treatment or a control. The main goal was to see if it improved their ability to navigate a maze after one year. This is a disease-control approach, not a cure.
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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.
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
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Locations
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Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
Naples, 80131, Italy
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Azienda Ospedaliero Universitaria Careggi
Florence, 50134, Italy
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Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Paris, 75012, France
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Childrens Hospital
Los Angeles, California, 90027, United States
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Duke Eye Center
Durham, North Carolina, 27705, United States
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Emory University
Atlanta, Georgia, 30322, United States
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Gartnavel General Hospital
Glasgow, G12 0YN, United Kingdom
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Hadassah Medical Center
Jerusalem, 91120, Israel
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Hosp Univ Fund Jimenez Diaz
Madrid, 28040, Spain
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Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
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IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
Roma, 00198, Italy
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Massachusetts General Hospital - Center for Celiac Research and Treatment
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
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NHS Lothian
Edinburgh, EH3 9HA, United Kingdom
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Ospedale San Paolo
Milan, 20142, Italy
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Radboudumc
Nijmegen, 6525EX, Netherlands
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Retina Consultants of Houston
Bellaire, Texas, 77401, United States
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Rigshospitalet Glostrup
Glostrup Municipality, 2600, Denmark
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Shiley Eye Institute Jacobs Retina Center
La Jolla, California, 92093 0946, United States
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St James University Hospital
Leeds, LS9 7TF, United Kingdom
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Stanford Health Care
Palo Alto, California, 94303, United States
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UZ Gent
Ghent, 9000, Belgium
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Univ of Michigan Medical Center
Ann Arbor, Michigan, 48105, United States
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Universite de Lausanne, Hopital ophtalmique Jules-Gonin
Lausanne, 1004, Switzerland
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University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
Basel, 4031, Switzerland
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University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213, United States
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VUMC Amsterdam
Amsterdam, 1105AZ, Netherlands
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VitreoRetinal Associates, PA
Gainesville, Florida, 32607, United States
Conditions
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