One-Shot gene injection aims to halt blindness in rare eye disease
NCT ID NCT04671433
Summary
This study tested whether a single injection of an experimental gene therapy could slow or stop vision loss in people with X-linked retinitis pigmentosa, a rare inherited eye disease. The therapy, called AAV5-hRKp.RPGR, delivers a working copy of a faulty gene directly into the eye. Researchers measured changes in participants' ability to navigate a mobility course and their retinal sensitivity over one year to see if the treatment was effective and safe.
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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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