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Title | Creator | Description | Identifier |
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Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Stability of Clinical Parameters of Carriers in Preparatory Phase | Lam, Byron L; Feuer, William J; Porciatti, Vittorio; Vandenbroucke, Ruth; Schiffman, Joyce; Abukhalil, Fawzi; Rosa, Potyra R; Cuprill-Nilson, Sophia; Guy, John R | The preparatory phase of the LHON gene therapy trial aims to characterize affected G11778A patients and carriers for the planned trial that will utilize allotopic expression via an adeno-associated virus vector. This report focuses on the natural history of visual outcome measures in asymptomatic G1... | 20140304_nanos_sciplatform2_01 |
2 |
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Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Stability of Clinical Parameters of Carriers in Preparatory Phase | Lam, Byron L; Feuer, William J; Porciatti, Vittorio; Vandenbroucke, Ruth; Schiffman, Joyce; Abukhalil, Fawzi; Rosa, Potyra R; Cuprill-Nilson, Sophia; Guy, John R | The preparatory phase of the LHON gene therapy trial aims to characterize affected G11778A patients and carriers for the planned trial that will utilize allotopic expression via an adeno-associated virus vector. This report focuses on the natural history of visual outcome measures in asymptomatic G1... | 20140304_nanos_sciplatform2_01-1 |