Title | Creator | Description | Identifier | ||
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1 | Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Stability of Clinical Parameters of Carriers in Preparatory Phase | Byron L. Lam; William J. Feuer; Vittorio Porciatti; Ruth Vandenbrke; Joyce C. Schiffman; Fawzi Abukhalil; Potyra R. Rosa; Sophia Cuprill-Nilson; John Guy | 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 | |
2 | Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Stability of Clinical Parameters of Carriers in Preparatory Phase | Byron L. Lam; William J. Feuer; Vittorio Porciatti; Ruth Vandenbroucke; Joyce Schiffman; Fawzi Abukhalil; Potyra R. Rosa; Sophia Cuprill-Nilson; John Guy | 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 |