Gene Therapy · 2010 · 105 citations · 34 references
Life SciencesGeneticsMolecular BiologyGene DeliveryGene ExpressionGenetic EngineeringGene Delivery VectorsPromoter DesignPromoter EngineeringPromoter RegulationPromoter SelectionSafety Of Gene TherapyPromoter MutagenesisPromotersToxicity Of Gene VectorsVector Dose OptimizationViral Vector Promoters
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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text
Medicine, Genetics, Ophthalmology +17
Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
James Bainbridge, Alexander J. Smith, Susie S. Barker et al. · New England Journal of Medicine · 2008 · 1.9K citations · Full text
Medicine, Genetics, Ophthalmology +15
Gene therapy restores vision in a canine model of childhood blindness
Gregory M. Acland, Jharna Ray, Qí Zhāng et al. · Nature Genetics · 2001 · 1.2K citations
William W. Hauswirth, Tomás S. Alemán, Shalesh Kaushal et al. · Human Gene Therapy · 2008 · 947 citations · Full text