Gene Therapy · 2009 · 278 citations · 27 references
Life SciencesBiotechnologyMolecular BiologyGene DeliveryGene TherapyVirologyVector DesignVector ProductionVector PurificationTransductionTransduction Efficiency AssessmentTransduction Efficiency OptimizationVector ImmunogenicityVector TransductionVector Yield OptimizationViral Vectors
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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