Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse

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

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Artur V. Cideciyan, Tomás S. Alemán, Sanford L. Boye et al. · Proceedings of the National Academy of Sciences · 2008 · 664 citations · Full text

Medicine, Genetics, Ophthalmology +17

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy

Qing Xie, Weishu Bu, Smita Bhatia et al. · Proceedings of the National Academy of Sciences · 2002 · 610 citations

Life Sciences, Biology, Molecular Biology +17

Positional cloning of the gene associated with X-linked juvenile retinoschisis

Christian Sauer, Andrea Gehrig, Regina Warneke-Wittstock et al. · Nature Genetics · 1997 · 497 citations

Medicine, Genetics, Molecular Genetics +12