Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy

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

A point mutation of the rhodopsin gene in one form of retinitis pigmentosa

Thaddeus P. Dryja, Terri L. McGee, Elias Reichel et al. · Nature · 1990 · 1.1K citations

Medicine, Genetics, Molecular Genetics +12

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

William W. Hauswirth, Tomás S. Alemán, Shalesh Kaushal et al. · Human Gene Therapy · 2008 · 947 citations · Full text

Medicine, Molecular Biology, Ophthalmology +15

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia

Haibin Xia, Qinwen Mao, Steven Eliason et al. · Nature Medicine · 2004 · 651 citations

Life Sciences, Molecular Genetics, Neuroscience +14