Exome sequencing identifies NMNAT1 mutations as a cause of Leber congenital amaurosis

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

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Albert M. Maguire, Katherine A. High, Alberto Auricchio et al. · The Lancet · 2009 · 858 citations

Medicine, Genetics, Molecular Genetics +12

Wallerian degeneration of injured axons and synapses is delayed by a Ube4b/Nmnat chimeric gene

Till G.A. Mack, Michael Reiner, Bogdan Beirowski et al. · Nature Neuroscience · 2001 · 614 citations

Life Sciences, Neurobiology, Neuroscience +14

Leber Congenital Amaurosis–A Model for Efficient Genetic Testing of Heterogeneous Disorders: LXIV Edward Jackson Memorial Lecture

Edwin M. Stone · American Journal of Ophthalmology · 2007 · 307 citations · Full text

Life Sciences, Genetics, Molecular Genetics +14

A <i>Ufd2/D4Cole1e</i> chimeric protein and overexpression of <i>Rbp7</i> in the slow Wallerian degeneration ( <i>Wld</i> ^S ) mouse

Laura Conforti, Andrea Tarlton, Till G.A. Mack et al. · Proceedings of the National Academy of Sciences · 2000 · 252 citations

Life Sciences, Genetics, Neuroscience +14