Elizabeth A. M. Windsor

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 · 660 citations · Full text

Slow Rod Kinetics, Gene Therapies, Ocular Disease +12

Human <i>RPE65</i> Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

Artur V. Cideciyan, William W. Hauswirth, Tomás S. Alemán et al. · Human Gene Therapy · 2009 · 311 citations · Full text

Identifying photoreceptors in blind eyes caused by <i>RPE65</i> mutations: Prerequisite for human gene therapy success

Samuel G. Jacobson, Tomás S. Alemán, Artur V. Cideciyan et al. · Proceedings of the National Academy of Sciences · 2005 · 279 citations · Full text

Gene Therapies, Retinal Gene Therapy, Developmental Biology +13

Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection

Samuel G. Jacobson, Gregory M. Acland, Tomás S. Alemán et al. · Molecular Therapy · 2006 · 220 citations · Full text

ABCA4 disease progression and a proposed strategy for gene therapy

Artur V. Cideciyan, Małgorzata Świder, Tomás S. Alemán et al. · Human Molecular Genetics · 2008 · 185 citations · Full text

Ocular Disease, Engineering, Genetics +18