Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
By A. Cideciyan, T. Aleman, S. Boye, S. Schwartz, S. Kaushal, A. J. Roman, J. Pang, A. Sumaroka, E. A. Windsor, James M. Wilson and 7 more. Proceedings of the National Academy of Sciences of the United States of America, 2008
714 citations recorded by the source corpus.
Subfields: Molecular Biology, Ophthalmology
View Paper: https://www.semanticscholar.org/paper/d75d029520be78b461a784097a251baf543da223
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- S. L. Ginn
- S. Jacobson
- I. Alexander
- A. Cideciyan
- Michael Edelstein
- M. Abedi
- R. Ratnakaram
- S. Ferrari
Citations in the corpus
- Retinitis Pigmentosa: Genes and Disease Mechanisms (2011, background): “Results in pre-clinical studies have recently led to three encouraging gene therapy clinical trials in which patients affected by LCA were sub-retinal injected with recombinant adeno-associated viral vectors (rAAV) containing the human RPE65 cDNA [24-26].”
- Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years (2011, methodology): “The latter condition draws from our previous observations of prolonged kinetics of rod but not cone dark adaptation in RPE65related LCA treated with gene therapy.(18) Further details are provided in the eAppendix.”
- Effect of genome size on AAV vector packaging. (2010, neutral): “(1) Packaging, (2) uncoating, (3) second-strand DNA synthesis, (4) annealing, and (5) homologous recombination.”
- Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “Using sub-retinal administration of recombinant adeno-associated viral vectors (rAAV) expressing RPE65, three independent clinical trials for LCA have been initiated [32–35].”
- Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, neutral): “50 Similarly, subretinal vector delivery has been performed in a number of clinical studies of gene transfer for RPE65 de fi ciency, 5-7 , 47 and in the immunologically The limitations imposed by the human immune response to AAV capsid are well illustrated in the hemophilia B trials.”