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Molecular Therapy. Methods & Clinical Development|October 27, 2021
AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorderCatherine Argyriou, Anna Polosa, Ji Yun Song, et al.Science Translational Medicine|June 24, 2011
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkeyLuk H Vandenberghe, Peter Bell, Albert M Maguire, et al.JAMA Network Open|May 28, 2024
Progesterone for Neurodevelopment in Fetuses With Congenital Heart Defects: A Randomized Clinical TrialJ William Gaynor, Julie S Moldenhauer, Erin E Zullo, et al.Journal of Virology|June 28, 2003
Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transductionEnrico M Surace, Alberto Auricchio, Samuel J Reich, et al.Translational Vision Science & Technology|May 23, 2022
Restoration of Vision and Retinal Responses After Adeno-Associated Virus-Mediated Optogenetic Therapy in Blind DogsSergei Nikonov, Puya Aravand, Arkady Lyubarsky, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|October 18, 2005
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindnessGregory M Acland, Gustavo D Aguirre, Jean Bennett, et al.Nature Reviews. Neurology|April 24, 2013
Progress in gene therapy for neurological disordersMichele Simonato, Jean Bennett, Nicholas M Boulis, et al.Molecular Therapy. Methods & Clinical Development|June 12, 2023
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoidsTess A V Afanasyeva, Dimitra Athanasiou, Pedro R L Perdigao, et al.The Journal of Clinical Investigation|April 17, 2008
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in miceMariacarmela Allocca, Monica Doria, Marco Petrillo, et al.Ophthalmology|December 18, 2016
Natural History of the Central Structural Abnormalities in Choroideremia: A Prospective Cross-Sectional StudyTomas S Aleman, Grace Han, Leona W Serrano, et al.Pageof 20