Arpad Palfi

Effective delivery of large genes to the retina by dual AAV vectors

Ivana Trapani, Pasqualina Colella, Andrea Sommella et al. · EMBO Molecular Medicine · 2013 · 262 citations · Full text

Medicine, Biotechnology, Ophthalmology +17

Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

Amelia Lane, Katarina Jovanović, Ciara Shortall et al. · Stem Cell Reports · 2020 · 187 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +17

RNA Interference–Mediated Suppression and Replacement of Human Rhodopsin In Vivo

Mary O’Reilly, Arpad Palfi, Naomi Chadderton et al. · The American Journal of Human Genetics · 2007 · 173 citations · Full text

Life Sciences, Molecular Biology, Molecular Genetics +10

A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement

Sara J. Bowne, Marian M. Humphries, Lori S. Sullivan et al. · European Journal of Human Genetics · 2011 · 155 citations · Full text

Life Sciences, Genetics, Molecular Genetics +12

Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

Sophia Millington‐Ward, Naomi Chadderton, Mary O’Reilly et al. · Molecular Therapy · 2011 · 141 citations · Full text

Medicine, Genetics, Ophthalmology +17