Effective delivery of large genes to the retina by dual AAV vectors I Trapani, P Colella, A Sommella, C Iodice, G Cesi, S de Simone, ... EMBO molecular medicine 6 (2), 194-211, 2014 | 260 | 2014 |
RNA interference–mediated suppression and replacement of human rhodopsin in vivo M O’Reilly, A Palfi, N Chadderton, S Millington-Ward, M Ader, T Cronin, ... The American Journal of Human Genetics 81 (1), 127-135, 2007 | 223 | 2007 |
Identification of an IMPDH1 mutation in autosomal dominant retinitis pigmentosa (RP10) revealed following comparative microarray analysis of transcripts derived from retinas of … A Kennan, A Aherne, A Palfi, M Humphries, A McKee, A Stitt, ... Human molecular genetics 11 (5), 547-558, 2002 | 212 | 2002 |
Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa S Millington-Ward, N Chadderton, M O'reilly, A Palfi, T Goldmann, C Kilty, ... Molecular therapy 19 (4), 642-649, 2011 | 176 | 2011 |
Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy N Chadderton, S Millington-Ward, A Palfi, M O'reilly, G Tuohy, ... Molecular Therapy 17 (4), 593-599, 2009 | 176 | 2009 |
Altered retinal microRNA expression profile in a mouse model of retinitis pigmentosa CJ Loscher, K Hokamp, PF Kenna, AC Ivens, P Humphries, A Palfi, ... Genome biology 8, 1-12, 2007 | 173 | 2007 |
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement SJ Bowne, MM Humphries, LS Sullivan, PF Kenna, L Tam, AS Kiang, ... European journal of human genetics 19 (10), 1074-1081, 2011 | 167 | 2011 |
Modeling and rescue of RP2 retinitis pigmentosa using iPSC-derived retinal organoids A Lane, K Jovanovic, C Shortall, D Ottaviani, AB Panes, N Schwarz, ... Stem cell reports 15 (1), 67-79, 2020 | 131 | 2020 |
Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach AS Kiang, A Palfi, M Ader, PF Kenna, S Millington-Ward, G Clark, ... Molecular Therapy 12 (3), 555-561, 2005 | 124 | 2005 |
Strategems in Vitro for Gene Therapies Directed to Dominant Mutations S Millington-Ward, B O'Neill, G Tuohy, N Al-Jandal, AS Kiang, PF Kenna, ... Human Molecular Genetics 6 (9), 1415-1426, 1997 | 111 | 1997 |
A common microRNA signature in mouse models of retinal degeneration CJ Loscher, K Hokamp, JH Wilson, T Li, P Humphries, GJ Farrar, A Palfi Experimental eye research 87 (6), 529-534, 2008 | 95 | 2008 |
Toward an elucidation of the molecular genetics of inherited retinal degenerations GJ Farrar, M Carrigan, A Dockery, S Millington-Ward, A Palfi, ... Human molecular genetics 26 (R1), R2-R11, 2017 | 83 | 2017 |
Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy N Chadderton, A Palfi, S Millington-Ward, O Gobbo, N Overlack, ... European Journal of Human Genetics 21 (1), 62-68, 2013 | 83 | 2013 |
Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene A Palfi, S Millington-Ward, N Chadderton, M O'Reilly, T Goldmann, ... Human gene therapy 21 (3), 311-323, 2010 | 70 | 2010 |
Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10) LCS Tam, AS Kiang, A Kennan, PF Kenna, N Chadderton, M Ader, A Palfi, ... Human molecular genetics 17 (14), 2084-2100, 2008 | 66 | 2008 |
RNAi‐based suppression and replacement of rds‐peripherin in retinal organotypic culture A Palfi, M Ader, AS Kiang, S Millington‐Ward, G Clark, M O'Reilly, ... Human mutation 27 (3), 260-268, 2006 | 66 | 2006 |
Differential calmodulin gene expression in the rodent brain A Palfi, E Kortvely, E Fekete, B Kovacs, S Varszegi, K Gulya Life sciences 70 (24), 2829-2855, 2002 | 52 | 2002 |
RNAi of COL1A1 in mesenchymal progenitor cells S Millington-Ward, HP McMahon, D Allen, G Tuohy, AS Kiang, A Palfi, ... European journal of human genetics 12 (10), 864-866, 2004 | 51 | 2004 |
Murine model of autosomal dominant retinitis pigmentosa generated by targeted deletion at codon 307 of the rds–peripherin gene N McNally, PF Kenna, D Rancourt, T Ahmed, A Stitt, WH Colledge, ... Human Molecular Genetics 11 (9), 1005-1016, 2002 | 51 | 2002 |
AAV‐mediated chronic over‐expression of SNAP‐25 in adult rat dorsal hippocampus impairs memory‐associated synaptic plasticity AG McKee, JS Loscher, NC O’Sullivan, N Chadderton, A Palfi, L Batti, ... Journal of neurochemistry 112 (4), 991-1004, 2010 | 46 | 2010 |