| Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis SH Cheng, RJ Gregory, J Marshall, S Paul, DW Souza, GA White, ... Cell 63 (4), 827-834, 1990 | 2163 | 1990 |
| PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo CR O'Riordan, A Lachapelle, C Delgado, V Parkes, SC Wadsworth, ... Human gene therapy 10 (8), 1349-1358, 1999 | 509 | 1999 |
| CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy MA Passini, J Bu, EM Roskelley, AM Richards, SP Sardi, CR O’Riordan, ... The Journal of clinical investigation 120 (4), 1253-1264, 2010 | 344 | 2010 |
| Modification of an adenoviral vector with biologically selected peptides: a novel strategy for gene delivery to cells of choice H Romanczuk, CE Galer, J Zabner, G Barsomian, SC Wadsworth, ... Human gene therapy 10 (16), 2615-2626, 1999 | 191 | 1999 |
| AAV vector-mediated correction of brain pathology in a mouse model of Niemann–Pick A disease MA Passini, SL Macauley, MR Huff, TV Taksir, J Bu, IH Wu, ... Molecular Therapy 11 (5), 754-762, 2005 | 165 | 2005 |
| Targeting adenoviral vectors using heterofunctional polyethylene glycol FGF2 conjugates J Lanciotti, A Song, J Doukas, B Sosnowski, G Pierce, R Gregory, ... Molecular Therapy 8 (1), 99-107, 2003 | 156 | 2003 |
| Analytical ultracentrifugation as an approach to characterize recombinant adeno-associated viral vectors B Burnham, S Nass, E Kong, ME Mattingly, D Woodcock, A Song, ... Human gene therapy methods 26 (6), 228-242, 2015 | 150 | 2015 |
| Stoichiometry of recombinant cystic fibrosis transmembrane conductance regulator in epithelial cells and its functional reconstitution into cells in vitro. J Marshall, S Fang, LS Ostedgaard, CR O'Riordan, D Ferrara, JF Amara, ... Journal of Biological Chemistry 269 (4), 2987-2995, 1994 | 150 | 1994 |
| Universal method for the purification of recombinant AAV vectors of differing serotypes SA Nass, MA Mattingly, DA Woodcock, BL Burnham, JA Ardinger, ... Molecular Therapy Methods & Clinical Development 9, 33-46, 2018 | 127 | 2018 |
| Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease JC Dodge, J Clarke, A Song, J Bu, W Yang, TV Taksir, D Griffiths, ... Proceedings of the National Academy of Sciences 102 (49), 17822-17827, 2005 | 113 | 2005 |
| Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy MA Passini, J Bu, AM Richards, CM Treleaven, JA Sullivan, CR O'Riordan, ... Human gene therapy 25 (7), 619-630, 2014 | 111 | 2014 |
| Scaleable chromatographic purification process for recombinant adeno‐associated virus (rAAV) CR O'Riordan, AL Lachapelle, KA Vincent, SC Wadsworth The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2000 | 94 | 2000 |
| Gene therapy for spinal cord disorders J Dodge, L Shihabuddin, M Passini, SH Cheng, C O'riordan US Patent 10,744,210, 2020 | 85 | 2020 |
| Nucleic acid delivery vehicles CR O'riordan, SC Wadsworth US Patent 6,287,857, 2001 | 83 | 2001 |
| Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia PT Drapkin, CR O’Riordan, SM Yi, JA Chiorini, J Cardella, J Zabner, ... The Journal of clinical investigation 105 (5), 589-596, 2000 | 83 | 2000 |
| Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse MA Passini, J Bu, JA Fidler, RJ Ziegler, JW Foley, JC Dodge, WW Yang, ... Proceedings of the National Academy of Sciences 104 (22), 9505-9510, 2007 | 81 | 2007 |
| Cyclic AMP-dependent protein kinase activation of cystic fibrosis transmembrane conductance regulator chloride channels in planar lipid bilayers. BC Tilly, MC Winter, LS Ostedgaard, C O'Riordan, AE Smith, MJ Welsh Journal of Biological Chemistry 267 (14), 9470-9473, 1992 | 73 | 1992 |
| Extensive transduction and enhanced spread of a modified AAV2 capsid in the non-human primate CNS J Naidoo, LM Stanek, K Ohno, S Trewman, L Samaranch, P Hadaczek, ... Molecular Therapy 26 (10), 2418-2430, 2018 | 61 | 2018 |
| Electrodiffusional ATP movement through the cystic fibrosis transmembrane conductance regulator HF Cantiello, GR Jackson Jr, CF Grosman, AG Prat, SC Borkan, Y Wang, ... American Journal of Physiology-Cell Physiology 274 (3), C799-C809, 1998 | 61 | 1998 |
| Direct liquid chromatography/mass spectrometry analysis for complete characterization of recombinant adeno-associated virus capsid proteins X Jin, L Liu, S Nass, C O'Riordan, E Pastor, XK Zhang Human Gene Therapy Methods 28 (5), 255-267, 2017 | 58 | 2017 |
