| Progress and problems with the use of viral vectors for gene therapy CE Thomas, A Ehrhardt, MA Kay Nature Reviews Genetics 4 (5), 346-358, 2003 | 3369 | 2003 |
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response CS Manno, GF Pierce, VR Arruda, B Glader, M Ragni, JJE Rasko, ... Nature medicine 12 (3), 342-347, 2006 | 2476 | 2006 |
| Adenovirus-associated virus vector–mediated gene transfer in hemophilia B AC Nathwani, EGD Tuddenham, S Rangarajan, C Rosales, J McIntosh, ... New England Journal of Medicine 365 (25), 2357-2365, 2011 | 2211 | 2011 |
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways D Grimm, KL Streetz, CL Jopling, TA Storm, K Pandey, CR Davis, ... Nature 441 (7092), 537-541, 2006 | 2135 | 2006 |
| Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics MA Kay, JC Glorioso, L Naldini Nature medicine 7 (1), 33-40, 2001 | 1892 | 2001 |
| RNA interference in adult mice AP McCaffrey, L Meuse, TTT Pham, DS Conklin, GJ Hannon, MA Kay Nature 418 (6893), 38-39, 2002 | 1687 | 2002 |
| Long-term safety and efficacy of factor IX gene therapy in hemophilia B AC Nathwani, UM Reiss, EGD Tuddenham, C Rosales, P Chowdary, ... New England Journal of Medicine 371 (21), 1994-2004, 2014 | 1437 | 2014 |
| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector MA Kay, CS Manno, MV Ragni, PJ Larson, LB Couto, A McClelland, ... Nature genetics 24 (3), 257-261, 2000 | 1332 | 2000 |
| Inhibition of hepatitis B virus in mice by RNA interference AP McCaffrey, H Nakai, K Pandey, Z Huang, FH Salazar, H Xu, ... Nature biotechnology 21 (6), 639-644, 2003 | 996 | 2003 |
| A nonviral minicircle vector for deriving human iPS cells F Jia, KD Wilson, N Sun, DM Gupta, M Huang, Z Li, NJ Panetta, ZY Chen, ... Nature methods 7 (3), 197-199, 2010 | 970 | 2010 |
| Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice H Azuma, N Paulk, A Ranade, C Dorrell, M Al-Dhalimy, E Ellis, S Strom, ... Nature biotechnology 25 (8), 903-910, 2007 | 922 | 2007 |
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B CS Manno, AJ Chew, S Hutchison, PJ Larson, RW Herzog, VR Arruda, ... Blood, The Journal of the American Society of Hematology 101 (8), 2963-2972, 2003 | 918 | 2003 |
| State-of-the-art gene-based therapies: the road ahead MA Kay Nature Reviews Genetics 12 (5), 316-328, 2011 | 867 | 2011 |
| In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses D Grimm, JS Lee, L Wang, T Desai, B Akache, TA Storm, MA Kay Journal of virology 82 (12), 5887-5911, 2008 | 861 | 2008 |
| Sarcoma derived from cultured mesenchymal stem cells J Tolar, AJ Nauta, MJ Osborn, A Panoskaltsis Mortari, RT McElmurry, ... Stem cells 25 (2), 371-379, 2007 | 830 | 2007 |
| In vivo activity of nuclease-resistant siRNAs JM Layzer, AP McCaffrey, AK Tanner, ZAN Huang, MA Kay, BA Sullenger Rna 10 (5), 766-771, 2004 | 785 | 2004 |
| Human tRNA-derived small RNAs in the global regulation of RNA silencing D Haussecker, Y Huang, A Lau, P Parameswaran, AZ Fire, MA Kay Rna 16 (4), 673-695, 2010 | 762 | 2010 |
| Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors RO Snyder, CH Miao, GA Patijn, SK Spratt, O Danos, D Nagy, AM Gown, ... Nature genetics 16 (3), 270-276, 1997 | 747 | 1997 |
| Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8 K Inagaki, S Fuess, TA Storm, GA Gibson, CF Mctiernan, MA Kay, H Nakai Molecular Therapy 14 (1), 45-53, 2006 | 741 | 2006 |
| Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system SR Yant, L Meuse, W Chiu, Z Ivics, Z Izsvak, MA Kay Nature genetics 25 (1), 35-41, 2000 | 636 | 2000 |
Hiroyuki NakaiDistinguished Professor of Molecular Medicine, Department of Molecular & Medical Genetics, OregonVerified email at ohsu.edu
