|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
|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
|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
|Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo|
H Nakai, SR Yant, TA Storm, S Fuess, L Meuse, MA Kay
Journal of virology 75 (15), 6969-6976, 2001
|AAV serotype 2 vectors preferentially integrate into active genes in mice|
H Nakai, E Montini, S Fuess, TA Storm, M Grompe, MA Kay
Nature genetics 34 (3), 297-302, 2003
|Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice|
H Nakai, S Fuess, TA Storm, S Muramatsu, Y Nara, MA Kay
Journal of virology 79 (1), 214-224, 2005
|Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy|
D Grimm, S Zhou, H Nakai, CE Thomas, TA Storm, S Fuess, T Matsushita, ...
Blood 102 (7), 2412-2419, 2003
|Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors|
H Nakai, TA Storm, MA Kay
Nature biotechnology 18 (5), 527-532, 2000
|Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver|
H Nakai, RW Herzog, JN Hagstrom, J Walter, SH Kung, EY Yang, SJ Tai, ...
Blood, The Journal of the American Society of Hematology 91 (12), 4600-4607, 1998
|Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo|
H Nakai, TA Storm, MA Kay
Journal of virology 74 (20), 9451-9463, 2000
|Sustained survival of human hepatocytes in mice: a model for in vivo infection with human hepatitis B and hepatitis delta viruses|
K Ohashi, PL Marion, H Nakai, L Meuse, JM Cullen, BB Bordier, ...
Nature medicine 6 (3), 327-331, 2000
|Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver|
H Nakai, Y Iwaki, MA Kay, LB Couto
Journal of virology 73 (7), 5438-5447, 1999
|Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver|
H Nakai, X Wu, S Fuess, TA Storm, D Munroe, E Montini, SM Burgess, ...
Journal of virology 79 (6), 3606-3614, 2005
|Characterization of genome integrity for oversized recombinant AAV vector|
B Dong, H Nakai, W Xiao
Molecular therapy 18 (1), 87-92, 2010
|Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction|
CH Miao, H Nakai, AR Thompson, TA Storm, W Chiu, RO Snyder, MA Kay
Journal of virology 74 (8), 3793-3803, 2000
|Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein|
M Burton, H Nakai, P Colosi, J Cunningham, R Mitchell, L Couto
Proceedings of the National Academy of Sciences 96 (22), 12725-12730, 1999
|The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver|
AP McCaffrey, P Fawcett, H Nakai, RL McCaffrey, A Ehrhardt, TTT Pham, ...
Molecular Therapy 16 (5), 931-941, 2008
|TP53 mutations emerge at early phase of myelodysplastic syndrome and are associated with complex chromosomal abnormalities|
H Kaneko, S Misawa, S Horiike, H Nakai, K Kashima
|Gene delivery to human chondrocytes by an adeno associated virus vector.|
Y Arai, T Kubo, S Fushiki, O Mazda, H Nakai, Y Iwaki, J Imanishi, ...
The Journal of rheumatology 27 (4), 979, 2000
|Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype|
D Grimm, K Pandey, H Nakai, TA Storm, MA Kay
Journal of virology 80 (1), 426-439, 2006