Research output per year
Research output per year
- 1 - 50 out of 83 results
Search results
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2022
AAV-vector based gene therapy for mitochondrial disease: progress and future perspectives
Hanaford, A. R., Cho, Y. J. & Nakai, H., Dec 2022, In: Orphanet journal of rare diseases. 17, 1, 217.Research output: Contribution to journal › Review article › peer-review
Open Access8 Scopus citations -
Expression of a Secretable, Cell-Penetrating CDKL5 Protein Enhances the Efficacy of Gene Therapy for CDKL5 Deficiency Disorder
Medici, G., Tassinari, M., Galvani, G., Bastianini, S., Gennaccaro, L., Loi, M., Mottolese, N., Alvente, S., Berteotti, C., Sagona, G., Lupori, L., Candini, G., Baggett, H. R., Zoccoli, G., Giustetto, M., Muotri, A., Pizzorusso, T., Nakai, H., Trazzi, S. & Ciani, E., Oct 2022, In: Neurotherapeutics. 19, 6, p. 1886-1904 19 p.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Multicolor labeling of airway neurons and analysis of parasympathetic heterogeneity
Pincus, A. B., Huang, S. J., Lebold, K. M., De La Torre, U., Proskocil, B. J., Drake, M. G., Nakai, H., Fryer, A. D. & Jacoby, D. B., Dec 2022, In: Scientific Reports. 12, 1, 5006.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
2020
Adeno-associated virus-binding antibodies detected in cats living in the Northeastern United States lack neutralizing activity
Adachi, K., Dissen, G. A., Lomniczi, A., Xie, Q., Ojeda, S. R. & Nakai, H., Dec 1 2020, In: Scientific Reports. 10, 1, 10073.Research output: Contribution to journal › Article › peer-review
Open Access6 Scopus citations -
In Vivo Repair of a Protein Underlying a Neurological Disorder by Programmable RNA Editing
Sinnamon, J. R., Kim, S. Y., Fisk, J. R., Song, Z., Nakai, H., Jeng, S., McWeeney, S. K. & Mandel, G., Jul 14 2020, In: Cell Reports. 32, 2, 107878.Research output: Contribution to journal › Article › peer-review
Open Access41 Scopus citations -
2019
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Cabanes-Creus, M., Ginn, S. L., Amaya, A. K., Liao, S. H. Y., Westhaus, A., Hallwirth, C. V., Wilmott, P., Ward, J., Dilworth, K. L., Santilli, G., Rybicki, A., Nakai, H., Thrasher, A. J., Filip, A. C., Alexander, I. E. & Lisowski, L., Mar 15 2019, In: Molecular Therapy Methods and Clinical Development. 12, p. 71-84 14 p.Research output: Contribution to journal › Article › peer-review
Open Access23 Scopus citations -
2018
A quantitative dot blot assay for AAV titration and its use for functional assessment of the adeno-associated virus assembly-activating proteins
Powers, J. M., Chang, X. L., Song, Z. & Nakai, H., Jun 12 2018, In: Journal of Visualized Experiments. 2018, 136, e56766.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
2017
Adeno-associated virus (AAV) assembly-activating protein is not an essential requirement for capsid assembly of AAV serotypes 4, 5, and 11
Earley, L. F., Powers, J. M., Adachi, K., Baumgart, J. T., Meyer, N. L., Xie, Q., Chapman, M. S. & Nakai, H., 2017, In: Journal of virology. 91, 3, e01980-16.Research output: Contribution to journal › Article › peer-review
Open Access67 Scopus citations -
Engineering a gene silencing viral construct that targets the cat hypothalamus to induce permanent sterility: An update
Dissen, G. A., Adachi, K., Lomniczi, A., Chatkupt, T., Davidson, B. L., Nakai, H. & Ojeda, S. R., Apr 1 2017, In: Reproduction in Domestic Animals. 52, p. 354-358 5 p.Research output: Contribution to journal › Article › peer-review
Open Access9 Scopus citations -
Site-directed RNA repair of endogenous Mecp2 RNA in neurons
Sinnamon, J. R., Kim, S. Y., Corson, G. M., Song, Z., Nakai, H., Adelman, J. P. & Mandel, G., Oct 31 2017, In: Proceedings of the National Academy of Sciences of the United States of America. 114, 44, p. E9395-E9402Research output: Contribution to journal › Article › peer-review
Open Access73 Scopus citations -
2015
Identification and characterization of nuclear and nucleolar localization signals in the adeno-associated virus serotype 2 assembly-activating protein
Earley, L. F., Kawano, Y., Adachi, K., Sun, X. X., Dai, M. S. & Nakai, H., 2015, In: Journal of virology. 89, 6, p. 3038-3048 11 p.Research output: Contribution to journal › Article › peer-review
Open Access30 Scopus citations -
2014
Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing
Adachi, K., Enoki, T., Kawano, Y., Veraz, M. & Nakai, H., Jan 17 2014, In: Nature communications. 5, 3075.Research output: Contribution to journal › Article › peer-review
Open Access102 Scopus citations -
2013
An Experimental and Computational Evolution-Based Method to Study a Mode of Co-evolution of Overlapping Open Reading Frames in the AAV2 Viral Genome
Kawano, Y., Neeley, S., Adachi, K. & Nakai, H., Jun 24 2013, In: PloS one. 8, 6, e66211.Research output: Contribution to journal › Article › peer-review
Open Access12 Scopus citations -
Intraganglionic AAV6 Results in Efficient and Long-Term Gene Transfer to Peripheral Sensory Nervous System in Adult Rats
Yu, H., Fischer, G., Ferhatovic, L., Fan, F., Light, A. R., Weihrauch, D., Sapunar, D., Nakai, H., Park, F. & Hogan, Q. H., Apr 16 2013, In: PloS one. 8, 4, e61266.Research output: Contribution to journal › Article › peer-review
Open Access38 Scopus citations -
2012
AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence
Wang, Z., Lisowski, L., Finegold, M. J., Nakai, H., Kay, M. A. & Grompe, M., Oct 2012, In: Molecular Therapy. 20, 10, p. 1902-1911 10 p.Research output: Contribution to journal › Article › peer-review
Open Access35 Scopus citations -
Adeno-associated virus serotype 8 (AAV8) delivery of recombinant A20 to skeletal muscle reduces pathological Activation of Nuclear Factor (NF)-κB in muscle of mdx mice
Charan, R. A., Niizawa, G., Nakai, H. & Clemens, P. R., Nov 6 2012, In: Molecular Medicine. 18, 12, p. 1527-1535 9 p.Research output: Contribution to journal › Article › peer-review
Open Access9 Scopus citations -
2011
A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction
Kotchey, N. M., Adachi, K., Zahid, M., Inagaki, K., Charan, R., Parker, R. S. & Nakai, H., Jun 2011, In: Molecular Therapy. 19, 6, p. 1079-1089 11 p.Research output: Contribution to journal › Article › peer-review
Open Access60 Scopus citations -
Direct injection into the dorsal root ganglion: Technical, behavioral, and histological observations
Fischer, G., Kostic, S., Nakai, H., Park, F., Sapunar, D., Yu, H. & Hogan, Q., Jul 15 2011, In: Journal of Neuroscience Methods. 199, 1, p. 43-55 13 p.Research output: Contribution to journal › Article › peer-review
Open Access74 Scopus citations -
Erratum: A new recombinant adeno-associated virus (AAV)-based random peptide display library system: Infection-defective AAV1.9-3 as a novel detargeted platform for vector evolution (Gene Therapy and Regulation (2010) 5 (31-55) DOI: 10.1142/S1568558610000197)
Adachi, K. & Nakai, H., Mar 2011, In: Gene Therapy and Regulation. 6, 1, p. 75 1 p.Research output: Contribution to journal › Comment/debate › peer-review
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2010
A new recombinant adeno-associated virus (AAV)-based random peptide display library system: Infection-defective AAV1.9-3 as a novel detargeted platform for vector evolution
Adachi, K. & Nakai, H., Oct 2010, In: Gene Therapy and Regulation. 5, 1, p. 31-55 25 p.Research output: Contribution to journal › Article › peer-review
Open Access14 Scopus citations -
Characterization of genome integrity for oversized recombinant AAV vector
Dong, B., Nakai, H. & Xiao, W., Jan 2010, In: Molecular Therapy. 18, 1, p. 87-92 6 p.Research output: Contribution to journal › Article › peer-review
Open Access184 Scopus citations -
Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunity
Eghtesad, S., Zheng, H., Nakai, H., Epperly, M. W. & Clemens, P. R., Sep 2010, In: Gene therapy. 17, 9, p. 1181-1190 10 p.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
2009
Rapidly evolving adeno-associated virus vectors: Increasing possibility of gene therapy with custom-made vectors
Nakai, H., 2009, In: Drug Delivery System. 24, 6, p. 582-591 10 p.Research output: Contribution to journal › Article › peer-review
Open Access -
2008
Efficient and Durable Gene Transfer to Transplanted Heart Using Adeno-associated Virus 9 Vector
Miyagi, N., Rao, V. P., Ricci, D., Du, Z., Byrne, G. W., Bailey, K. R., Nakai, H., Russell, S. J. & McGregor, C. G. A., May 2008, In: Journal of Heart and Lung Transplantation. 27, 5, p. 554-560 7 p.Research output: Contribution to journal › Article › peer-review
Open Access24 Scopus citations -
Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver
Inagaki, K., Piao, C., Kotchey, N. M., Wu, X. & Nakai, H., Oct 2008, In: Journal of virology. 82, 19, p. 9513-9524 12 p.Research output: Contribution to journal › Article › peer-review
Open Access47 Scopus citations -
Recombinant adeno-associated virus type 8-mediated extensive therapeutic gene delivery into skeletal muscle of α-sarcoglycan-deficient mice
Nishiyama, A., Ampong, B. N., Ohshima, S., Shin, J. H., Nakai, H., Imamura, M., Miyagoe-Suzuki, Y., Okada, T. & Takeda, S., Jul 1 2008, In: Human Gene Therapy. 19, 7, p. 719-730 12 p.Research output: Contribution to journal › Article › peer-review
10 Scopus citations -
Simulation analysis of intracellular Na+ and Cl- homeostasis during β1-adrenergic stimulation of cardiac myocyte
Kuzumoto, M., Takeuchi, A., Nakai, H., Oka, C., Noma, A. & Matsuoka, S., Jan 2008, In: Progress in Biophysics and Molecular Biology. 96, 1-3, p. 171-186 16 p.Research output: Contribution to journal › Review article › peer-review
Open Access52 Scopus citations -
The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver
McCaffrey, A. P., Fawcett, P., Nakai, H., McCaffrey, R. L., Ehrhardt, A., Pham, T. T. T., Pandey, K., Xu, H., Feuss, S., Storm, T. A. & Kay, M. A., May 2008, In: Molecular Therapy. 16, 5, p. 931-941 11 p.Research output: Contribution to journal › Article › peer-review
Open Access84 Scopus citations -
2007
DNA palindromes with a modest arm length of ≳20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice
Inagaki, K., Lewis, S. M., Wu, X., Ma, C., Munroe, D. J., Fuess, S., Storm, T. A., Kay, M. A. & Nakai, H., Oct 2007, In: Journal of virology. 81, 20, p. 11290-11303 14 p.Research output: Contribution to journal › Article › peer-review
Open Access51 Scopus citations -
The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice
Inagaki, K., Ma, C., Storm, T. A., Kay, M. A. & Nakai, H., Oct 2007, In: Journal of virology. 81, 20, p. 11304-11321 18 p.Research output: Contribution to journal › Article › peer-review
Open Access50 Scopus citations -
2006
Erratum: Successful transduction of liver in hemophilia by AAV- factor IX and limitations imposed by the host immune response (Nature Medicine (2006) 12 (342-347))
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P. K., Nakai, H., Chew, A., Leonard, D., Wright, J. F., & 11 others, May 2006, In: Nature medicine. 12, 5, p. 592 1 p.Research output: Contribution to journal › Comment/debate › peer-review
Open Access4 Scopus citations -
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype
Grimm, D., Pandey, K., Nakai, H., Storm, T. A. & Kay, M. A., Jan 2006, In: Journal of virology. 80, 1, p. 426-439 14 p.Research output: Contribution to journal › Article › peer-review
Open Access92 Scopus citations -
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8
Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A. & Nakai, H., Jul 2006, In: Molecular Therapy. 14, 1, p. 45-53 9 p.Research output: Contribution to journal › Article › peer-review
Open Access485 Scopus citations -
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Manno, C. S., Arruda, V. R., Pierce, G. F., Glader, B., Ragni, M., Rasko, J., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Nakai, H., Chew, A., Leonard, D., Wright, J. F., Lessard, R. R., & 10 others, Mar 2006, In: Nature medicine. 12, 3, p. 342-347 6 p.Research output: Contribution to journal › Article › peer-review
1706 Scopus citations -
2005
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver
Nakai, H., Wu, X., Fuess, S., Storm, T. A., Munroe, D., Montini, E., Burgess, S. M., Grompe, M. & Kay, M. A., Mar 2005, In: Journal of virology. 79, 6, p. 3606-3614 9 p.Research output: Contribution to journal › Article › peer-review
Open Access151 Scopus citations -
Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver
Ohashi, K., Nakai, H., Couto, L. B. & Kay, M. A., Mar 2005, In: Human Gene Therapy. 16, 3, p. 299-306 8 p.Research output: Contribution to journal › Article › peer-review
14 Scopus citations -
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
Nakai, H., Fuess, S., Storm, T. A., Muramatsu, S. I., Nara, Y. & Kay, M. A., Jan 2005, In: Journal of virology. 79, 1, p. 214-224 11 p.Research output: Contribution to journal › Article › peer-review
Open Access262 Scopus citations -
2003
AAV serotype 2 vectors preferentially integrate into active genes in mice
Nakai, H., Montini, E., Fuess, S., Storm, T. A., Grompe, M. & Kay, M. A., Jul 1 2003, In: Nature genetics. 34, 3, p. 297-302 6 p.Research output: Contribution to journal › Article › peer-review
340 Scopus citations -
Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo
Nakai, H., Fuess, S., Storm, T. A., Meuse, L. A. & Kay, M. A., Jan 1 2003, In: Molecular Therapy. 7, 1, p. 112-121 10 p.Research output: Contribution to journal › Article › peer-review
Open Access23 Scopus citations -
Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice
Nakai, H., Montini, E., Fuess, S., Storm, T. A., Meuse, L., Finegold, M., Grompe, M. & Kay, M. A., Jan 1 2003, In: Molecular Therapy. 7, 1, p. 101-111 11 p.Research output: Contribution to journal › Article › peer-review
Open Access47 Scopus citations -
Inhibition of hepatitis B virus in mice by RNA interference
McCaffrey, A. P., Nakai, H., Pandey, K., Huang, Z., Salazar, F. H., Xu, H., Wieland, S. F., Marion, P. L. & Kay, M. A., Jun 1 2003, In: Nature biotechnology. 21, 6, p. 639-644 6 p.Research output: Contribution to journal › Article › peer-review
594 Scopus citations -
Looking into the safety of AAV vectors [2]
Kay, M. A. & Nakai, H., Jul 17 2003, In: Nature. 424, 6946, p. 251 1 p.Research output: Contribution to journal › Letter › peer-review
Open Access61 Scopus citations -
Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors
Nakai, H., Storm, T. A., Fuess, S. & Kay, M. A., Jun 10 2003, In: Human Gene Therapy. 14, 9, p. 871-881 11 p.Research output: Contribution to journal › Article › peer-review
26 Scopus citations -
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy
Grimm, D., Zhou, S., Nakai, H., Thomas, C. E., Storm, T. A., Fuess, S., Matsushita, T., Allen, J., Surosky, R., Lochrie, M., Meuse, L., McClelland, A., Colosi, P. & Kay, M. A., Oct 1 2003, In: Blood. 102, 7, p. 2412-2419 8 p.Research output: Contribution to journal › Article › peer-review
192 Scopus citations -
2002
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction
Nakai, H., Thomas, C. E., Storm, T. A., Fuess, S., Powell, S., Wright, J. F. & Kay, M. A., Nov 2002, In: Journal of virology. 76, 22, p. 11343-11349 7 p.Research output: Contribution to journal › Article › peer-review
Open Access83 Scopus citations -
2001
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
Nakai, H., Yant, S. R., Storm, T. A., Fuess, S., Meuse, L. & Kay, M. A., 2001, In: Journal of virology. 75, 15, p. 6969-6976 8 p.Research output: Contribution to journal › Article › peer-review
Open Access390 Scopus citations -
Young Investigator Award
Nakai, H., 2001, In: The journal of gene medicine. 3, 6, p. 599-600 2 p.Research output: Contribution to journal › Article › peer-review
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2000
Gene delivery to human chondrocytes by an adeno associated virus vector
Arai, Y., Kubo, T., Fushiki, S., Mazda, O., Nakai, H., Iwaki, Y., Imanishi, J. & Hirasawa, Y., 2000, In: Journal of Rheumatology. 27, 4, p. 979-982 4 p.Research output: Contribution to journal › Article › peer-review
76 Scopus citations -
Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
Nakai, H., Storm, T. A. & Kay, M. A., May 2000, In: Nature biotechnology. 18, 5, p. 527-532 6 p.Research output: Contribution to journal › Article › peer-review
183 Scopus citations -
Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: Cell cycling does not influence hepatocyte transduction
Miao, C. H., Nakai, H., Thompson, A. R., Storm, T. A., Chiu, W., Snyder, R. O. & Kay, M. A., 2000, In: Journal of virology. 74, 8, p. 3793-3803 11 p.Research output: Contribution to journal › Article › peer-review
Open Access117 Scopus citations