Publications 論文

Sukegawa M, Miyagawa Y, Kuroda S, Yamazaki Y, Yamamoto M, Adachi K, Sato H, Sato Y, Taniai N, Yoshida H, Umezawa A, Sakai M, Okada T. Mesenchymal stem cell origin contributes to the antitumor effect of oncolytic virus carriers. Mol Ther Oncol. 32(4):200896. doi: 10.1016/j.omton.2024.200896. eCollection 2024 Dec 19.

Kardani K, Ghouse SM, Jabbar MAD, Rajasubramanian N, Gil JS, Stemmer-Rachamimov A, Soda Y, Martuza RL, Hara T, Wakimoto H, Rabkin SH. Immunocompetent murine glioblastoma stem-like cell models exhibiting distinct phenotypes. Neurooncol Adv. 2024 Dec 7;7(1):vdae215. doi: 10.1093/noajnl/vdae215. eCollection 2025 Jan-Dec. PMID: 39896074

Nakamura N, Kanda J, Kondo T, Kitano T, Ikeda T, Imada K, Takaya R, Kubo T, Mitsuyuki S, Oka S, Yonezawa A, Takeoka T, Akasaka T, Hishizawa M, Yago K, Tsunemine H, Watanabe M, Itoh M, Takaori-Kondo A; Kyoto Stem Cell Transplantation Group (KSCTG). Comparison of methotrexate dosing protocols for graft-versus-host disease prophylaxis after unrelated hematopoietic stem cell transplantation. Cytotherapy. 2024 Nov 17:S1465-3249(24)00935-6. doi: 10.1016/j.jcyt.2024.11.009. Epub ahead of print. PMID: 39652019.

Onishi A, Tsunekawa Y, Mandai M, Ishimaru A, Ohigashi Y, Sho J, Yasuda K, Suzuki K, Izpisua Belmonte JC, Matsuzaki F, Takahashi M. Optimization of HITI-Mediated Gene Insertion for Rhodopsin and Peripherin-2 in Mouse Rod Photoreceptors: Targeting Dominant Retinitis Pigmentosa. Invest Ophthalmol Vis Sci. 65(13):38. doi: 10.1167/iovs.65.13.38. 2024 Nov 4.

Hamada R, Arai Y, Kitawaki T, Nakamura N, Murao M, Matsushita M, Miyasaka J, Asano T, Jo T, Nishikori M, Kanda J, Mizumoto C, Yamashita K, Ikeguchi R, Takaori-Kondo A. Fluctuation of physical function during chimeric antigen receptor T-cell therapy during rehabilitation intervention: Real-world data and risk factor analyses. EJHaem. 2024 Nov 4;5(6):1252-1259. doi: 10.1002/jha2.1043. PMID: 39691237; PMCID: PMC11647737.

Nakamura N, Tsunemine H, Ikunari R, Sakai T, Arima N. COVID-19 antibody titers after tixagevimab-cilgavimab injection in patients with hematologic diseases; a single-center, prospective study. Leuk Lymphoma. 2024 Aug;65(8):1117-1126. doi: 10.1080/10428194.2024.2343519. Epub 2024 Apr 16. PMID: 38626450.

Nakamura N, Ikunari R, Tanaka Y, Tsunemine H, Takeda J, Arima N. Pathogenic TNFRSF13B Variant in an Adult Japanese Patient with Common Variable Immunodeficiency. Intern Med. 2024 Jul 11. doi: 10.2169/internalmedicine.4057-24. Epub ahead of print. PMID: 38987180.

Nitahara-Kasahara Y, Posadas-Herrera G, Hirai K, Oda Y, Snagu-Miyamoto N, Yamanashi Y, Okada T. Characterization of disease-specific alterations in metabolites and effects of mesenchymal stromal cells on dystrophic muscles. Front Cell Dev Biol.(section Stem Cell Research) 12:1363541 doi: 10.3389/fcell.2024.1363541. 2024 Jun 14.

Nakamura N, Yamamoto N, Kondo T, Matsumoto M, Ikunari R, Sakai T, Tanaka Y, Tsunemine H, Takeda J, Kanda J, Nannya Y, Ogawa S, Takaori-Kondo A, Arima N. Sustained remission after cord blood transplantation for breast cancer with lung metastases and myelodysplastic syndrome. Int J Hematol. 2024 Jun;119(6):762-767. doi: 10.1007/s12185-024-03762-8. Epub 2024 Mar 25. PMID: 38523199.

Tsutsui M, Wada M, Arima A, Tsunekawa Y, Sasaki T, Sakamoto K, Yokota K, Baba Y, Kawai T, Okada T. Identifying viral vector characteristics by nanopore sensing. ACS Nano. Jun 18;18(24):15695-15704.　doi: 10.1021/acsnano.4c01888. Epub 2024 Jun 5.

Kurosawa Y, Tsunekawa Y, Wada M, Aizen Y, Nitahara-Kasahara Y, Okada T. Purification of adeno-associated viral vector serotype 9 using ceramic hydroxyapatite chromatography and its analysis. Curr Protoc. 4(6):e1068. doi: 10.1002/cpz1.1068. 2024 Jun.

Nakamura N, Jo T, Arai Y, Kitawaki T, Nishikori M, Mizumoto C, Kanda J, Yamashita K, Nagao M, Takaori-Kondo A. Utilizing red blood cell distribution width (RDW) as a reliable biomarker to predict treatment effects after chimeric antigen receptor T cell therapy. Clin Exp Med. 2024 May 21;24(1):105. doi: 10.1007/s10238-024-01373-5. PMID: 38771501; PMCID: PMC11108946.

Nakamura N, Tsunemine H, Ikunari R, Tanaka Y, Arima N. Red blood cell distribution width is a useful biomarker to predict bleeding and thrombosis risks in patients with immune thrombocytopenic purpura. EJHaem. 2024 Apr 30;5(3):431-439. doi: 10.1002/jha2.897. PMID: 38895062; PMCID: PMC11182403.

Numata J, Tsunemine H, Imai A, Nakamura N, Sakai T, Itoh T, Arima N. Recurrence of solitary plasmacytoma in the liver 10 years after the onset of multiple bone lesions. J Clin Exp Hematop. 2024;64(1):65-70. doi: 10.3960/jslrt.23056. PMID: 38538320; PMCID: PMC11079988. Mar 28 2024

Nakamura N, Arima N, Takakuwa T, Yoshioka S, Imada K, Fukushima K, Hotta M, Fuchida SI, Kanda J, Uoshima N, Shimura Y, Tanaka H, Ohta K, Kosugi S, Yagi H, Yoshihara S, Yamamura R, Adachi Y, Hanamoto H, Shibayama H, Hosen N, Ito T, Shimazaki C, Takaori-Kondo A, Kuroda J, Matsumura I, Hino M; Kansai Myeloma Forum. Efficacy of elotuzumab for multiple myeloma deteriorates after daratumumab: a multicenter retrospective study. Ann Hematol. 2024 Dec;103(12):5681-5690. doi: 10.1007/s00277-024-05705-z. Epub 2024 Mar 16. PMID: 38492020.

Hirohata K, Yamaguchi Y, Maruno T, Shibuya R, Torisu T, Onishi T, Chono H, Mineno J, Yuzhe Y, Higashiyama K, Masumi-Koizumi K, Uchida K, Yamamoto T, Uchida E, Okada T, Uchiyama S. Applications and Limitations of Equilibrium Density Gradient Analytical Ultracentrifugation for the Quantitative Characterization of Adeno-Associated Virus Vectors. Anal Chem. 2024 Jan 16;96(2):642-651. doi: 10.1021/acs.analchem.3c01955. Epub 2024 Jan 2.

Nakamura N, Jo T, Arai Y, Kitawaki T, Nishikori M, Mizumoto C, Kanda J, Yamashita K, Nagao M, Takaori-Kondo A. Clinical Impact of Cytokine Release Syndrome on Prolonged Hematotoxicity after Chimeric Antigen Receptor T Cell Therapy: KyoTox A-Score, a Novel Prediction Model. Transplant Cell Ther. 2024 Apr;30(4):404-414. doi: 10.1016/j.jtct.2024.01.073. Epub 2024 Jan 26. PMID: 38281589.

Jo T, Arai Y, Kitawaki T, Nakamura N, Nishikori M, Mizumoto C, Kanda J, Yamashita K, Nagao M, Takaori-Kondo A. KyoTox-e score; prediction of post-CAR-T prolonged thrombocytopenia using peripheral blood data before apheresis. Bone Marrow Transplant. 2024 Mar;59(3):419-421. doi: 10.1038/s41409-023-02175-1. Epub 2023 Dec 19. PMID: 38110621.

Yoshida K, Tsunekawa Y, Kurihara K, Watanabe K, Makino-Manabe Y, Wada M, Tanaka T, Ide T, Okada T. Engineering a highly durable adeno-associated virus receptor for analytical applications. Mol Ther Methods Clin Dev. 2023 31: 101157 doi.org/10.1016/j.omtm.2023.101157. Dec 14.

Arakawa M, Sakamoto Y, Miyagawa Y, Nito C, Takahashi S, Nitahara-Kasahara Y, Suda S, Yamazaki Y, Sakai M, Kimura K, Okada T. iPSC-derived mesenchymal stem cells attenuate cerebral ischemia-reperfusion injury by inhibiting inflammatory signaling and oxidative stress. Mol Ther Methods Clin Dev. 2023 Jul 15:30:333-349. doi: 10.1016/j.omtm.2023.07.005. eCollection 2023 Sep 14.

Miyaoka R, Tsunekawa Y, Kurosawa Y, Sasaki T, Onodera A, Sakamoto K, Kakiuchi Y, Wada M, Nitahara-Kasahara Y, Hayashita-Kinoh H, Okada T. Development of a novel purification method for AAV vectors using tangential flow filtration. Biotechnol Bioeng., 2023 Nov;120(11):3311-3321. doi: 10.1002/bit.28524. Epub 2023 Aug 16.

Maruyama M, Sakai A, Fukunaga T, Miyagawa Y, Okada T, Hamada M, Suzuki H. Neat1 lncRNA organizes the inflammatory gene expressions in the dorsal root ganglion in neuropathic pain caused by nerve injury. Front. Immunol. 14:1185322. doi.: 10.3389/fimmu.2023.1185322. 2023 Aug 8

Li D, Johmura Y, Morimoto S, Doi M, Nakanishi K, Ozawa M, Tsunekawa Y, Inoue-Yamauchi A, Naruse H, Matsukawa T, Takeshita Y, Suzuki N, Aoki M, Nishiyama A, Zeng X, Konishi C, Suzuki N, Nishiyama A, Harris AS, Morita M, Yamaguchi K, Furukawa Y, Nakai K, Tsuji S, Yamazaki S, Yamanashi Y, Shimada S, Okada T, Okano H, Toda T, Nakanishi M. LONRF2 is a protein quality control ubiquitin ligase whose deficiency causes late-onset neurological deficits. Nat Aging 2023 Aug;3(8):1001-1019.
doi: 10.1038/s43587-023-00464-4. Epub 2023 Jul 20.

Nitahara-Kasahara Y, Nakayama S, Kimura K, Yamaguchi S, Kakiuchi Y, Nito C, Hayashi M, Nakaishi T, Ueda Y, Okada T. Immunomodulatory amnion mesenchymal stem cells preserve muscle function in a mouse model of Duchenne muscular dystrophy. Stem Cell Res Ther. 14(1):108. doi: 10.1186/s13287-023-03337-0. 2023 Apr 27

Wada M, Uchida N, Posadas-Herrera G, Hayashita-Kinoh H, Tsunekawa Y, Hirai Y, Okada T. Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor. Gene Ther. 30(7-8):641-648. doi: 10.1038/s41434-023-00398-x. Epub 2023 Mar 28.

Pilaz LJ, Liu J, Joshi K, Tsunekawa Y, Musso CM, D'Arcy BR, Suzuki IK, Alsina FC, Kc P, Sethi S, Vanderhaeghen P, Polleux F, Silver DL. Subcellular mRNA localization and local translation of Arhgap11a in radial glial progenitors regulates cortical development. Neuron 111(6):839-856.e5. doi: 10.1016/j.neuron.2023.02.023. 2023 Mar 15

Hashimoto A, Hirose T, Hashimoto K, Mizumoto S, Nitahara-Kasahara Y, Saka S, Yoshizawa T, Okada T, Yamada S, Kosho T, Watanabe T, Miyata S, Nomura Y. Collagen network formation in in vitro models of musculocontractural Ehlers-Danlos syndrome. Genes 14(2):308. doi: 10.3390/genes14020308. 2023 Jan 24

Meshitsuka S, Ninomiya R, Nagamura-Inoue T, Okada T, Futami M, Tojo A. CRISPR/Cas9 and AAV mediated insertion of b2 microglobulin-HLA-G fusion gene protects mesenchymal stromal cells from allogeneic rejection and potentiates the use for off-the-shelf cell therapy. Regen Ther. Oct 14;21:442-452. doi: 10.1016/j.reth.2022.09.009. eCollection 2022 Dec

Tone D, Ode KL, Zhang Q, Fujishima H, Yamada RG, Nagashima Y, Matsumoto K, Wen Z, Yoshida SY, Mitani TT, Arisato Y, Ohno RI, Ukai-Tadenuma M, Yoshida Garçon J, Kaneko M, Shi S, Ukai H, Miyamichi K, Okada T, Sumiyama K, Kiyonari H, Ueda HR. Distinct phosphorylation states of mammalian CaMKIIβ control the induction and maintenance of sleep. PLoS Biol. 20(10):e3001813. doi: 10.1371/journal.pbio.3001813. eCollection 2022 Oct 4

Kuroda S, Miyagawa Y, Sukegawa M, Tomono T, Yamamoto M, Adachi K, Verlengia G, Goins WF, Cohen JB, Glorioso JC, Okada T. Evaluation of parameters for efficient purification and long-term storage of herpes simplex virus-based vectors. Mol Ther Methods Clin Dev. Jun 13;26:132-143. doi: 10.1016/j.omtm.2022.06.007. eCollection 2022 Sep 8

Igarashi T, Ohsawa I, Kobayashi M, Miyazaki K, Igarashi T, Kameya S, Shiozawa AL, Ikeda Y, Miyagawa Y, Sakai M, Okada T, Iwao S, Takahashi H. Drinking hydrogen water improves photoreceptor structure and function in retinal degeneration 6 mice. Sci Rep. 12(1):13610. doi: 10.1038/s41598-022-17903-8. 2022 Aug 10

Tokuoka H, Imae R, Nakashima H, Manya H, Masuda C, Hoshino S, Kobayashi K, Lefeber DJ, Matsumoto R, Okada T, Endo T, Kanagawa M, Toda T. CDP-ribitol prodrug treatment ameliorates ISPD-deficient muscular dystrophy mouse model. Nat Commun. 13:1847. doi: 10.1038/s41467-022-29473-4. 2022 Apr 14

Nito C, Suda S, Nitahara-Kasahara Y, Okada T, Kimura K. Dental-Pulp Stem Cells as a Therapeutic Strategy for Ischemic Stroke. Biomedicines 10(4)737. doi.org/10.3390/biomedicines10040737. 2022 Mar 22

Nitahara-Kasahara Y,Mizumoto S, Inoue U.Y, Saka S, Posadas-Herrera G, Nakamura-Takahashi A, Takahashi Y, Hashimoto A, Konishi K, Miyata S, Masuda C, Matsumoto E, Maruoka Y, Yoshizawa T, Tanase T, Inoue T, Yamada S, Nomura Y, Takeda S, Watanabe A, Kosho T, Okada T. A new mouse model of Ehlers-Danlos syndrome generated using CRISPR/Cas9-mediated genomic editing. Dis Model Mech . 14(12):dmm048963. doi: 10.1242/dmm.048963. Epub 2021 Dec. 23.

Nitahara-Kasahara Y, Posadas-Herrera G, Nakamura-Takahashi Y, Mizumoto S, Inoue U.Y, Inoue T, Nomura Y, Takeda S, Yamada S, Kosho T, Okada T. Myopathy Associated with Dermatan Sulfate-Deficient Decorin in Musculocontractural Ehlers-Danlos Syndrome: a Mouse Model Investigation. Front Cell Dev Biol . 11;9:695021. doi: 10.3389/fcell.2021.695021. eCollection. 2021 Oct.

Adachi K, Tomono T, Okada H, Shiozawa Y, Yamamoto M, Miyagawa Y, Okada T. A PCR-amplified transgene fragment flanked by a single copy of a truncated ITR for rAAV production prevents unnecessary plasmid DNA packaging. Gene Ther . doi: 10.1038/s41434-021-00299-x. 2021 Oct. 11

Furstenau D, Peer CJ, Hughes TE, Uchida N, Tisdale J, Hall OM, Figg WD, Hsieh M. Alemtuzumab Clearance, Lymphocyte Count, and T-Cell Chimerism After Hematopoietic Stem Cell Transplant in Sickle Cell Disease. Pharmacotherapy Online ahead of print doi: 10.1002/phar.2641. 2021 Oct. 20

Kumkhaek C, Uchida N, Tisdale JF, Rodgers GP. Comparison of CD34+ cells isolated from frozen cord blood and fresh adult peripheral blood of sickle cell disease patients in gene correction of the sickle mutation at late-stage erythroid differentiation. Br J Haematol . 194(5):e80-e84. doi: 10.1111/bjh.17564. Sep 2021

Tsunekawa Y, Kunikane Terhune R, Matsuzaki F. Protocol for de novo gene targeting via in utero electroporation. Methods Mol Bio l. 2312:309-320. doi: 10.1007/978-1-0716-1441-9_19. 2021 Jul.

Leonard A, Sharma A, Uchida N, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease. Blood Adv . 5(9):2403-2411. doi: 10.1182/bloodadvances.2021004232. 2021 May 11

Uchida N, Li L, Nassehi T, Drysdale CM, Yapundich M, Gamer J, Haro-Mora JJ, Demirci S, Leonard A, Bonifacino AC, Krouse AE, Linde NS, Allen C, Peshwa MV, De Ravin SS, Donahue RE, Malech HL, Tisdale JF. Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models. Cell Rep Med . 2(4), doi: 10.1016/j.xcrm.2021.100247. 2021 Apr.

Uchida N, Ferrara F, Drysdale CM, Yapundich M, Gamer J, Nassehi T, DiNicola J, Shibata Y, Wielgosz M, Kim YS, Bauler M, Throm RE, Haro-Mora JJ, Demirci S, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ryu BY, Tisdale JF. Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and co-expressed truncated erythropoietin receptor. Sci Transl Med . 13(591):eabb0411 doi:10.1126/scitranslmed.abb0411. 2021 Apr.28

Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH. Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells. Nat Med . doi: 10.1038/s41591-021-01284-y. 2021 Mar. 18

Uchida N, Drysdale CM, Nassehi T, Gamer J, Yapundich M, DiNicola J, Shibata Y, Hinds M, Gudmundsdottir B, Haro-Mora JJ, Demirci S, Tisdale JF. Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease. Mol Ther Methods Clin Dev . 21:121-132, doi: 10.1016/j.omtm.2021.02.022. 2021 Mar. 3

Hirose T, Mizumoto S, Hashimoto A, Takahashi Y, Yoshizawa T, Nitahara-Kasahara Y, Takahashi N, Nakayama J, Takehana K, Okada T, Nomura Y, Yamada S, Kosho T, Watanabe T. Systemic investigation of the skin in Chst14−/− mice: a model for skin fragility in musculocontractural Ehlers–Danlos syndrome caused by CHST14 variants (mcEDS-CHST14). Glycobiology 31(2): 137-150, doi: 10.1093/glycob/cwaa058. 2021 Feb. 9

Nitahara Kasahara Y, Kuraoka M, Oda Y, Hayashita Kinoh H, Takeda S, Okada T. Enhanced cell survival and therapeutic benefits of IL-10-expressing multipotent mesenchymal stromal cells for muscular dystrophy. Stem Cell Res Ther . 12:105, doi: 10.1186/s13287-021-02168-1. 2021 Feb.

Nitahara Kasahara Y, Kuraoka M, Posadas Herrera G, Hayashita Kinoh H, Maruoka Y, Nakamura Takahashi A, Kimura K, Takeda S, Okada T. Dental pulp stem cells can improve muscle dysfunction in animal models of Duchenne muscular dystrophy. Stem Cell Res Ther . 12:78, doi: 10.1186/s13287-020-02099-3. 2021 Jan.

Igarashi T, Nakamoto K, Kobayashi M, Suzuki H, Arima T, Tobita Y, Takao K, Igarashi T, Okuda T, Okada T, Takahashi H. Brain-derived neurotrophic factor in human aqueous humor with glaucoma. J Nippon Med Sch . 88:128-132. 2021

Takahashi K, Igarashi T, Miyake K, Kobayashi M, Katakai Y, Hayashita - Kinoh H, Fujimoto C, Kameya S, Takahashi H, Okada T. Amount of green fluorescent protein in the anterior chamber after intravitreal injection of triple-mutated self-complementary AAV2 vectors is not affected by previous vitrectomy surgery. J Nippon Med Sch . 88(2):103-108 doi: 10.1272/jnms.JNMS.2021_88-203. 2021 Apr. 24

Igarashi T, Nakamoto K, Kobayashi M, Suzuki H, Igarashi T, Okada T, Takahashi H. Serum brain-derived neurotrophic factor in glaucoma patients in Japan: An observational study. J Nippon Med Sch . 87:339-345, doi: 10.1272/jnms.JNMS.2020_87-605. 2021 Jan. 8

Stringaris K, Hoyt RF, Davidson-Moncada JK, Pantin JM, Tisdale JF, Uchida N, Raines LN, Reger R, Sato N, Dunbar CE, Hunt TJ, Clevenger RR, Krouse A, Metzger ME, Bonifacino AC, Telford W, Choyke PL, Engels T, Donahue RE, Childs RW*. Intrabone transplantation of CD34+ cells with optimized delivery does not enhance engraftment in a rhesus macaque model. Blood Adv 4(24):6148-6156, 2020 Dec. 22

Demirci S, Haro-Mora JJ, Leonard A, Drysdale C, Malide D, Keyvanfar K, Essawi K, Vizcardo R, Tamaoki N, Restifo NP, Tisdale JF, Uchida N. Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation. Stem Cell Res Ther 11(1):493, 2020 Nov. 24

Hayashita-Kinoh H, Posadas-Herrera G, Nitahara-Kasahara Y, Kuraoka M,　Okada H, Chiyo T, Takeda S, Okada T. Improved transduction of canine　X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent mesenchymal stromal cell pretreatment. Mol Ther Methods Clin Dev 20:133-141, 2020

Demirci S, Zeng J, Wu Y, Uchida N, Shen AH, Pellin D, Gamer J, Yapundich M, Drysdale C, Bonanno J, Bonifacino AC, Krouse A, Linde NS, Engels T, Donahue RE, Haro-Mora JJ, Leonard A, Nassehi T, Luk K, Porter SN, Lazzarotto CR, Tsai SQ, Weiss M, Pruett-Miller SM, Wolfe SA, Bauer DE, Tisdale JF. BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity. J Clin Invest 130:6677-6687, 2020

Yamaguchi T, Uchida E, Okada T, Ozawa K, Onodera M, Kume A, Shimada T, Takahashi S, Tani K, Nasu Y, Mashimoto T, Mizuguchi H, Mitani K, Maki K. Aspects of gene therapy products using current genome-editing technology in Japan. Hum Gene Ther 31:1043-1053, 2020

Terada K, Miyake K, Yamaguchi H, Miyake N, Yamanaka K, Kojima S, Ito E, Inokuchi K, Okada T.　TERT and TERC mutations detected in cryptic dyskeratosis congenita suppress telomerase activity. Int J Lab Hematol 42:316-321, 2020

Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L, Schmidt M, Kanter J, Walters MC, Thompson A, Asmal M, Tisdale JF. Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease. Blood Adv 4:2058-2063, 2020

Kuroda S, Miyagawa Y, Sato Y, Yamamoto M, Adachi K, Kinoh H, Goins WF, Cohen JB, Glorioso JC, Taniai N, Yoshida H, OkadaT. Protocol optimization for the production of the non-cytotoxic JΔNI5 herpes simplex virus vector deficient in expression of immediately early genes. Mol Ther Methods Clin Dev 17:612-621, 2020

Nakamura-Takahashi A, Tanase T, Matsunaga S, Shintani S, Abe S, Nitahara-Kasahara Y, Watanabe A, Hirai Y, Okada T, Yamaguchi A, Kasahara M. High-Level Expression of Alkaline Phosphatase by Adeno-Associated Virus Vector Ameliorates Pathological Bone Structure in a Hypophosphatasia Mouse Model. Calcif Tissue Int 106:665-677, 2020

Hirose T, Mizumoto S, Hashimoto A, Takahashi Y, Yoshizawa T, Nitahara-Kasahara Y, Takahashi N, Nakayama J, Takehana K, Okada T, Nomura Y, Yamada S, Kosho T, Watanabe T. Systemic investigation of the skin in Chst14−/− mice: a model for skin fragility in musculocontractural Ehlers–Danlos syndrome caused by CHST14 variants (mcEDS-CHST14). Glycobiology doi: 10.1093/glycob/cwaa058. 2020

Shimada S, Yoshizawa T, Takahashi Y, Nitahara-Kasahara Y, Okada T, Nomura Y, Yamanaka H, Kosho T, Matsumoto K. Backcrossing to an Appropriate Genetic Background Improves the Birth Rate of Carbohydrate Sulfotransferase 14 Gene-deleted Mice. Exp Anim 69:407-413, 2020

Shiozawa Lee A, Igarashi T, Kobayashi M, Nakamoto K, Kameya S, Fujishita S, Takahashi H, Okada T. Tyrosine triple mutated AAV2-BDNF gene therapy in an inner retinal injury model induced by intravitreal injection of N–methyl-D-aspartate (NMDA). Mol Vision 26:409-422, 2020

Ishii A, Okada H, Hayashita-Kinoh H, Shin JH, Tamaoka A, Okada T, Takeda S. rAAV8 and rAAV9-mediated long-term muscle transduction with tacrolimus (FK506) in non-human primates. Mol Ther Methods Clin Dev 18:44-49, 2020

Uchida N, Leonard A, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Sharma A, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor. Haematologica 105:e497, 2020

Ito T, Sakai A, Maruyama M, Miyagawa Y, Okada T, Fukayama H & Suzuki H. Dorsal Root Ganglia Homeobox downregulation in primary sensory neurons contributes to neuropathic pain in rats. Mol Pain 16:1744806920904462, 2020

Tomono T, Hirai Y, Chono H, Mineno J, Ishii A, Onodera M, Tamaoka A & Okada T. Infectivity Assessment of Recombinant Adeno-Associated Virus and Wild-Type Adeno-Associated Virus Exposed to Various Diluents and Environmental Conditions. Hum Gene Ther Methods 30:137-143, 2019

Shimazaki K, Kobari T, Oguro K, Yokota H, Kasahara Y, Murashima Y, Watanabe E, Kawai K & Okada T. Hippocampal GAD67 Transduction Using rAAV8 Regulates Epileptogenesis in EL Mice. Mol Ther Methods Clin Dev 13:180-186, 2019

Li H, Okada H, Suzuki S, Sakai K, Izumi H, Matsushima Y, Ichinohe N, Goto YI, Okada T & Inoue K. Gene suppressing therapy for Pelizaeus-Merzbacher disease using artificial microRNA. JCI Insight 4:16, 2019

Zhang B, Nakata M, Lu M, Nakae J, Okada T, Ogawa W & Yada T. Protective role of AgRP neuron's PDK1 against salt-induced hypertension. Biochem Biophys Res Commun 500:910-916, 2018

Yoshizawa T, Mizumoto S, Takahashi Y, Shimada S, Sugahara K, Nakayama J, Takeda S, Nomura Y, Nitahara-Kasahara Y, Okada T, Matsumoto K, Yamada S & Kosho T. Vascular abnormalities in the placenta of Chst14-/- fetuses: implications in the pathophysiology of perinatal lethality of the murine model and vascular lesions in human CHST14/D4ST1 deficiency. Glycobiology 28:80-89, 2018

Tomono T, Hirai Y, Okada H, Miyagawa Y, Adachi K, Sakamoto S, Kawano Y, Chono H, Mineno J, Ishii A, Shimada T, Onodera M, Tamaoka A & Okada T. Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9. Mol Ther Methods Clin Dev 11:180-190, 2018

Sowa K, Nito C, Nakajima M, Suda S, Nishiyama Y, Sakamoto Y, Nitahara-Kasahara Y, Nakamura-Takahashi A, Ueda M, Kimura K & Okada T. Impact of Dental Pulp Stem Cells Overexpressing Hepatocyte Growth Factor after Cerebral Ischemia/Reperfusion in Rats. Mol Ther Methods Clin Dev 10:281-290, 2018

Nito C, Sowa K, Nakajima M, Sakamoto Y, Suda S, Nishiyama Y, Nakamura-Takahashi A, Nitahara-Kasahara Y, Ueda M, Okada T & Kimura K. Transplantation of human dental pulp stem cells ameliorates brain damage following acute cerebral ischemia. Biomed Pharmacother 108:1005-1014, 2018

Nakajima M, Suda S, Sowa K, Sakamoto Y, Nito C, Nishiyama Y, Aoki J, Ueda M, Yokobori S, Yamada M, Yokota H, Okada T & Kimura K. AMPA Receptor Antagonist Perampanel Ameliorates Post-Stroke Functional and Cognitive Impairments. Neuroscience 386:256-264, 2018

Iwasaki Y, Sendo M, Dezaki K, Hira T, Sato T, Nakata M, Goswami C, Aoki R, Arai T, Kumari P, Hayakawa M, Masuda C, Okada T, Hara H, Drucker D J, Yamada Y, Tokuda M & Yada T. GLP-1 release and vagal afferent activation mediate the beneficial metabolic and chronotherapeutic effects of D-allulose. Nat Commun 9:113, 2018

Ikeue R, Nakamura-Takahashi A, Nitahara-Kasahara Y, Watanabe A, Muramatsu T, Sato T & Okada T. Bone-Targeted Alkaline Phosphatase Treatment of Mandibular Bone and Teeth in Lethal Hypophosphatasia via an scAAV8 Vector. Mol Ther Methods Clin Dev 10:361-370, 2018

Fukuoka M, Takahashi M, Fujita H, Chiyo T, Popiel H A, Watanabe S, Furuya H, Murata M, Wada K, Okada T, Nagai Y & Hohjoh H. Supplemental Treatment for Huntington's Disease with miR-132 that Is Deficient in Huntington's Disease Brain. Mol Ther Nucleic Acids 11:79-90, 2018

Takahashi K, Igarashi T, Miyake K, Kobayashi M, Yaguchi C, Iijima O, Yamazaki Y, Katakai Y, Miyake N, Kameya S, Shimada T, Takahashi H & Okada T. Improved Intravitreal AAV-Mediated Inner Retinal Gene Transduction after Surgical Internal Limiting Membrane Peeling in Cynomolgus Monkeys. Mol Ther 25:296-302, 2017

Sakai A, Saitow F, Maruyama M, Miyake N, Miyake K, Shimada T, Okada T & Suzuki H. MicroRNA cluster miR-17-92 regulates multiple functionally related voltage-gated potassium channels in chronic neuropathic pain. Nat Commun 8:16079, 2017

Okawa R, Iijima O, Kishino M, Okawa H, Toyosawa S, Sugano-Tajima H, Shimada T, Okada T, Ozono K, Ooshima T & Nakano K. Gene therapy improves dental manifestations in hypophosphatasia model mice. J Periodontal Res 52:471-478, 2017

Nakata M, Yamamoto S, Okada T & Yada T. AAV-mediated IL-10 gene transfer counteracts inflammation in the hypothalamic arcuate nucleus and obesity induced by high-fat diet. Neuropeptides 62:87-92, 2017

Nakajima M, Nito C, Sowa K, Suda S, Nishiyama Y, Nakamura-Takahashi A, Nitahara-Kasahara Y, Imagawa K, Hirato T, Ueda M, Kimura K & Okada T. Mesenchymal Stem Cells Overexpressing Interleukin-10 Promote Neuroprotection in Experimental Acute Ischemic Stroke. Mol Ther Methods Clin Dev6:102-111, 2017

Miyoshi S, Tezuka T, Arimura S, Tomono T, Okada T & Yamanashi Y. DOK7 gene therapy enhances motor activity and life span in ALS model mice. EMBO Mol Med 9:880-889, 2017

Ito N, Sakai A, Miyake N, Maruyama M, Iwasaki H, Miyake K, Okada T, Sakamoto A & Suzuki H. miR-15b mediates oxaliplatin-induced chronic neuropathic pain through BACE1 down-regulation. Br J Pharmacol 174:386-395, 2017

Tomono T, Hirai Y, Okada H, Adachi K, Ishii A, Shimada T, Onodera M, Tamaoka A & Okada T. Ultracentrifugation-free chromatography-mediated large-scale purification of recombinant adeno-associated virus serotype 1 (rAAV1). Mol Ther Methods Clin Dev3:15058, 2016

Nakata M, Zhang B, Yang Y, Okada T, Shintani N, Hashimoto H & Yada T. High-Fat Diet Augments VPAC1 Receptor-Mediated PACAP Action on the Liver, Inducing LAR Expression and Insulin Resistance. J Diabetes Res 2016:9321395, 2016

Nakata M, Yamamoto S, Okada T, Gantulga D, Okano H, Ozawa K & Yada T. IL-10 gene transfer upregulates arcuate POMC and ameliorates hyperphagia, obesity and diabetes by substituting for leptin. Int J Obes (Lond) 40:425-433, 2016

Nakata M, Gantulga D, Santoso P, Zhang B, Masuda C, Mori M, Okada T & Yada T. Paraventricular NUCB2/Nesfatin-1 Supports Oxytocin and Vasopressin Neurons to Control Feeding Behavior and Fluid Balance in Male Mice. Endocrinology 157:2322-2332, 2016

Nakamura-Takahashi A, Miyake K, Watanabe A, Hirai Y, Iijima O, Miyake N, Adachi K, Nitahara-Kasahara Y, Kinoshita H, Noguchi T, Abe S, Narisawa S, Millan J L, Shimada T & Okada T. Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector. Mol Ther Methods Clin Dev3:15059, 2016

Kuraoka M, Kimura E, Nagata T, Okada T, Aoki Y, Tachimori H, Yonemoto N, Imamura M & Takeda S. Serum Osteopontin as a Novel Biomarker for Muscle Regeneration in Duchenne Muscular Dystrophy. Am J Pathol 186:1302-1312, 2016

Igarashi T, Miyake K, Kobayashi M, Kameya S, Fujimoto C, Nakamoto K, Takahashi H, Igarashi T, Miyake N, Iijima O, Hirai Y, Shimada T, Okada T & Takahashi H. Tyrosine triple mutated AAV2-BDNF gene therapy in a rat model of transient IOP elevation. Mol Vis 22:816-826, 2016

Tanokashira D, Motoki K, Minegishi S, Hosaka A, Mamada N, Tamaoka A, Okada T, Lakshmana M K & Araki W. LRP1 Downregulates the Alzheimer's beta-Secretase BACE1 by Modulating Its Intraneuronal Trafficking. eNeuro 2:0006-0015, 2015

Ohtsuka Y, Kanagawa M, Yu C C, Ito C, Chiyo T, Kobayashi K, Okada T, Takeda S & Toda T. Fukutin is prerequisite to ameliorate muscular dystrophic phenotype by myofiber-selective LARGE expression. Sci Rep 5:8316, 2015

Iijima O, Miyake K, Watanabe A, Miyake N, Igarashi T, Kanokoda C, Nakamura-Takahashi A, Kinoshita H, Noguchi T, Abe S, Narisawa S, Millan J L, Okada T & Shimada T. Prevention of Lethal Murine Hypophosphatasia by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells. Hum Gene Ther 26:801-812, 2015

Hironaka K, Yamazaki Y, Hirai Y, Yamamoto M, Miyake N, Miyake K, Okada T, Morita A & Shimada T. Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector. Sci Rep 5:13104, 2015

Hayashita-Kinoh H, Yugeta N, Okada H, Nitahara-Kasahara Y, Chiyo T, Okada T & Takeda S. Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance. Mol Ther 23:627-637, 2015

Dohi T, Miyake K, Aoki M, Ogawa R, Akaishi S, Shimada T, Okada T & Hyakusoku H. Tissue Inhibitor of Metalloproteinase-2 Suppresses Collagen Synthesis in Cultured Keloid Fibroblasts. Plast Reconstr Surg Glob Open 3:e520, 2015

Darambazar G, Nakata M, Okada T, Wang L, Li E, Shinozaki A, Motoshima M, Mori M & Yada T. Paraventricular NUCB2/nesfatin-1 is directly targeted by leptin and mediates its anorexigenic effect. Biochem Biophys Res Commun 456:913-918, 2015

Nitahara-Kasahara Y, Hayashita-Kinoh H, Chiyo T, Nishiyama A, Okada H, Takeda S & Okada T. Dystrophic mdx mice develop severe cardiac and respiratory dysfunction following genetic ablation of the anti-inflammatory cytokine IL-10. Hum Mol Genet 23:3990-4000, 2014

Masamizu Y, Tanaka Y R, Tanaka Y H, Hira R, Ohkubo F, Kitamura K, Isomura Y, Okada T & Matsuzaki M. Two distinct layer-specific dynamics of cortical ensembles during learning of a motor task. Nat Neurosci 17:987-994, 2014

Hira R, Ohkubo F, Masamizu Y, Ohkura M, Nakai J, Okada T & Matsuzaki M. Reward-timing-dependent bidirectional modulation of cortical microcircuits during optical single-neuron operant conditioning. Nat Commun 5:5551, 2014

Arimura S, Okada T, Tezuka T, Chiyo T, Kasahara Y, Yoshimura T, Motomura M, Yoshida N, Beeson D, Takeda S & Yamanashi Y. DOK7 gene therapy benefits mouse models of diseases characterized by defects in the neuromuscular junction. Science 345:1505-1508, 2014