Publication list

  • Hotta A, Schrepfer S, Nagy A.
    Genetically engineered hypoimmunogenic cell therapy.
    Nat Rev Bioeng. 2024. DOI: 10.1038/s44222-024-00219-9
    [PubMed link] [Journal link] [Link to PDF]
  • Munezane H, Imamura K, Fujimoto N, Hotta A, Yukitake H, Inoue H.
    Elimination of the extra chromosome of Dup15q syndrome iPSCs for cellular and molecular investigation.
    Eur J Cell Biol. 2024 Jul 18;103(3):151446.
    [PubMed link] [Journal link]
  • Lee J, Naoe Y, Bang U, Nakagama Y, Saito A, Kido Y, Hotta A.
    Neutralization sensitivity of SARS-CoV-2 Omicron variants FL.1 and GE.1 by therapeutic antibodies and XBB sera.
    Virology. 2024 Jul;595:110067. Epub 2024 Apr 10.
    [PubMed link] [Journal link]
  • Gao P, Inada Y, Hotta A, Sakurai H, Ikeya M.
    iMSC-mediated delivery of ACVR2B-Fc fusion protein reduces heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva.
    Stem Cell Res Ther. 2024 Mar 18;15(1):83.
    [PubMed link] [Journal link]
  • Nakanishi-Koakutsu M, Miki K, Naka Y, Sasaki M, Wakimizu T, Napier SC, Okubo C, Narita M, Nishikawa M, Hata R, Chonabayashi K, Hotta A, Imahashi K, Nishimoto T, Yoshida Y.
    CD151 expression marks atrial- and ventricular- differentiation from human induced pluripotent stem cells.
    Commun Biol. 2024 Feb 28;7(1):231.
    [PubMed link] [Journal link]
  • Achón Buil B, Rentsch NH, Weber RZ, Rickenbach C, Halliday SJ, Hotta A, Tackenberg C, Rust R.
    Beneath the radar: immune-evasive cell sources for stroke therapy.
    Trends Mol Med. 2024 Mar;30(3):223-238. Epub 2024 Jan 25.
    [PubMed link] [Journal link]
  • Hotta A, Lee J.
    Hiding from allogeneic NK cells and macrophages by a synthetic receptor.
    Cell Stem Cell. 2023 Nov 2;30(11):1393-1394. doi: 10.1016/j.stem.2023.10.005.
    [PubMed link] [Journal link]
  • Hirayama R, Toyohara K, Watanabe K, Otsuki T, Araoka T, Mae SI, Horinouchi T, Yamamura T, Okita K, Hotta A, Iijima K, Nozu K, Osafune K.
    iPSC-derived type IV collagen α5-expressing kidney organoids model Alport syndrome.
    Commun Biol. 2023 Sep 28;6(1):854. doi: 10.1038/s42003-023-05203-4.
    [PubMed link] [Journal link]
  • Kita Y, Okuzaki Y, Naoe Y, Lee J, Bang U, Okawa N,Ichiki A, Jonouchi T, Sakurai H, Kojima Y,Hotta A
    Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs.
    Stem Cell Reports. 2023 Aug 24;18:1-13. doi: 10.1016/j.stemcr.2023.07.007
    [PubMed link] [Journal link]
  • Kawada R, Jonouchi T, Kagita A, Sato M, Hotta A, Sakurai H.
    Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs.
    Sci Rep. 2023 Jan 11;13(1):94. doi: 10.1038/s41598-022-26614-z.
    [PubMed link] [Journal link]
  • Ueda T, Shiina S, Iriguchi S, Terakura S, Kawai Y, Kabai R, Sakamoto S, Watanabe A, Ohara K, Wang B, Xu H, Minagawa A, Hotta A, Woltjen K, Uemura Y, Kodama Y, Seno H, Nakatsura T, Tamada K, Kaneko S.
    Optimization of the proliferation and persistency of CAR T cells derived from human induced pluripotent stem cells.
    Nat Biomed Eng.. 2023 Jan;7(1):24-37. doi: 10.1038/s41551-022-00969-0
    [PubMed link] [Journal link]
  • Watanabe K, Gee P, Hotta A.
    Preparation of NanoMEDIC extracellular vesicles to deliver CRISPR-Cas9 ribonucleoproteins for genomic exon skipping.
    Methods Mol Biol.. 2023;2587:427-453. doi: 10.1007/978-1-0716-2772-3_22.
    [PubMed link] [Journal link]
  • Park G, Shin M, Lee W, Hotta A, Kobayashi T, Kosodo Y.
    Direct visualization of the transition status during neural differentiation by dual-fluorescent reporter human pluripotent stem cells.
    Stem Cell Reports. 2022 Sep 13;17(9):1903-1913. doi: 10.1016/j.stemcr.2022.07.001.
    [PubMed link] [Journal link]
  • Kitano Y, Nishimura S, Kato TM, Ueda A, Takigawa K, Umekage M, Nomura M, Kawakami A, Ogawa H, Xu H, Hotta A, Takasu N, Tsukahara M.
    Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application.
    Mol Ther Methods Clin Dev. 2022 May 29;26:15-25. doi: 10.1016/j.omtm.2022.05.010.
    [PubMed link] [Journal link]
  • Taha EA, Lee J, Hotta A.
    Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges.
    J Control Release. 2022 Jan 10;342:345-361.doi: 10.1016/j.jconrel.2022.01.013
    [PubMed link] [Journal link]
  • Kenjo E, Hozumi H, Makita Y, Iwabuchi KA, Fujimoto N, Matsumoto S, Kimura M, Amano Y, Ifuku M, Naoe Y, Inukai N, Hotta A.
    Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice.
    Nat Commun. 2021 Dec 8; 12, 7101. doi: 10.1038/s41467-021-26714-w
    [PubMed link] [Journal link]
  • Xu H, Kita Y, Bang U, Gee P, Hotta A.
    Optimized electroporation of CRISPR-Cas9/gRNA ribonucleoprotein complex for selection-free homologous recombination in human pluripotent stem cells.
    STAR Protocols. 2021 Nov 19; doi: 10.1016/j.xpro.2021.100965
    [PubMed link] [Journal link]
  • Hashimoto R, Sakamoto A, Deguchi S, Yi R, Sano E, Hotta A, Takahashi K, Yamanaka S, Takayama K.
    Dual inhibition of TMPRSS2 and Cathepsin B prevents SARS-CoV-2 infection in iPS cells.
    Mol Ther Nucleic Acids. 2021 Dec 3;26:1107-1114. doi: 10.1016/j.omtn.2021.10.016. Epub 2021 Oct 20.
    [PubMed link] [Journal link]
  • Noda M, Tatsumi K, Matsui H, Matsunari Y, Sato T, Fukuoka Y,Hotta A, Okano T, Kichikawa K, Sugimoto M, Shima M, Nishio K.
    Development of alternative gene transfer techniques for ex vivo and in vivo gene therapy in a canine model.
    Regen Ther. 2021 Sep 10;18:347-354. doi: 10.1016/j.reth.2021.08.009. eCollection 2021 Dec.
    [PubMed link] [Journal link]
  • Okubo C, Narita M, Inagaki A, Nishikawa M, Hotta A, Yamanaka S, Yoshida Y.
    Expression dynamics of HAND1/2 in in vitro human cardiomyocyte differentiation.
    Stem Cell Reports, 2021 Aug 10;16(8):1906-1922. doi: 10.1016/j.stemcr.2021.06.014. Epub 2021 Jul 22.
    [PubMed link] [Journal link]
  • Uchimura T, Asano T, Nakata T, Hotta A, Sakurai H.
    A muscle fatigue-like contractile decline was recapitulated using skeletal myotubes from Duchenne muscular dystrophy patient-derived iPSCs.
    Cell Rep Med., 2021 Jun 4;2(6):100298. doi: 10.1016/j.xcrm.2021.100298. eCollection 2021 Jun 15.
    [PubMed link] [Journal link]
  • Wang B, Iriguchi S, Waseda M, Ueda N, Ueda T, Xu H, Minagawa A, Ishikawa A, Yano H, Ishi T, Ito R, Goto M, Takahashi R, Uemura Y, Hotta A, Kaneko S.
    Generation of hypoimmunogenic T cells from genetically engineered allogeneic human induced pluripotent stem cells.
    Nat Biomed Eng., 2021 May;5(5):429-440. doi: 10.1038/s41551-021-00730-z. Epub 2021 May 17.
    [PubMed link] [Journal link]
  • Nalbandian M, Zhao M, Sasaki-Honda M, Jonouchi T, Lucena-Cacace A, Mizusawa T, Yasuda M, Yoshida Y, Hotta A, Sakurai H.
    Characterization of hiPSC-Derived Muscle Progenitors Reveals Distinctive Markers for Myogenic Cell Purification Toward Cell Therapy.
    Stem Cell Reports, 2021 Apr 13;16(4):883-898. doi: 10.1016/j.stemcr.2021.03.004. Epub 2021 Apr 1.
    [PubMed link] [Journal link]
  • Kagita A, Lung MSY, Xu H, Kita Y, Sasakawa N, Iguchi T, Ono M, Wang XH, Gee P, and Hotta A.
    Efficient ssODN-Mediated Targeting by Avoiding Cellular Inhibitory RNAs through Precomplexed CRISPR-Cas9/sgRNA Ribonucleoprotein.
    Stem Cell Reports, 2021 Mar 11; S2213-6711(21)00093-X. doi: 10.1016/j.stemcr.2021.02.013.
    [PubMed link] [Journal link]
  • Yamada H, Shirata N, Makino S, Miyake T, Oliva Trejo JA, Yamamoto-Nonaka K, Kikyo M, Empitu MA, Kadariswantiningsih IN, Kimura M, Ichimura K, Yokoi H, Mukoyama M, Hotta A, Nishimori K, Yanagita M, and Asanuma K .
    MAGI-2 orchestrates the localization of backbone proteins in the slit diaphragm of podocytes.
    Kidney Int., 2020 Oct 31:S0085-2538(20)31235-7. doi: 10.1016/j.kint.2020.09.027.
    [PubMed link] [Journal link]
  • Shimizu T, Mae SI, Araoka T, Okita K, Hotta A, Yamagata K, Osafune K.
    A novel ADPKD model using kidney organoids derived from disease-specific human iPSCs.
    Biochem Biophys Res Commun, 2020 Sep 3;529(4):1186-1194. doi: 10.1016/j.bbrc.2020.06.141. Epub 2020 Aug 4.
    [PubMed link] [Journal link]
  • Kuraoka S, Tanigawa S, Taguchi A, Hotta A, Nakazato H, Osafune K, Kobayashi A, Nishinakamura R.
    PKD1-Dependent Renal Cystogenesis in Human Induced Pluripotent Stem Cell-Derived Ureteric Bud/Collecting Duct Organoids.
    J Am Soc Nephrol., 2020 Oct;31(10):2355-2371. doi: 10.1681/ASN.2020030378. Epub 2020 Aug 3.
    [PubMed link] [Journal link]
  • Zhao M, Tazumi A, Takayama S, Takenaka-Ninagawa N, Nalbandian M, Nagai M, Nakamura Y, Nakasa M, Watanabe A, Ikeya M, Hotta A, Ito Y, Sato T, Sakurai H.
    Induced Fetal Human Muscle Stem Cells with High Therapeutic Potential in a Mouse Muscular Dystrophy Model.
    Stem Cell Reports, 2020 Jul 14;15(1):80-94. doi: 10.1016/j.stemcr.2020.06.004. Epub 2020 Jul 2.
    [PubMed link] [Journal link]
  • Sasaki-Honda M, Kagita A, Jonouchi T, Araki T, Hotta A, Sakurai H.
    Generation of a transgene-free iPSC line and genetically modified line from a facioscapulohumeral muscular dystrophy type 2 (FSHD2) patient with SMCHD1 p.Lys607Ter mutation.
    Stem Cell Res, 2020 Jun 24;47:101884. doi: 10.1016/j.scr.2020.101884
    [PubMed link] [Journal link]
  • Gee P, Lung MSY, Okuzaki Y, Sasakawa N, Iguchi T, Makita Y, Hozumi H, Miura Y, Yang LF, Iwasaki M, Wang XH, Waller MA, Shirai N, Abe YO, Fujita Y, Watanabe K, Kagita A, Iwabuchi KA, Yasuda M, Xu H, Noda T, Komano J, Sakurai H, Inukai N, and Hotta A.
    Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping.
    Nature Communications, 2020 Mar 13; 11, 1334. doi: 10.1038/s41467-020-14957-y.
    [PubMed link] [Journal link]
  • Suzuki D, Flahou C, Yoshikawa N, Stirblyte I, Hayashi Y, Sawaguchi A, Akasaka M, Nakamura S, Higashi N, Xu H, Matsumoto T, Fujio K, Manz MG, Hotta A, Takizawa H, Eto K, Sugimoto N.
    iPSC-derived platelets depleted of HLA class I are inert to anti-HLA class I and natural killer cell immunity.
    Stem Cell Reports, 2020 Jan 14;14(1):49-59. doi: 10.1016/j.stemcr.2019.11.011. Epub 2019 Dec 26.
    [PubMed link] [Journal link]
  • Morisaka H*, Yoshimi K*, Okuzaki Y*, Gee P, Kunihiro Y, Sonpho E, Xu H, Sasakawa N, Naito Y, Nakada S, Yamamoto T, Sano S, Hotta A, Takeda J, Mashimo T§. (*: Co-1st author, §: Co-correspondance)
    CRISPR-Cas3 induces broad and unidirectional genome editing in human cells.
    Nature Communications, 2019 Dec 6; 10, 5302. doi: doi:10.1038/s41467-019-13226-x
    [PubMed link] [Journal link]
    • Commentary on our institutional website.
    • Cas3 plasmid vectors are available from Addgene.
  • Huaigeng Xu, Bo Wang, Miyuki Ono, Akihiro Kagita, Kaho Fujii, Noriko Sasakawa, Tatsuki Ueda, Peter Gee, Misato Nishikawa, Masaki Nomura, Fumiyo Kitaoka, Tomoko Takahashi, Keisuke Okita, Yoshinori Yoshida, Shin Kaneko, and Akitsu Hotta.
    Targeted Disruption of HLA Genes via CRISPR-Cas9 Generates iPSCs with Enhanced Immune Compatibility.
    Cell Stem Cell, 2019 Mar 7;. doi: 10.1016/j.stem.2019.02.005.
    [PubMed link] [Journal link]
  • Korogi Y, Gotoh S, Ikeo S, Yamamoto Y, Sone N, Tamai K, Konishi S, Nagasaki T, Matsumoto H, Ito I, Chen-Yoshikawa TF, Date H, Hagiwara M, Asaka I, Hotta A, Mishima M, Hirai T.
    In vitro disease modeling of Hermansky-Pudlak Syndrome type 2 using human induced pluripotent stem cell-derived alveolar organoids.
    Stem Cell Reports, 2019 Mar 5;12(3):431-440. doi: 10.1016/j.stemcr.2019.01.014. Epub 2019 Feb 14.
    [PubMed link] [Journal link]
  • Minagawa A, Yoshikawa T, Yasukawa M, Hotta A, Kunitomo M, Iriguchi S, Takiguchi M, Kassai Y, Imai E, Yasui Y, Kawai Y, Zhang R, Uemura Y, Miyoshi H, Nakanishi M, Watanabe A, Hayashi A, Kawana K, Fujii T, Nakatsura T, Kaneko S.
    Enhancing T cell receptor stability in rejuvenated iPSC-derived T cells improves their use in cancer immunotherapy.
    Cell Stem Cell, 2018 Dec 6;23(6):850-858.e4. doi: 10.1016/j.stem.2018.10.005. Epub 2018 Nov 15.
    [PubMed link] [Journal link]
  • Seo H, Chen SJ, Hashimoto K, Endo H, Nishi Y, Ohta A, Yamamoto T, Hotta A, Sawaguchi A, Hayashi H, Koseki N, Murphy GJ, Fukuda K, Sugimoto N, Eto K.
    A β1-tubulin-based megakaryocyte maturation reporter system identifies novel drugs that promote platelet production.
    Blood Adv., 2018 Sep 11;2(17):2262-2272. doi: 10.1182/bloodadvances.2018019547.
    [PubMed link] [Journal link]
  • Sasaki-Honda M, Jonouchi T, Arai M, Hotta A, Mitsuhashi S, Nishino I, Matsuda R, Sakurai H.
    A Patient-derived iPSC Model Revealed Oxidative Stress Increases Facioscapulohumeral Muscular Dystrophy-causative DUX4.
    Hum Mol Genet. , 2018 Aug 9. doi: 10.1093/hmg/ddy293.
    [PubMed link] [Journal link]
  • Honda-Ozaki F, Terashima M, Niwa A, Saiki N, Kawasaki Y, Ito H, Hotta A, Nagahashi A, Igura K, Asaka I, Li HL, Yanagimachi M, Furukawa F, Kanazawa N, Nakahata T, Saito MK.
    Pluripotent Stem Cell Model of Nakajo-Nishimura Syndrome Untangles Proinflammatory Pathways Mediated by Oxidative Stress.
    Stem Cell Reports, 2018 May 1. pii: S2213-6711(18)30172-3. doi: 10.1016/j.stemcr.2018.04.004.
    [PubMed link] [Journal link]
  • Ikeda T, Hikichi T, Miura H, Shibata H, Mitsunaga K, Yamada Y, Woltjen K, Miyamoto K, Hiratani I, Yamada Y, Hotta A, Yamamoto T, Okita K, Masui S.
    Srf destabilizes cellular identity by suppressing cell-type-specific gene expression programs.
    Nat Commun., 2018 Apr 11;9(1):1387. doi: 10.1038/s41467-018-03748-1.
    [PubMed link] [Journal link]
  • Oshima K, Saiki N, Tanaka M, Imamura H, Niwa A, Tanimura A, Nagahashi A, Hirayama A, Okita K, Hotta A, Kitayama S, Osawa M, Kaneko S, Watanabe A, Asaka I, Fujibuchi W, Imai K, Yabe H, Kamachi Y, Hara J, Kojima S, Tomita M, Soga T, Noma T, Nonoyama S, Nakahata T, Saito MK.
    Human AK2 links intracellular bioenergetic redistribution to the fate of hematopoietic progenitors.
    Biochem Biophys Res Commun., 2018 Mar 4;497(2):719-725. doi: 10.1016/j.bbrc.2018.02.139. Epub 2018 Feb 17.
    [PubMed link] [Journal link]
  • Ishida K, Xu H, Sasakawa N, Lung MSY, Kudryashev JA, Gee P, Hotta A.
    Site-specific randomization of the endogenous genome by a regulatable CRISPR-Cas9 piggyBac system in human cells.
    Scientific Reports., 2018 Jan 10;8:310. doi:10.1038/s41598-017-18568-4.
    [PubMed link] [Journal link]
    • CRONUS-piggyBac vectors are available from Addgene.
  • Kojima Y, Sasaki K, Yokobayashi S, Sakai Y, Nakamura T, Yabuta Y, Nakaki F, Nagaoka S, Woltjen K, Hotta A, Yamamoto T, Saitou M.
    Evolutionarily distinctive transcriptional and signaling programs drive human germ cell lineage specification from pluripotent stem cells.
    Cell Stem Cell., 2017 Oct 5;21(4):517-532.e5. doi: 10.1016/j.stem.2017.09.005.
    [PubMed link] [Journal link]
  • Chen Z, Chang WY, Etheridge A, Strickfaden H, Jin Z, Palidwor G, Cho JH, Wang K, Kwon SY, Doré C, Raymond A, Hotta A, Ellis J, Kandel RA, Dilworth FJ, Perkins TJ, Hendzel MJ, Galas DJ, Stanford WL.
    Reprogramming progeria fibroblasts re-establishes a normal epigenetic landscape.
    Aging Cell., 2017 Aug;16(4):870-887. doi: 10.1111/acel.12621.
    [PubMed link] [Journal link]
  • Takada S, Kambe N, Kawasaki Y, Niwa A, Honda-Ozaki F, Kobayashi K, Osawa M, Nagahashi A, Semi K, Hotta A, Asaka I, Yamada Y, Nishikomori R, Heike T, Matsue H, Nakahata T, Saito MK.
    Pluripotent stem cell models of Blau syndrome reveal an IFN-γ-dependent inflammatory response in macrophages.
    J Allergy Clin Immunol., 2017 Jun 3; pii: S0091-6749(17)30685-1. doi: 10.1016/j.jaci.2017.04.013.
    [PubMed link] [Journal link]
  • Imamura K, Izumi Y, Watanabe A, Tsukita K, Woltjen K, Yamamoto T, Hotta A, Kondo T, Kitaoka S, Ohta A, Tanaka A, Watanabe D, Morita M, Takuma H, Tamaoka A, Kunath T, Wray S, Furuya H, Era T, Makioka K, Okamoto K, Fujisawa T, Nishitoh H, Homma K, Ichijo H, Julien JP, Obata N, Hosokawa M, Akiyama H, Kaneko S, Ayaki T, Ito H, Kaji R, Takahashi R, Yamanaka S, Inoue H.
    The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis.
    Sci Transl Med. 2017 May 24;9(391). pii: eaaf3962. doi: 10.1126/scitranslmed.aaf3962.
    [PubMed link] [Journal link]
  • Hirosawa M, Fujita Y, Parr CJC, Hayashi K, Kashida S, Hotta A, Woltjen K, Saito H.
    Cell-type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch.
    Nucleic Acids Res 2017 May 19. doi: 10.1093/nar/gkx309.
    [PubMed link] [Journal link]
  • Gee P, Xu H, Hotta A.
    Cellular reprogramming, genome Editing, and alternative CRISPR Cas9 technologies for precise gene therapy of Duchenne muscular dystrophy.
    Stem Cells International, 2017 May 18; Article ID 8765154.
    [PubMed link] [Journal link]
  • Imamura K, Sahara N, Kanaan NM, Tsukita K, Kondo T, Kutoku Y, Ohsawa Y, Sunada Y, Kawakami K, Hotta A, Yawata S, Watanabe D, Hasegawa M, Trojanowski JQ, Lee VM, Suhara T, Higuchi M, Inoue H.
    Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons.
    Sci. Rep., 2016 Oct 10;6:34904.
    [PubMed link] [Journal link]
  • Choi IY, Lim H, Estrellas K, Mula J, Cohen TV, Zhang Y, Donnelly CJ, Richard JP, Kim YJ, Kim H, Kazuki Y, Oshimura M, Li HL, Hotta A, Rothstein J, Maragakis N, Wagner KR, Lee G.
    Concordant but varied phenotypes among Duchenne muscular dystrophy patient-specific myoblasts derived using a human iPSC-based model.
    Cell Rep., 2016 Jun 7;15(10):2301-12.
    [PubMed link] [Journal link]
  • Komura S, Semi K, Itakura F, Shibata H, Ohno T, Hotta A, Woltjen K, Yamamoto T, Akiyama H, Yamada Y.
    An EWS-FLI1-induced osteosarcoma model unveiled a crucial role of impaired osteogenic differentiation on osteosarcoma development.
    Stem Cell Reports., 2016 Apr 12;6(4):592-606.
    [PubMed link] [Journal link]
  • Morita Y, Andersen P, Hotta A, Tsukahara Y, Sasagawa N, Hayashida N, Koga C, Nishikawa M, Saga Y, Evans SM, Koshiba-Takeuchi K, Nishinakamura R, Yoshida Y, Kwon C, Takeuchi JK.
    Sall1 transiently marks undifferentiated heart precursors and regulates their fate.
    J Mol Cell Cardiol., 2016 Feb 11, 92:158-62.
    [PubMed link] [Journal link]
  • Li HL, Gee P, Ishida K, and Hotta A.
    Efficient genomic correction methods in human iPS cells using CRISPR-Cas9 system.
    Methods, 2015 Oct 23, pii: S1046-2023(15)30133-X.
    [PubMed link] [Journal link]
  • Ishida K*, Gee P*, and Hotta A. (*: equal contribution)
    Minimizing off-target mutagenesis risks caused by programmable nucleases.
    Int. J. Mol. Sci., 2015 Oct 16, 16 (10), 24751-24771
    [PubMed link] [Journal link]
  • Hotta A, and Yamanaka S.
    From genomics to gene therapy: induced pluripotent stem cells meet genome editing.
    Annu. Rev. Genet., 2015 Sep 25, 49:47-70.
    [PubMed link] [Journal link]
  • Hotta A.
    Genome editing gene therapy for Duchenne muscular dystrophy .
    J. Neuromuscul. Dis., 2015 Sep 22, 2 (4) 343-355
    [Journal link]
  • Ishikawa M, Ohnishi H, Skerleva D, Sakamoto T, Yamamoto N, Hotta A, Ito J, Nakagawa T.
    Transplantation of neurons derived from human iPS cells cultured on collagen matrix into guinea-pig cochleae.
    J Tissue Eng. Regen. Med., 2015 Jul 24.
    [PubMed link] [Journal link]
  • Iizuka H, Kagoya Y, Kataoka K, Yoshimi A, Miyauchi M, Taoka K, Kumano K, Yamamoto T, Hotta A, Arai S, Kurokawa M.
    Targeted gene correction of RUNX1 in induced pluripotent stem cells derived from familial platelet disorder with propensity to myeloid malignancy restores normal megakaryopoiesis.
    Experimental Hematology, 2015 June 11, 43(10): p849-57.
    [PubMed link] [Journal link]
  • Fujita H, Esaki T, Masujima T, Hotta A, Kim SH, Noji H, Watanabe TM.
    Comprehensive chemical secretory measurement of single cells trapped in a micro-droplet array with mass spectrometry.
    RSC Advances., 2015 Jan 20, 5(22):16968-71.
    [doi:10.1039/c4ra12021c] [Journal link]
  • Li HL, Fujimoto N, Sasakawa N, Shirai S, Ohkame T, Sakuma T, Tanaka M, Amano N, Watanabe A, Sakurai H, Yamamoto T, Yamanaka S, and Hotta A
    Precise correction of the Dystrophin gene in Duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.
    Stem Cell Reports, 2015; Vol.4 (1): p143-154.
    [PubMed link] [Journal link]
  • Rahmani W, Abbasi S, Hagner A, Raharjo E, Kumar R, Hotta A, Magness S, Metzger D, Biernaskie J.
    Hair follicle dermal stem cells regenerate the dermal sheath, repopulate the dermal papilla, and modulate hair type.
    Dev. Cell, 2014; Vol.31 (5): p543-558
    [PubMed link] [Journal link]
  • Matsui H, Fujimoto N, Sasakawa N, Ohinata Y, Shima M, Yamanaka S, Sugimoto M, and Hotta A
    Delivery of full-length Factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A.
    PLOS ONE, 2014; Vol.9 (8): e104957
    [PubMed link] [Journal link]
    • >朝日新聞で報道されました。
  • Kondo T, Funayama M, Tsukita K, Hotta A Yasuda A, Nori S, Kaneko S, Nakamura M, Takahashi R, Okano H, Yamanaka S, and Inoue H
    Focal transplantation of human iPSC-derived glial-rich neural progenitors improves lifespan of ALS mice.
    Stem Cell Reports, 2014; Vol.3 (2): p242-249.
    [PubMed link] [Journal link]
  • Li HL, Nakano T, Hotta A
    Genetic correction using engineered nucleases for gene therapy applications.
    Development Growth & Differentiation, 2014; Vol.56 (1): p63-77.
    [PubMed link] [Journal link]
  • Morizane A, Doi D, Kikuchi T, Okita K, Hotta A, Kawasaki T, Hayashi T, Onoe H, Shiina T, Yamanaka S, Takahash J
    Direct comparison of autologous and allogeneic transplantation of iPSC-derived neural cells in the brain of a nonhuman primate.
    Stem Cell Reports, 2013; Vol.1 (4): p283-292.
    [PubMed link] [Journal link]
  • Yamashita A, Liu S, Woltjen K, Thomas B, Meng G, Hotta A, Takahashi K, Ellis J, Yamanaka S, Rancourt DE.
    Cartilage tissue engineering identifies abnormal human induced pluripotent stem cells.
    Scientific Reports, 2013; Vol.3: 1978.
    [PubMed link] [Journal link]
  • Tanaka A, Woltjen K, Miyake K, Hotta A, Ikeya M, Yamamoto T, Nishino T, Shoji E, Sehara-Fujisawa A,Manabe Y, Fujii N, Hanaoka K, Era T, Yamashita S, Isobe K, Kimura E, Sakurai H.
    Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi myopathy in vitro.
    PLoS ONE, 2013; Vol.8 (4): e61540.
    [PubMed link] [Journal link]
  • Fussner E*, Djuric U*, Strauss M, Hotta A, Perez-Iratxeta C, Lanner F, Dilworth FJ, Ellis J, Bazett-Jones DP.
    (*: equal contribution)
    Constitutive heterochromatin reorganization during somatic cell reprogramming.
    EMBO Journal, 2011; Vol.30 (9): p1778-1789.
    [PubMed link] [Journal link]
  • Kinoshita T, Nagamatsu G, Kosaka T, Takubo K, Hotta, A, Ellis J, Suda T.
    Ataxia-telangiectasia mutated (ATM) deficiency decreases reprogramming efficiency and leads to genomic instability in iPS cells.
    Biochem Biophys Res Commun, 2011; Vol.407 (2): p321-326.
    [PubMed link] [Journal link]
  • Cheung AY, Horvath LM, Grafodatskaya D, Pasceri P, Weksberg R, Hotta, A, Carrel L, Ellis J.
    Isolation of MECP2-null Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation.
    Hum Mol Genet, 2011; Vol 20 (11): p2103-2115.
    [PubMed link] [Journal link]
  • Kattman SJ, Witty AD, Gagliardi M, Dubois NC, Niapour M, Hotta A, Ellis J, Keller G.
    Stage-specific optimization of Activin/Nodal and BMP signaling promotes cardiac differentiation of mouse and human pluripotent stem cell lines.
    Cell Stem Cell, 2011; Vol.8 (2): p228-240.
    [PubMed link] [Journal link]
  • Hotta, A, Cheung, AY, Farra, N, Garcha, K, Chang, WY, Pasceri, P, Stanford, WL, Ellis, J.
    EOS lentiviral vector selection system for human induced pluripotent stem cells.
    Nature Protocols, 2009; Vol.4 (12): p1828-1844.
    [PubMed link] [Journal link]
  • Rastegar, M, Hotta, A, Pasceri, P, Makarem, M, Cheung, AY, Elliott, S, Park, KJ, Adachi, M, Jones, FS, Clarke, ID, Dirks, P, Ellis, J.
    MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy.
    PLoS ONE, 2009; Vol.4 (8): e6810.
    [PubMed link] [Journal link]
  • Hotta, A, Cheung, AY, Farra, N, Vijayaragavan, K, Seguin, CA, Draper, JS, Pasceri, P, Maksakova, IA, Mager, DL, Rossant, J, Bhatia, M, Ellis, J.
    Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency.
    Nature Methods, 2009; Vol.6 (5): p370-376.
    [PubMed link] [Journal link]
  • Hotta, A, Ellis, J.
    Retroviral vector silencing during iPS cell induction: an epigenetic beacon that signals distinct pluripotent states.
    J Cell Biochem 2008; Vol.105 (4): p940-948.
    [PubMed link] [Journal link]
  • Buzina, A, Lo, MY, Moffett, A, Hotta, A, Fussner, E, Bharadwaj, RR, Pasceri, P, Garcia-Martinez, JV, Bazett-Jones, DP, Ellis, J.
    β-globin LCR and intron elements cooperate and direct spatial reorganization for gene therapy.
    PLoS Genet, 2008; Vol.4 (4): e1000051.
    [PubMed link] [Journal link]
  • Ellis, J, Hotta, A, Rastegar, M.
    Retrovirus silencing by an epigenetic TRIM.
    Cell, 2007; Vol.131 (1): p13-14.
    [PubMed link] [Journal link]
  • Morshed, M, Ando, M, Yamamoto, J, Hotta, A, Kaneoka, H, Kojima, J, Nishijima, K, Kamihira, M, Iijima, S.
    YY1 binds to regulatory element of chicken lysozyme and ovalbumin promoters.
    Cytotechnology, 2006; Vol.52 (3): p159-170.
    [PubMed link] [Journal link]
  • Hotta, A, Saito, Y, Kyogoku, K, Kawabe, Y, Nishijima, K, Kamihira, M, Iijima, S.
    Characterization of transient expression system for retroviral vector production.
    J Biosci Bioeng, 2006; Vol.101 (4): p361-368.
    [PubMed link] [Journal link]
  • Hotta, A, Kamihira, M, Itoh, K, Morshed, M, Kawabe, Y, Ono, K, Matsumoto, H, Nishijima, K, Iijima, S.
    Production of anti-CD2 chimeric antibody by recombinant animal cells.
    J Biosci Bioeng, 2004; Vol.98 (4): p298-303.
    [PubMed link] [Journal link]
  • Ono, K, Kamihira, M, Kuga, Y, Matsumoto, H, Hotta, A, Itoh, T, Nishijima, K, Nakamura, N, Matsuda, H, Iijima, S.
    Production of anti-prion scFv-Fc fusion proteins by recombinant animal cells.
    J Biosci Bioeng, 2003; Vol.95 (3): p231-238.
    [PubMed link] [Journal link]
  • Ifuku M, Iwabuchi KA, Tanaka M, Lung MSY, Hotta A.
    Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells.
    Methods Mol Biol., 2018;1828:191-217. doi: 10.1007/978-1-4939-8651-4_12.
    [PubMed link] [出版社リンク][2018年9月1日発行]
  • 奥嵜雄也, 堀田秋津
    ゲノム編集の非ウイルス性送達法および臨床応用戦略
    血液フロンティア, 2018; Vol.28 (07): p1077-1087.
    [出版社リンク][2018年6月30日発行]
  • 岩渕久美子, 堀田秋津
    12章　ゲノム編集によるデュシェンヌ型筋ジストロフィーの治療戦略
    医療応用をめざすゲノム編集　最新動向から技術・倫理的課題まで, 2018; p122-136. 真下知士, 金田安史 編
    [出版社リンク][2018年6月25日発行]
  • 鍵田明宏, 徐 淮耕, 堀田秋津
    ゲノム編集技術を利用した筋ジストロフィー研究および治療戦略
    実験医学増刊「超高齢社会に挑む骨格筋のメディカルサイエンス」, 2018; Vol.28 (3): p27-36 (p343-352).
    [出版社リンク][2018年4月20日発行]
  • 岩渕久美子, 堀田秋津
    ゲノム編集技術とその応用の現状
    血液フロンティア, 2018; Vol.28 (03): p343-352.
    [出版社リンク][2018年2月28日発行]
  • 堀田秋津
    iPS細胞の遺伝子変異をゲノム編集で修復する ー疾患研究と遺伝子治療の最先端
    生物の科学　遺伝, 2017; Vol.71 (05): p415-424.
    [出版社リンク][2017年9月発行]
  • 井福正隆, 堀田秋津
    iPS細胞とゲノム編集の融合による新展開
    日本臨牀, 2017; Vol.75 (05): p788-794.
    [出版社リンク][2017年5月1日発行]
  • 蒔田幸正, 穂積裕幸, 堀田秋津
    筋ジストロフィー治療応用を目指したゲノム編集技術の開発動向
    Clinical Calcium, 2017; Vol.27 (3): p67-75 (p391-399).
    [出版社リンク][2017年2月28日発行]
  • 石田賢太郎, 徐 淮耕, 堀田秋津
    第2章-8.　ヒトでのゲノム編集 -遺伝子治療応用へと動き出した現状
    実験医学増刊号　"All About ゲノム編集"
    2016年 Vol.34 No.20 p133-140. 真下知士, 山本 卓 編
    [出版社リンク][2016年12月10日発行]
  • 堀田秋津
    iPS細胞におけるゲノム編集
    学友会会誌
    2016年 Vol.36 p45-48.
    [目次PDFリンク][2016年12月発行]
  • 徐 淮耕, 堀田秋津
    Duchenne型筋ジストロフィーに対するゲノム編集戦略
    医学のあゆみ, 2016; Vol.259 (1): p73-79.
    [出版社リンク][2016年10月1日発行]
  • 李 紅梅, 堀田秋津
    第2章　ヒト人工多能性幹細胞(iPS細胞)でのゲノム編集
    実験医学別冊 "論文だけではわからない ゲノム編集成功の秘訣Q&A"
    2015年11月号; p96-105. 山本 卓 編
    [出版社リンク][2015年12月10日発行]
  • 堀田秋津, 石田賢太郎, 佐伯涼太
    疾患iPS細胞研究を加速する遺伝子改変技術の進歩
    病理と臨床, 2015年6月号; Vol.33 (6): p622-626.
    [出版社リンク][2015年6月1日発行]
  • Itaru Tsuge, Suzuki Shigehiko, Motoko Naito, Akitsu Hotta, Haruhisa Inoue
    The use of patient iPSCs for the study of neurological diseases
    Journal of Clinical and Experimental Medicine (Igaku No Ayumi), 2015; Vol.252 (7): p824-828.
    [Journal link (Japanese only)]
  • Li LH, Ryota S, Hotta A
    Chapter 5「Genome editing technologies towards stem cell research」
    Experimental Medicine "Regenerative Medicine 2015: Front line of stem cell and disease research"
    2015; Vol.33 (2): p194 (346)-200 (352).
    [Journal link (Japanese only)]
  • Hotta, A
    Genome engineering technologies to accelerate the applications of iPS cells
    Journal of Clinical and Experimental Medicine (Igaku No Ayumi), 2015; Vol.252 (2): p183-188.
    [Journal link (Japanese only)]
  • Li, LH and Hotta, A.
    Chapter 3: Editing in Cultured Human Cells: From Cell Lines to iPS Cells
    Targeted Genome Editing Using Site-Specific Nucleases, Nov 2014; Editor, Takashi Yamanoto
    [Springer Press Link]
  • Hotta, A and Yamanaka, S.
    Chapter 2: Induced Pluripotent Stem Cells
    Biomaterials and Regenerative Medicine, Sep 29th 2014: p19-33, Editor: Peter Ma
    [Cambridge University PressLink]
  • Li, LH and Hotta, A
    Future Prospects for Gene Therapy using CRISPR/Cas9
    Experimental Medicine, 2014; Vol.32 (11): p220-224.
    [Journal link]
  • Li, HL, Sakuma, T, Hotta, A, and Yamamoto, T.
    Genomic Modifications：Gene Targeting by TALENs.
    Standard ES·iPS Cell Experiments, Mar 5, 2014, Supervising Editer:Norio Nakatsuji, Editing:Hirofumi Suemori
    [Journal link]
  • Hotta, A
    Gene delivery technologies for cell reprogramming.
    Monthly Cell, 2014; Vol.46 (5): p220-224.
    [Journal link]
  • Hotta, A and Yamanaka, S.
    Chapter 2: Induced Pluripotent Stem Cells
    Biomaterials and Regenerative Medicine, Sep 2014; Editor, Peter Ma
    [Cambridge University Press Link]
  • Li, LH and Hotta, A.
    Chapter 3: Editing Cultured Human Cells: From Cell Lines to iPS Cells
    Targeted Genome Editing Using Site-Specific Nucleases, Jan 14th 2014; p45-69, Editor: Takashi Yamanoto
    [Springer Press Link]
  • Li, HL, Woltjen, K, Takahasi, K, Yamanaka, S, and Hotta, A.
    Genome Editing in Human iPS Cells by TALENs.
    Cell Technology, 2013; Vol.32 (5): p526-531.
    [Journal link] [J-STAGE link]
  • Hotta, A.
    Chemical control of iPS cells
    Trends In The Sciences, 2011; Vol.16 (5): p62-65.
    [Journal link]
  • Sciabica, K, Woltjen, K, Hotta, A.
    Multiplex gene-expression assay for human induced Pluripotent stem cells.
    Genetic Engineering & Biotechnology News (GEN), 2010; Vol.30 (3).
    [Journal link]
  • Kawabe Y, Hotta A, Ono K, Esaka K, Nishijima K, Kamihira M, Iijima S
    Production of chimeric antibodies by transgenic chicken bioreactors.
    Animal Cell Technology: Basic & Applied Aspects, 2006; Vol.14: p309-315.
    [Journal link]
  • Hotta A, Saito K, Nishijima K, Kamihira M, Iijima S
    Preparation of high-titer retroviral vectors using transient expression system.
    Animal Cell Technology: Basic & Applied Aspects, 2006; Vol.14: p293-299.
    [Journal link]
  • Hotta, A, Ono, K, Mizuarai, S, Yamaguchi, K, Nishijima, K, Kamihira, M, Iijima, S.
    Production of pharmaceutical proteins by transgenic avian.
    Animal Cell Technology: Basic & Applied Aspects, 2004; Vol.13 (0): p299-303.
    [Journal link]


Best of Cell Stem Cell 2019 Cover

Our HLA editing iPSC paper was selected for the Best of Cell Stem Cell 2019! Out of nearly 200 papers published with Cell Stem Cell in 2019, the Top 10 papers based on paper view and citation index were highlighted, including our paper. In addition, Ouchida-san's cover art is featured on the center of the journal cover. You can download the special issue of Cell Stem Cell for free from the link above.


Top 50 Life and Biological Sciences Articles Highlighted

There is no paper cover for Nature Communications as it is an online journal, but our collaboration paper with Drs. Mashimo and Takeda was featured for the Top 50 Life and Biological Sciences Articles in Nature Communications! Out of hundreds of Life Science papers, the Top 50 papers were selected based on access numbers and our paper is the 20th place.


Cell Stem Cell, April 2019 Cover

The nice illustration was drawn by Science illustrator Misaki Ouchida from the CiRA PR office. The up-right knit hats, a metaphor of iPSCs, do not fit for dogs, but by editing HLA by means of genome editing, they transformed into scarves for fiting in various dogs around the world. For researchers, it is a great honor to be highlighed on the cover. Many thanks to Ouchida-san for drawing this!


Stem Cell Reports Best of 2014-2015 Cover


Nature Methods, May 2009 Cover


Journal of Cellular Biochemistry, Nov 2008 Cover


  • April 27, 2009　Yomiuri Shimbun
  • April 27, 2009　The Globe And Mail (Canadian News paper)

    • "Green-coloured stem cells help scientists see clearly under the microscope"

  • July 15, 2010: NHK Today's Close-up

    • [Outer link (Japanese)]

  • September 18, 2010: NHK Special Program

    • [Outer link (Japanese)]

  • January 10, 2011: NHK Morning News

    • [Outer link (Japanese)]

  • November 27, 2014: NHK WORLD News

    • [Outer link]
    "Restoring gene in iPS cells to create normal cells"

  • April 24, 2015: NHK NEWS WEB

    • "Genome editing in human embryo"

  • September 20, 2015: TBS Next Door ＋

    • [Link][YouTube]

  • March 2, 2019: NHK WORLD Global Agenda "Genome Science Editing Humanity's Future"

    • [Link]