Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy by Peter Gee, Huaigeng Xu, Akitsu Hotta

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iMSC-mediated delivery of ACVR2B-Fc fusion protein reduces heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva by Pan Gao, Yoshiko Inada, Akitsu Hotta, Hidetoshi Sakurai, Makoto Ikeya

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A muscle fatigue-like contractile decline was recapitulated using skeletal myotubes from Duchenne muscular dystrophy patient-derived iPSCs by Tomoya Uchimura, Toshifumi Asano, Takao Nakata, Akitsu Hotta, Hidetoshi Sakurai

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Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs by Ryu Kawada, Tatsuya Jonouchi, Akihiro Kagita, Masae Sato, Akitsu Hotta, Hidetoshi Sakurai

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Optimized simple culture protocol for inducing mature myotubes from MYOD1-overexpressed human iPS cells by Eiji Wada, Nao Susumu, Yuya Okuzaki, Akitsu Hotta, Hidetoshi Sakurai, Yukiko K. Hayashi

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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 by Mitsuru Sasaki-Honda, Akihiro Kagita, Tatsuya Jonouchi, Toshiyuki Araki, Akitsu Hotta, Hidetoshi Sakurai

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Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A. by Hideto Matsui, Naoko Fujimoto, Noriko Sasakawa, Yasuhide Ohinata, Midori Shima, Shinya Yamanaka, Mitsuhiko Sugimoto, Akitsu Hotta

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Dual inhibition of TMPRSS2 and Cathepsin Bprevents SARS-CoV-2 infection in iPS cells by Rina Hashimoto, Ayaka Sakamoto, Sayaka Deguchi, Renxing Yi, Emi Sano, Akitsu Hotta, Kazutoshi Takahashi, Shinya Yamanaka, Kazuo Takayama

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An EWS-FLI1-Induced Osteosarcoma Model Unveiled a Crucial Role of Impaired Osteogenic Differentiation on Osteosarcoma Development by Shingo Komura, Katsunori Semi, Fumiaki Itakura, Hirofumi Shibata, Takatoshi Ohno, Akitsu Hotta, Knut Woltjen, Takuya Yamamoto, Haruhiko Akiyama, Yasuhiro Yamada

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Contractile Activity of Myotubes Derived from Human Induced Pluripotent Stem Cells: A Model of Duchenne Muscular Dystrophy by Kantaro Yoshioka, Akira Ito, Masanobu Horie, Kazushi Ikeda, Sho Kataoka, Keiichiro Sato, Taichi Yoshigai, Hidetoshi Sakurai, Akitsu Hotta, Yoshinori Kawabe, Masamichi Kamihira

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Focal Transplantation of Human iPSC-Derived Glial-Rich Neural Progenitors Improves Lifespan of ALS Mice by Takayuki Kondo, Misato Funayama, Kayoko Tsukita, Akitsu Hotta, Akimasa Yasuda, Satoshi Nori, Shinjiro Kaneko, Masaya Nakamura, Ryosuke Takahashi, Hideyuki Okano, Shinya Yamanaka, Haruhisa Inoue

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Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice by Eriya Kenjo, Hiroyuki Hozumi, Yukimasa Makita, Kumiko A. Iwabuchi, Naoko Fujimoto, Satoru Matsumoto, Maya Kimura, Yuichiro Amano, Masataka Ifuku, Youichi Naoe, Naoto Inukai, Akitsu Hotta

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Beta-globin LCR and intron elements cooperate and direct spatial reorganization for gene therapy. by Alla Buzina, Mandy Y M Lo, Angela Moffett, Akitsu Hotta, Eden Fussner, Rikki R Bharadwaj, Peter Pasceri, J Victor Garcia-Martinez, David P Bazett-Jones, James Ellis

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Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application by Yuko Kitano, Sayaka Nishimura, Tomoaki M. Kato, Anna Ueda, Kaho Takigawa, Masafumi Umekage, Masaki Nomura, Ayane Kawakami, Haruna Ogawa, Huaigeng Xu, Akitsu Hotta, Naoko Takasu, Masayoshi Tsukahara

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Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9 by Hongmei Lisa Li, Naoko Fujimoto, Noriko Sasakawa, Saya Shirai, Tokiko Ohkame, Tetsushi Sakuma, Michihiro Tanaka, Naoki Amano, Akira Watanabe, Hidetoshi Sakurai, Takashi Yamamoto, Shinya Yamanaka, Akitsu Hotta

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iPSC-derived type IV collagen α5-expressing kidney organoids model Alport syndrome by Ryuichiro Hirayama, Kosuke Toyohara, Kei Watanabe, Takeya Otsuki, Toshikazu Araoka, Shin-Ichi Mae, Tomoko Horinouchi, Tomohiko Yamamura, Keisuke Okita, Akitsu Hotta, Kazumoto Iijima, Kandai Nozu, Kenji Osafune

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Srf destabilizes cellular identity by suppressing cell-type-specific gene expression programs by Takashi Ikeda, Takafusa Hikichi, Hisashi Miura, Hirofumi Shibata, Kanae Mitsunaga, Yosuke Yamada, Knut Woltjen, Kei Miyamoto, Ichiro Hiratani, Yasuhiro Yamada, Akitsu Hotta, Takuya Yamamoto, Keisuke Okita, Shinji Masui

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MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy. by Mojgan Rastegar, Akitsu Hotta, Peter Pasceri, Maisam Makarem, Aaron Y L Cheung, Shauna Elliott, Katya J Park, Megumi Adachi, Frederick S Jones, Ian D Clarke, Peter Dirks, James Ellis

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CRISPR-Cas3 induces broad and unidirectional genome editing in human cells by Hiroyuki Morisaka, Kazuto Yoshimi, Yuya Okuzaki, Peter Gee, Yayoi Kunihiro, Ekasit Sonpho, Huaigeng Xu, Noriko Sasakawa, Yuki Naito, Shinichiro Nakada, Takashi Yamamoto, Shigetoshi Sano, Akitsu Hotta, Junji Takeda, Tomoji Mashimo

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CD151 expression marks atrial- and ventricular- differentiation from human induced pluripotent stem cells by Misato Nakanishi-Koakutsu, Kenji Miki, Yuki Naka, Masako Sasaki, Takayuki Wakimizu, Stephanie C. Napier, Chikako Okubo, Megumi Narita, Misato Nishikawa, Reo Hata, Kazuhisa Chonabayashi, Akitsu Hotta, Kenichi Imahashi, Tomoyuki Nishimoto, Yoshinori Yoshida

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