Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases

by Kentaro Ishida, Peter Gee, Akitsu Hotta
Published 2015-10-01

Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy

by Peter Gee, Huaigeng Xu, Akitsu Hotta
Published 2017-01-01

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
Published 2021-06-01

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
Published 2020-08-01

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
Published 2014-01-01

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
Published 2016-04-01

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
Published 2014-08-01

Development of alternative gene transfer techniques for ex vivo and in vivo gene therapy in a canine model

by Masashi Noda, Kohei Tatsumi, Hideto Matsui, Yasunori Matsunari, Takeshi Sato, Yasushi Fukuoka, Akitsu Hotta, Teruo Okano, Kimihiko Kichikawa, Mitsuhiko Sugimoto, Midori Shima, Kenji Nishio
Published 2021-12-01

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
Published 2008-04-01

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
Published 2015-01-01

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
Published 2018-04-01

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
Published 2009-08-01

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
Published 2019-12-01

iPSC-Derived Platelets Depleted of HLA Class I Are Inert to Anti-HLA Class I and Natural Killer Cell Immunity

by Daisuke Suzuki, Charlotte Flahou, Norihide Yoshikawa, Ieva Stirblyte, Yoshikazu Hayashi, Akira Sawaguchi, Marina Akasaka, Sou Nakamura, Natsumi Higashi, Huaigeng Xu, Takuya Matsumoto, Kosuke Fujio, Markus G. Manz, Akitsu Hotta, Hitoshi Takizawa, Koji Eto, Naoshi Sugimoto
Published 2020-01-01

Correction: Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy In Vitro

by Akihito Tanaka, Knut Woltjen, Katsuya Miyake, Akitsu Hotta, Makoto Ikeya, Takuya Yamamoto, Tokiko Nishino, Emi Shoji, Atsuko Sehara-Fujisawa, Yasuko Manabe, Nobuharu Fujii, Kazunori Hanaoka, Takumi Era, Satoshi Yamashita, Ken-ichi Isobe, En Kimura, Hidetoshi Sakurai
Published 2013-01-01

Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro.

by Akihito Tanaka, Knut Woltjen, Katsuya Miyake, Akitsu Hotta, Makoto Ikeya, Takuya Yamamoto, Tokiko Nishino, Emi Shoji, Atsuko Sehara-Fujisawa, Yasuko Manabe, Nobuharu Fujii, Kazunori Hanaoka, Takumi Era, Satoshi Yamashita, Ken-Ichi Isobe, En Kimura, Hidetoshi Sakurai
Published 2013-01-01

In Vitro Disease Modeling of Hermansky-Pudlak Syndrome Type 2 Using Human Induced Pluripotent Stem Cell-Derived Alveolar Organoids

by Yohei Korogi, Shimpei Gotoh, Satoshi Ikeo, Yuki Yamamoto, Naoyuki Sone, Koji Tamai, Satoshi Konishi, Tadao Nagasaki, Hisako Matsumoto, Isao Ito, Toyofumi F. Chen-Yoshikawa, Hiroshi Date, Masatoshi Hagiwara, Isao Asaka, Akitsu Hotta, Michiaki Mishima, Toyohiro Hirai
Published 2019-03-01

Correction: Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy.

by Akihito Tanaka, Knut Woltjen, Katsuya Miyake, Akitsu Hotta, Makoto Ikeya, Takuya Yamamoto, Tokiko Nishino, Emi Shoji, Atsuko Sehara-Fujisawa, Yasuko Manabe, Nobuharu Fujii, Kazunori Hanaoka, Takumi Era, Satoshi Yamashita, Ken-ichi Isobe, En Kimura, Hidetoshi Sakurai
Published 2013-01-01

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

by In Young Choi, HoTae Lim, Kenneth Estrellas, Jyothi Mula, Tatiana V. Cohen, Yuanfan Zhang, Christopher J. Donnelly, Jean-Philippe Richard, Yong Jun Kim, Hyesoo Kim, Yasuhiro Kazuki, Mitsuo Oshimura, Hongmei Lisa Li, Akitsu Hotta, Jeffrey Rothstein, Nicholas Maragakis, Kathryn R. Wagner, Gabsang Lee
Published 2016-06-01

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

by Peter Gee, Mandy S. Y. Lung, Yuya Okuzaki, Noriko Sasakawa, Takahiro Iguchi, Yukimasa Makita, Hiroyuki Hozumi, Yasutomo Miura, Lucy F. Yang, Mio Iwasaki, Xiou H. Wang, Matthew A. Waller, Nanako Shirai, Yasuko O. Abe, Yoko Fujita, Kei Watanabe, Akihiro Kagita, Kumiko A. Iwabuchi, Masahiko Yasuda, Huaigeng Xu, Takeshi Noda, Jun Komano, Hidetoshi Sakurai, Naoto Inukai, Akitsu Hotta
Published 2020-03-01