Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

by Yoshitsugu Aoki, Toshifumi Yokota, Matthew J. A. Wood
Published 2013-01-01

Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies

by Norio Motohashi, Yuko Shimizu-Motohashi, Thomas C. Roberts, Yoshitsugu Aoki
Published 2019-09-01

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

by Yuko Shimizu-Motohashi, Hirofumi Komaki, Norio Motohashi, Shin’ichi Takeda, Toshifumi Yokota, Yoshitsugu Aoki
Published 2019-01-01

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient.

by Takashi Saito, Akinori Nakamura, Yoshitsugu Aoki, Toshifumi Yokota, Takashi Okada, Makiko Osawa, Shin'ichi Takeda
Published 2010-01-01

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.

by Joshua Lee, Yusuke Echigoya, William Duddy, Takashi Saito, Yoshitsugu Aoki, Shin'ichi Takeda, Toshifumi Yokota
Published 2018-01-01

Application of Urine-Derived Stem Cells to Cellular Modeling in Neuromuscular and Neurodegenerative Diseases

by Mitsuto Sato, Mitsuto Sato, Hotake Takizawa, Akinori Nakamura, Bradley J. Turner, Fazel Shabanpoor, Yoshitsugu Aoki
Published 2019-12-01

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

by Maria K. Tsoumpra, Seiji Fukumoto, Toshio Matsumoto, Shin'ichi Takeda, Matthew J.A. Wood, Yoshitsugu Aoki
Published 2019-07-01

Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective

by Stephanie A. Fernandes, Andrew G. L. Douglas, Miguel A. Varela, Matthew J. A. Wood, Yoshitsugu Aoki
Published 2013-01-01

Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophy.

by Hideya Mizuno, Akinori Nakamura, Yoshitsugu Aoki, Naoki Ito, Soichiro Kishi, Kazuhiro Yamamoto, Masayuki Sekiguchi, Shin'ichi Takeda, Kazuo Hashido
Published 2011-01-01

Accelerometric outcomes of motor function related to clinical evaluations and muscle involvement in dystrophic dogs.

by Mutsuki Kuraoka, Yuko Nitahara-Kasahara, Hisateru Tachimori, Naohiro Kato, Hiroyuki Shibasaki, Akihiko Shin, Yoshitsugu Aoki, En Kimura, Shin'ichi Takeda
Published 2018-01-01

Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.

by Yusuke Echigoya, Joshua Lee, Merryl Rodrigues, Tetsuya Nagata, Jun Tanihata, Ashkan Nozohourmehrabad, Dharminder Panesar, Bailey Miskew, Yoshitsugu Aoki, Toshifumi Yokota
Published 2013-01-01

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

by Yusuke Echigoya, Yoshitsugu Aoki, Bailey Miskew, Dharminder Panesar, Aleksander Touznik, Tetsuya Nagata, Jun Tanihata, Akinori Nakamura, Kanneboyina Nagaraju, Toshifumi Yokota
Published 2015-01-01

Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot

by Hitoshi Suzuki, Yoshitsugu Aoki, Toshiki Kameyama, Takashi Saito, Satoru Masuda, Jun Tanihata, Tetsuya Nagata, Akila Mayeda, Shin’ichi Takeda, Toshifumi Tsukahara
Published 2016-10-01

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

by Shouta Miyatake, Yoshitaka Mizobe, Maria K. Tsoumpra, Kenji Rowel Q. Lim, Yuko Hara, Fazel Shabanpoor, Toshifumi Yokota, Shin’ichi Takeda, Yoshitsugu Aoki
Published 2019-03-01

Solid-Phase Synthesis of Difficult Purine-Rich PNAs through Selective Hmb Incorporation: Application to the Total Synthesis of Cell Penetrating Peptide-PNAs

by Julien Tailhades, Julien Tailhades, Hotake Takizawa, Michael J. Gait, Don A. Wellings, John D. Wade, John D. Wade, Yoshitsugu Aoki, Fazel Shabanpoor, Fazel Shabanpoor
Published 2017-10-01

The nSMase2/Smpd3 gene modulates the severity of muscular dystrophy and the emotional stress response in mdx mice

by Yasunari Matsuzaka, Jun Tanihata, Yoshiko Ooshima, Daisuke Yamada, Masayuki Sekiguchi, Shouta Miyatake, Yoshitsugu Aoki, Mika Terumitsu, Ryu Yashiro, Hirofumi Komaki, Akihiko Ishiyama, Yasushi Oya, Yukiko U. Inoue, Takayoshi Inoue, Shin’ichi Takeda, Kazuo Hashido
Published 2020-11-01