Genetic Approaches for the Treatment of Facioscapulohumeral Muscular Dystrophy

por Lim, Kenji Rowel Q., Yokota, Toshifumi
Publicado 2021

Eteplirsen in the treatment of Duchenne muscular dystrophy

por Lim, Kenji Rowel Q, Maruyama, Rika, Yokota, Toshifumi
Publicado 2017

Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy

por Lim, Kenji Rowel Q., Yoon, Chantal, Yokota, Toshifumi
Publicado 2018

Current understanding and treatment of cardiac and skeletal muscle pathology in laminin-α2 chain-deficient congenital muscular dystrophy

por Nguyen, Quynh, Lim, Kenji Rowel Q, Yokota, Toshifumi
Publicado 2019

Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies

por Nguyen, Quynh, Lim, Kenji Rowel Q., Yokota, Toshifumi
Publicado 2020

DUX4 Signalling in the Pathogenesis of Facioscapulohumeral Muscular Dystrophy

por Lim, Kenji Rowel Q., Nguyen, Quynh, Yokota, Toshifumi
Publicado 2020

Genotype–Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry

por Lim, Kenji Rowel Q., Nguyen, Quynh, Yokota, Toshifumi
Publicado 2020

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

por Echigoya, Yusuke, Lim, Kenji Rowel Q., Nakamura, Akinori, Yokota, Toshifumi
Publicado 2018

Cardiac Involvement in Dystrophin-Deficient Females: Current Understanding and Implications for the Treatment of Dystrophinopathies

por Lim, Kenji Rowel Q., Sheri, Narin, Nguyen, Quynh, Yokota, Toshifumi
Publicado 2020

The Immunology of Takotsubo Syndrome

por Lim, Kenji Rowel Q., Mann, Douglas L., Kenzaka, Tsuneaki, Hayashi, Tomohiro
Publicado 2023

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

por Lim, Kenji Rowel Q., Nguyen, Quynh, Dzierlega, Kasia, Huang, Yiqing, Yokota, Toshifumi
Publicado 2020

A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-...

por Anwar, Saeed, He, Merry, Lim, Kenji Rowel Q., Maruyama, Rika, Yokota, Toshifumi
Publicado 2021

Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background

por Rodrigues, Merryl, Echigoya, Yusuke, Maruyama, Rika, Lim, Kenji Rowel Q., Fukada, So-ichiro, Yokota, Toshifumi
Publicado 2016

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

por Miyatake, Shouta, Mizobe, Yoshitaka, Tsoumpra, Maria K., Lim, Kenji Rowel Q., Hara, Yuko, Shabanpoor, Fazel, Yokota, Toshifumi, Takeda, Shin’ichi, Aoki, Yoshitsugu
Publicado 2019

Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform

por Lim, Kenji Rowel Q., Shah, Md Nur Ahad, Woo, Stanley, Wilton-Clark, Harry, Zhabyeyev, Pavel, Wang, Faqi, Maruyama, Rika, Oudit, Gavin Y., Yokota, Toshifumi
Publicado 2021

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

por Aslesh, Tejal, Erkut, Esra, Ren, Jun, Lim, Kenji Rowel Q., Woo, Stanley, Hatlevig, Susan, Moulton, Hong M., Gosgnach, Simon, Greer, John, Maruyama, Rika, Yokota, Toshifumi
Publicado 2023

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

por Lim, Kenji Rowel Q., Woo, Stanley, Melo, Dyanna, Huang, Yiqing, Dzierlega, Kasia, Shah, Md Nur Ahad, Aslesh, Tejal, Roshmi, Rohini Roy, Echigoya, Yusuke, Maruyama, Rika, Moulton, Hong M., Yokota, Toshifumi
Publicado 2022

eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping

por Chiba, Shuntaro, Lim, Kenji Rowel Q, Sheri, Narin, Anwar, Saeed, Erkut, Esra, Shah, Md Nur Ahad, Aslesh, Tejal, Woo, Stanley, Sheikh, Omar, Maruyama, Rika, Takano, Hiroaki, Kunitake, Katsuhiko, Duddy, William, Okuno, Yasushi, Aoki, Yoshitsugu, Yokota, Toshifumi
Publicado 2021