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In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice

Publikováno v: Methods in Molecular Biology ISBN: 9781071630358

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::a23a171a5abe369001b11c9720e225f5
https://doi.org/10.1007/978-1-0716-3036-5_23

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

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

Publikováno v: Nucleic Acids Research

Exon skipping using antisense oligonucleotides (ASOs) has recently proven to be a powerful tool for mRNA splicing modulation. Several exon-skipping ASOs have been approved to treat genetic diseases worldwide. However, a significant challenge is the d

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9a7af91e7beb693ca290bf9c2edfb7ca
https://doi.org/10.1093/nar/gkab442

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

Autor: Quynh Nguyen, Narin Sheri, Toshifumi Yokota, Kenji Rowel Q. Lim

Publikováno v: Genes
Genes, Vol 11, Iss 765, p 765 (2020)

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive condition caused primarily by out-of-frame mutations in the dystrophin gene. In males, DMD presents with progressive body-wide muscle deterioration, culminating in death as a result of c

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4e040a54cc43a48d724e17f5cf146f52
http://europepmc.org/articles/PMC7397028

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