Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.

Hara, Yuko; Mizobe, Yoshitaka; Miyatake, Shouta; Takizawa, Hotake; Nagata, Tetsuya; Yokota, Toshifumi; Takeda, Shin'ichi; Aoki, Yoshitsugu

Hara, Yuko; Mizobe, Yoshitaka; Miyatake, Shouta; Takizawa, Hotake; Nagata, Tetsuya; Yokota, Toshifumi; Takeda, Shin'ichi; Aoki, Yoshitsugu.

Afiliação

Hara Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan.
Mizobe Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan.
Miyatake S; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan.
Takizawa H; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan.
Nagata T; Department of Neurology and Neurological Science, Tokyo Medical and Dental University, Tokyo, Japan.
Yokota T; Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, Edmonton, AB, Canada.
Takeda S; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan.
Aoki Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan. tsugu56@ncnp.go.jp.

Methods Mol Biol ; 1828: 553-564, 2018.

Article em En | MEDLINE | ID: mdl-30171567

Assuntos

Éxons; Laminina/deficiência; Distrofias Musculares/genética; Oligonucleotídeos Antissenso/genética; Splicing de RNA; Animais; Modelos Animais de Doenças; Fibroblastos/metabolismo; Regulação da Expressão Gênica; Imuno-Histoquímica; Camundongos; Camundongos Transgênicos; Morfolinos/administração & dosagem; Morfolinos/genética; Músculo Esquelético/metabolismo; Músculo Esquelético/patologia; Oligonucleotídeos Antissenso/administração & dosagem

Palavras-chave

Dystrophin; Eteplirsen; Exon skipping; Laminin-α2 chain; Merosin-deficient congenital muscular dystrophy type 1A (MDC1A); NS-065/NCNP-01; Phosphorodiamidate morpholino oligomer (PMO); dy 3K/dy 3K mouse

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Splicing de RNA / Éxons / Oligonucleotídeos Antissenso / Laminina / Distrofias Musculares Idioma: En Ano de publicação: 2018 Tipo de documento: Article

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