Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies.

Hwang, Jaeho; Yokota, Toshifumi

Hwang, Jaeho; Yokota, Toshifumi.

Affiliation

Hwang J; Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, Edmonton Canada.
Yokota T; Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, Edmonton Canada.

Expert Rev Mol Med ; 21: e5, 2019 10 02.

Article in En | MEDLINE | ID: mdl-31576784

Subject(s)

Exons/genetics; Gene Editing; Genetic Therapy; Muscular Dystrophy, Duchenne/genetics; Muscular Dystrophy, Duchenne/therapy; Oligonucleotides, Antisense/genetics; Animals; Humans

Key words

Antisense oligonucleotide; CRISPR; Duchenne muscular dystrophy; Golodirsen; NS-065/NCNP-01 (viltolarsen); dysferlinopathy; eteplirsen; exon skipping; genome editing; limb-girdle muscular dystrophy type 2C; splice modulation

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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Exons / Oligonucleotides, Antisense / Muscular Dystrophy, Duchenne / Gene Editing Limits: Animals / Humans Language: En Journal: Expert Rev Mol Med Journal subject: BIOLOGIA MOLECULAR Year: 2019 Type: Article

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