Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice-Reference-Cited by-同舟云学术

Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice

Published:2020-02-01 Issue:1 Volume:30 Page:50-65
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Datson Nicole A.¹,Bijl Suzanne¹,Janson Anneke¹,Testerink Janwillem¹,van den Eijnde Rani¹,Weij Rudie¹,Puoliväli Jukka²,Lehtimäki Kimmo²,Bragge Timo²,Ahtoniemi Toni²,van Deutekom Judith C.¹

Affiliation:

1. BioMarin Nederland BV, Leiden, the Netherlands.

2. Charles River Discovery Research Services, Kuopio, Finland.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2019.0824

Reference56 articles.

1. Dystrophin and mutations: one gene, several proteins, multiple phenotypes

2. Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

3. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

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