Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results-Reference-Cited by-同舟云学术

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

Published:2019-04 Issue:2 Volume:29 Page:92-103
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

van Putten Maaike¹,Tanganyika-de Winter Christa¹,Bosgra Sieto²,Aartsma-Rus Annemieke¹

Affiliation:

1. Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.

2. Biomarin Nederland BV, Leiden, the Netherlands.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2018.0759

Reference61 articles.

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4. Exon skipping: a first in class strategy for Duchenne muscular dystrophy

5. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

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