Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model-Reference-Cited by-同舟云学术

Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model

Published:2019-06 Issue:3 Volume:29 Page:148-160
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Echevarría Lucía¹²,Aupy Philippine¹,Relizani Karima¹²,Bestetti Thomas¹,Griffith Graziella¹²,Blandel Florence¹,Komisarski Marek³,Haeberli Adrian³,Svinartchouk Fedor¹²,Garcia Luis¹,Goyenvalle Aurélie¹

Affiliation:

1. Université de Versailles St- Quentin, U1179 INSERM, UFR des Sciences de la Santé, Montigny le Bretonneux, France.

2. SQY Therapeutics, Université de Versailles St-Quentin, Montigny le Bretonneux, France.

3. SYNTHENA AG, Bern, Switzerland.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2018.0773

Reference35 articles.

1. The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

2. Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

3. Current and Emerging Therapies for Duchenne Muscular Dystrophy

4. Exon-skipping advances for Duchenne muscular dystrophy

5. Oligodeoxynucleotide stability in subcellular extracts and culture media

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