Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon
Author:
Affiliation:
1. UVSQ, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.
Publisher
Mary Ann Liebert Inc
Link
https://www.liebertpub.com/doi/pdf/10.1089/nat.2024.0002
Reference11 articles.
1. Neurodevelopmental, emotional, and behavioural problems in Duchenne muscular dystrophy in relation to underlying dystrophin gene mutations
2. RNA-targeted drugs for neuromuscular diseases
3. Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
4. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
5. Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides
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