Impact of the Inhibition of Organic Anion Transporter on Tricyclo-DNA-Mediated Exon Skipping in the mdx Mouse Model
Author:
Affiliation:
1. Université de Versailles Saint Quentin en Yveline, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.
2. SQY Therapeutics, UVSQ, Montigny le Bretonneux, France.
Publisher
Mary Ann Liebert Inc
Subject
Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry
Link
https://www.liebertpub.com/doi/pdf/10.1089/nat.2023.0046
Reference16 articles.
1. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
2. Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides
3. Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial
4. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
5. Antisense oligonucleotides: absorption, distribution, metabolism, and excretion
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