Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
Author:
Affiliation:
1. McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Charlotte, NC, United States
Funder
Carolinas HealthCare Foundation
Publisher
Informa UK Limited
Subject
Pharmaceutical Science,General Medicine
Link
https://tandfonline.com/doi/pdf/10.1080/10717544.2017.1337827
Reference30 articles.
1. Pharmacokinetics, Biodistribution, Stability and Toxicity of a Cell-Penetrating Peptide−Morpholino Oligomer Conjugate
2. Effects of Complement Depletion on the Pharmacokinetics and Gene Delivery Mediated by Cationic Lipid–DNA Complexes
3. Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice
4. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
5. Antisense oligonucleotides in therapy for neurodegenerative disorders
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1. Novel Lipid-Oligonucleotide Conjugates Containing Long-Chain Sulfonyl Phosphoramidate Groups: Synthesis and Biological Properties;Applied Sciences;2021-01-27
2. Delivery of antisense oligonucleotide using polyethylenimine-based lipid nanoparticle modified with cell penetrating peptide;Drug Delivery;2019-01-01
3. In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice;Methods in Molecular Biology;2018
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