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Polymers 2017, 9(5), 177;

Evaluation of Amphiphilic Peptide Modified Antisense Morpholino Oligonucleotides In Vitro and in Dystrophic mdx Mice

McColl-Lockwood Laboratory for Muscular Dystrophy Research, Carolinas Medical Center, 1000 Blythe Blvd, Charlotte, NC 28231, USA
Author to whom correspondence should be addressed.
Academic Editors: Ravin Narain and Shiyong Liu
Received: 21 March 2017 / Revised: 28 April 2017 / Accepted: 8 May 2017 / Published: 15 May 2017
(This article belongs to the Special Issue Polymers and Nanogels for Gene Therapy)
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A series of amphiphilic peptides modified PMO (Pt-PMO) were prepared, and their antisense effect and toxicity were evaluated both in vitro and in mdx mice. The results showed that the exon-skipping performance of Pt-PMO are relative to the structure of the conjugated peptide: the Pt3/Pt4 composed of six/seven arginines and one myristoylation modified PMO showed more efficacy and with less toxicity as compared to others, confirming that appropriate hydrophilic-lipophilic balance (HLB) and cationic sequence numbers play a crucial role in improving cell uptake and corresponding exon-skipping efficiency. This was observed particularly in enhanced delivery efficiency of PMO comparable to B-PMO in vitro, while 6-fold improved exon-skipping was achieved against naked PMO in vivo. The multi-PMO modified Pt8-PMO also showed improved exon-skipping both in vitro and in vivo, though there is lower efficiency in systemic delivery as compared to Pt4-PMO. These data suggest that with optimization of peptide in component, charge density has clear potential for exploration towards achieving higher efficiency of antisense oligonucleotide systemic delivery, and thus is more applicable for clinical application. View Full-Text
Keywords: amphiphilic peptide; exon-skipping; antisense oligonucleotide; muscular dystrophy amphiphilic peptide; exon-skipping; antisense oligonucleotide; muscular dystrophy

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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Wang, M.; Wu, B.; Lu, P.; Shah, S.N.; Tucker, J.D.; Bollinger, L.E.; Lu, Q. Evaluation of Amphiphilic Peptide Modified Antisense Morpholino Oligonucleotides In Vitro and in Dystrophic mdx Mice. Polymers 2017, 9, 177.

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