Identification of a novel muscle targeting peptide in mdx mice.

Peptides
Yiqi SeowMatthew J A Wood

Abstract

Exon-skipping oligonucleotides are a well-researched therapeutic strategy for Duchenne's muscular dystrophy (DMD). Despite remarkable successes in animal models with intramuscular and intravenous delivery of unmodified oligonucleotides, the ability to specifically target both normal and dystrophic muscle with a simple peptide ligand could decrease the therapeutic dose required and reduce the potential for toxicity. Thus, 3 rounds of in vivo phage display utilizing a 12-mer peptide library were performed with mdx mice and a peptide motif with potential for targeting to muscle but not liver was identified. This motif was shown to have enhanced binding affinity to C2C12 myoblasts over a scrambled control peptide and in vivo application of a fluorescein-labeled peptide containing the identified motif resulted in increased specificity for the heart and quadriceps muscle after tail-vein administration in C57BL/6 mice. This work has many potential applications for oligonucleotide or drug delivery to muscle for myopathies.

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Citations

Dec 5, 2013·Chemical Reviews·Bethany Powell Gray, Kathlynn C Brown
Apr 16, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Xianjun GaoHaiFang Yin
May 20, 2015·Molecules : a Journal of Synthetic Chemistry and Natural Product Chemistry·Tirsa L E van WesteringMatthew J A Wood
Apr 17, 2013·Biotechnology and Applied Biochemistry·L'ubomíra TóthováPeter Celec
Apr 30, 2013·Biotechnology Advances·Janka BábíčkováPeter Celec
Nov 3, 2016·Expert Opinion on Biological Therapy·S FletcherS D Wilton
Aug 1, 2020·Frontiers in Pharmacology·Yi LiPeifu Tang
Oct 20, 2020·Expert Opinion on Biological Therapy·Anne-Fleur E Schneider, Annemieke Aartsma-Rus
Oct 18, 2020·Molecular Therapy : the Journal of the American Society of Gene Therapy·Feng YueShihuan Kuang

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