Morpholinos with cell-penetrating peptides for in vivo applications
Jon Moulton
Wednesday, 27 June 2007 22:50 UTC
Following are references related to conjugation of Morpholino oligos with arginine-rich peptides.
Amantana A, Moulton HM, Cate ML, Reddy MT, Whitehead T, Hassinger JN, Youngblood DS, Iversen PL. Pharmacokinetics, Biodistribution, Stability and Toxicity of a Cell-Penetrating Peptide-Morpholino Oligomer Conjugate. Bioconjug Chem. 2007 Jun 21; [Epub ahead of print] {in-vivo rat data}
Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Steinhaus JP, Moulton HM, Iversen PL, Wilton SD. Morpholino Oligomer-Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse. Mol Ther. 2007 Jun 19; [Epub ahead of print] {in-vivo mouse data}
Deas TS, Bennett CJ, Jones SA, Tilgner M, Ren P, Behr MJ, Stein DA, Iversen PL, Kramer LD, Bernard KA, Shi PY. In vitro resistance selection and in vivo efficacy of morpholino oligomers against West Nile virus. Antimicrob Agents Chemother. 2007 May 7; [Epub ahead of print] {in-vivo mouse data}
Burrer R, Neuman BW, Ting JPC, Stein DA, Moulton HM, Iversen PL, Kuhn P, Buchmeier MJ. Antiviral effects of antisense morpholino oligomers in murine coronavirus infection models. J. Virol. 2007 Epub 2007 Mar 5.{in-vivo mouse data}
Abes S, Moulton HM, Clair P, Prevot P, Youngblood DS, Wu RP, Iversen PL, Lebleu B. Vectorization of morpholino oligomers by the (R-Ahx-R)(4) peptide allows efficient splicing correction in the absence of endosomolytic agents. J Control Release. 2006 Sep 30; [Epub ahead of print] {nice mechanism study, also a good place to start on how to conjugate}
Youngblood DS, Hatlevig SA, Hassinger JN, Iversen PL, Moulton HM. Stability of cell-penetrating Peptide-morpholino oligomer conjugates in human serum and in cells. Bioconjug Chem. 2007 Jan-Feb;18(1):50-60.
McClorey G, Fall AM, Moulton HM, Iversen PL, Rasko JE, Ryan M, Fletcher S, Wilton SD. Induced dystrophin exon skipping in human muscle explants. Neuromuscul Disord. 2006 Oct;16(9-10):583-90. Epub 2006 Aug 21 {application paper, little background on the conjugates}
McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther. 2006 Oct;13(19):1373-81. Epub 2006 May 25 {application paper, little background on the conjugates}
Deas TS, Binduga-Gajewska I, Tilgner M, Ren P, Stein DA, Moulton HM, Iversen PL, Kauffman EB, Kramer LD, Shi PY. Inhibition of flavivirus infections by antisense oligomers specifically suppressing viral translation and RNA replication. J Virol. 2005 Apr;79(8):4599-609 {application paper, little background on the conjugates}
Nelson MH, Stein DA, Kroeker AD, Hatlevig SA, Iversen PL, Moulton HM. Arginine-rich peptide conjugation to morpholino oligomers: effects on antisense activity and specificity. Bioconjug Chem. 2005 Jul-Aug;16(4):959-66. {Nice paper specifically on the conjugates}
Moulton HM, Moulton JD. Arginine-rich cell-penetrating peptides with uncharged antisense oligomers. Drug Discov Today. 2004 Oct 15;9(20):870. {argument for the effectiveness of the Morpholino-peptide system}
Moulton HM, Nelson MH, Hatlevig SA, Reddy MT, Iversen PL. Cellular uptake of antisense morpholino oligomers conjugated to arginine-rich peptides. Bioconjug Chem. 2004 Mar-Apr;15(2):290-9. {Nice paper specifically on the conjugates}
Moulton HM, Moulton JD. Peptide-assisted delivery of steric-blocking antisense oligomers. Curr Opin Mol Ther. 2003 Apr;5(2):123-32. {review}
Moulton HM, Hase MC, Smith KM, Iversen PL. HIV Tat peptide enhances cellular delivery of antisense morpholino oligomers. Antisense Nucleic Acid Drug Dev. 2003 Feb;13(1):31-43. {early exploration with less-optimal peptide}
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