Morpholino Oligomers

See color plate.) Polymer-coated stent delivery of c-myc antisense phosphorodiamidate morpholino oligomers into swine vessels. 

Abes, S., Moulton, H. M., Clair, R, Prevot, P, Youngblood, D. S., Wu, R. P, Iversen, P. L., and Lebleu, B. (2006) Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endoso-molytic agents. J Control Release. 116, 304-313. 

Studies with PNA analogs have shown that these analogs can inhibit translation in cell-free systems, but to date no data have been reported from cellular studies (114,115). For morpholino oligomers, antisense activity has been reported in both cell-free and cellular assays (116, 117). Numerous oligonucleotides... 

Liu GZ, He J, Dou SP et al (2004) Pretargeting in tumored mice with radiolabeled morpholino oligomer showing low kidney uptake. Eur J Nucl Med Mol Imaging 31 417-424 Liu S, Edwards DS (1999) Tc-99m-labelled small peptides as diagnostic radiopharmaceuticals. Chem Rev 99 2235-2268... 

Figure 28. Morpholino oligomers synthesis starting from hexopyranoses.

Moulton H M, Hase M C, Smith K M, et al. (2003). HIV Tat peptide enhances cellular delivery of antisense morpholino oligomers. Antisense Nucleic Acid Drug Dev. 13 31-43. 

Devi G R, Beer T M, et al. (2005). In vivo bioavailability and pharmacokinetics of a c-MYC antisense phosphorodiamidate morpholino oligomer, AVI-4126, in solid tumors. Clin. Cancer Res. 11 3930-3938. 

Pannier A K, Arora V, Iversen P L, et al. (2004). Transdermal delivery of phospho-rodiamidate Morpholino oligomers across hairless mouse skin. Int. J. Pharm. 275 217-226. 

Iversen P L (2001). Phosphorodiamidate morpholino oligomers favorable properties for sequence-specific gene inactivation. Curr. Opin. Mol. Ther. 3 235-238. 

Iversen P L, Arora V, Acker A J, et al. (2003). Efficacy of antisense morpholino oligomer targeted to c-myc in prostate cancer xenograft murine model and a Phase I safety study in humans. Clin. Cancer Res. 9 2510-2519. 

FIGURE 4.1 Chemical modifications to ribose sugar or phosphate linkages. BNA, bridged nucleic acid LNA, locked nucleic acid PMO, phosphorodiamidate morpholino oligomer. 

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