Abes, R., Arzumanov, A.A., Moulton, H.M., Abes, S., Ivanova, G.D., Iversen, P.L., Gait, M.J., and Lebleu, B. (2007). Cell-penetrating-peptide-based delivery of oligonucleotides: an overview. Biochem Soc Trans 35, 775-779.

Abes, S., Moulton, H.M., Clair, P., 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 endosomolytic agents. J Control Release 116, 304-313.

Alter, J., Lou, F., Rabinowitz, A., Yin, H., Rosenfeld, J., Wilton, S.D., Partridge, T.A., and Lu, Q.L. (2006). Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12, 175-177.

Arora, V., Devi, G.R., and Iversen, P.L. (2004). Neutrally charged phosphorodiamidate mor-pholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol 5, 431-439.

Bostick, B., Yue, Y., Long, C., and Duan, D. (2008). Prevention of dystrophin-deficient cardio-myopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression. Circ Res 102, 121-130.

Bruno, I.G., Jin, W., and Cote, G.J. (2004). Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements. Hum Mol Genet 13, 2409-2420.

Denti, M.A., Incitti, T., Sthandier, O., Nicoletti, C., De Angelis, F.G., Rizzuto, E., Auricchio, A., Musaro, A., and Bozzoni, I. (2008). Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. Hum Gene Ther 19, 601-608.

Ding, Y., Xi, Y., Chen, T., Wang, J.Y., Tao, D.L., Wu, Z.L., Li, Y.P., Li, C., Zeng, R., and Li, L. (2008). Caprin-2 enhances canonical Wnt signaling through regulating LRP5/6 phosphoryla-tion. J Cell Biol 182, 865-872.

Fletcher, S., Honeyman, K., Fall, A.M., Harding, P.L., Johnsen, R.D., Steinhaus, J.P., Moulton, H.M., Iversen, P.L., and Wilton, S.D. (2007). Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Mol Ther 15, 1587-1592.

Foster, K., Foster, H., and Dickson, J.G. (2006). Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Ther 13, 1677-1685.

Futaki, S., Nakase, I., Suzuki, T., Youjun, Z., and Sugiura, Y. (2002). Translocation of branched-chain arginine peptides through cell membranes: flexibility in the spatial disposition of positive charges in membrane-permeable peptides. Biochemistry 41, 7925-7930.

Gebski, B.L., Mann, C.J., Fletcher, S., and Wilton, S.D. (2003). Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet 12, 1801-1811.

Ivanova, G.D., Arzumanov, A., Abes, R., Yin, H., Wood, M.J., Lebleu, B., and Gait, M.J. (2008). Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 36, 6418-6428.

Jearawiriyapaisarn, N., Moulton, H.M., Buckley, B., Roberts, J., Sazani, P., Fucharoen, S., Iversen, P.L., and Kole, R. (2008). Sustained dystrophin expression induced by peptide-conju-gated morpholino oligomers in the muscles of mdx mice. Mol Ther 16, 1624-1629.

Lebleu, B., Moulton, H.M., Abes, R., Ivanova, G.D., Abes, S., Stein, D.A., Iversen, P.L., Arzumanov, A.A., and Gait, M.J. (2008). Cell penetrating peptide conjugates of steric block oligonucleotides. Adv Drug Deliv Rev 60, 517-529.

Li, Y.F., and Morcos, P.A. (2008). Design and synthesis of dendritic molecular transporter that achieves efficient in vivo delivery of morpholino antisense oligo. Bioconjug Chem 19, 1464-1470.

Lu, Q.L., Mann, C.J., Lou, F., Bou-Gharios, G., Morris, G.E., Xue, S.A., Fletcher, S., Partridge, T.A., and Wilton, S.D. (2003). Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med 9, 1009-1014.

Lu, Q.L., Rabinowitz, A., Chen, Y.C., Yokota, T., Yin, H., Alter, J., Jadoon, A., Bou-Gharios, G., and Partridge, T. (2005). Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc. Natl Acad Sci U S A 102, 198-203.

Mann, C.J., Honeyman, K., Cheng, A.J., Ly, T., Lloyd, F., Fletcher, S., Morgan, J.E., Partridge, T.A., and Wilton, S.D. (2001). Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A 98, 42-47.

Matsuda, R., Nishikawa, A., and Tanaka, H. (1995). Visualization of dystrophic muscle fibers in mdx mouse by vital staining with Evans blue: evidence of apoptosis in dystrophin-deficient muscle. J Biochem (Tokyo) 118, 959-964.

Nasevicius, A., and Ekker, S.C. (2000). Effective targeted gene 'knockdown' in zebrafish. Nat Genet 26, 216-220.

Quinlan, J.G., Hahn, H.S., Wong, B.L., Lorenz, J.N., Wenisch, A.S., and Levin, L.S. (2004). Evolution of the mdx mouse cardiomyopathy: physiological and morphological findings. Neuromuscul Disord 14, 491-496.

Sazani, P., Gemignani, F., Kang, S.H., Maier, M.A., Manoharan, M., Persmark, M., Bortner, D., and Kole, R. (2002). Systemically delivered antisense oligomers upregulate gene expression in mouse tissues. Nat Biotechnol 20, 1228-1233.

Silver, M.M., Banerjee, D., and Hudson, A.J. (1983). Segmental myofiber necrosis in myotonic dystrophy - An immunoperoxidase study of immunoglobulins in skeletal muscle. Am J Pathol 112, 294-301.

Summerton, J., and Weller, D. (1997). Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug. Dev 7, 187-195.

Townsend, D., Yasuda, S., Li, S., Chamberlain, J.S., and Metzger, J.M. (2008). Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Mol Ther 16, 832-835.

van Deutekom, J.C., Janson, A.A., Ginjaar, I.B., Frankhuizen, W.S., Aartsma-Rus, A., Bremmer-Bout, M., den Dunnen, J.T., Koop, K., van der Kooi, A.J., Goemans, N.M., et al. (2007). Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 357, 2677-2686.

Vitiello, L., Bassi, N., Campagnolo, P., Zaccariotto, E., Occhi, G., Malerba, A., Pigozzo, S., Reggiani, C., Ausoni, S., Zaglia, T., et al. (2008). In vivo delivery of naked antisense oligos in aged mdx mice: analysis of dystrophin restoration in skeletal and cardiac muscle. Neuromuscul Disord 18, 597-605.

Wagner, K.R., Lechtzin, N., and Judge, D.P. (2007). Current treatment of adult Duchenne muscular dystrophy. Biochim Biophys Acta 1772, 229-237.

Wu, B., Moulton, H.M., Iversen, P.L., Jiang, J., Li, J., Spurney, C.F., Sali, A., Guerron, A.D., Nagaraju, K., Doran, T., et al. (2008). Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci U S A 105, 14814-14819.

Wu, B., Li, Y., Morcos, P., Doran, T., Lu, P., Lu, Q. (2009). Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol Ther 17, 864-871.

Yin, H., Lu, Q., and Wood, M. (2008). Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 16, 38-45.

Yuan, J., Stein, D.A., Lim, T., Qiu, D., Coughlin, S., Liu, Z., Wang, Y., Blouch, R., Moulton, H.M., Iversen, P.L., et al. (2006). Inhibition of coxsackievirus B3 in cell cultures and in mice by peptide-conjugated morpholino oligomers targeting the internal ribosome entry site. J Virol 80, 11510-11519.

0 0

Post a comment