Carlson College of Veterinary Medicine

S. Abes, Moulton, H. M., Turner, J., Clair, P., Richard, J. P., Iversen, P., Gait, M. J., and Lebleu, B., “Peptide-based delivery of nucleic acids: design, mechanism of uptake and applications to splice-correcting oligonucleotides.”, Biochemical Society transactions, vol. 35, no. Pt 1, pp. 53-5, 2007.

A. Amantana, Moulton, H. M., Cate, M. L., Reddy, M. T., Whitehead, T., Hassinger, J. N., Youngblood, D. S., and Iversen, P. L., “Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate.”, Bioconjugate chemistry, vol. 18, no. 4, pp. 1325-31, 2007.

D. S. Youngblood, Hatlevig, S. A., Hassinger, J. N., Iversen, P. L., and Moulton, H. M., “Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells.”, Bioconjugate chemistry, vol. 18, no. 1, pp. 50-60, 2007.

R. Abes, Arzumanov, A., Moulton, H. M., Abes, S., Ivanova, G., Gait, M. J., Iversen, P., and Lebleu, B., “Arginine-rich cell penetrating peptides: design, structure-activity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides.”, Journal of peptide science : an official publication of the European Peptide Society, vol. 14, no. 4, pp. 455-60, 2008.

B. Lebleu, Moulton, H. M., Abes, R., Ivanova, G. D., Abes, S., Stein, D. A., Iversen, P. L., Arzumanov, A. A., and Gait, M. J., “Cell penetrating peptide conjugates of steric block oligonucleotides.”, Advanced drug delivery reviews, vol. 60, no. 4-5, pp. 517-29, 2008.

H. Yin, Moulton, H. M., Seow, Y., Boyd, C., Boutilier, J., Iverson, P., and Wood, M. J. A., “Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.”, Human molecular genetics, vol. 17, no. 24, pp. 3909-18, 2008.

R. Abes, Moulton, H. M., Clair, P., Yang, S. - T., Abes, S., Melikov, K., Prevot, P., Youngblood, D. S., Iversen, P. L., Chernomordik, L. V., and Lebleu, B., “Delivery of steric block morpholino oligomers by (R-X-R)4 peptides: structure-activity studies.”, Nucleic acids research, vol. 36, no. 20, pp. 6343-54, 2008.

B. Wu, Moulton, H. M., Iversen, P. L., Jiang, J., Li, J., Li, J., Spurney, C. F., Sali, A., Guerron, A. D., Nagaraju, K., Doran, T., Lu, P., Xiao, X., and Lu, Q. Long, “Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.”, Proceedings of the National Academy of Sciences of the United States of America, vol. 105, no. 39, pp. 14814-9, 2008.

N. Jearawiriyapaisarn, Moulton, H. M., Buckley, B., Roberts, J., Sazani, P., Fucharoen, S., Iversen, P. L., and Kole, R., “Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.”, Molecular therapy : the journal of the American Society of Gene Therapy, vol. 16, no. 9, pp. 1624-9, 2008.

D. L. Swenson, Warfield, K. L., Warren, T. K., Lovejoy, C., Hassinger, J. N., Ruthel, G., Blouch, R. E., Moulton, H. M., Weller, D. D., Iversen, P. L., and Bavari, S., “Chemical modifications of antisense morpholino oligomers enhance their efficacy against Ebola virus infection.”, Antimicrobial agents and chemotherapy, vol. 53, no. 5, pp. 2089-99, 2009.

H. Yin, Moulton, H. M., Betts, C., Seow, Y., Boutilier, J., Iverson, P. L., and Wood, M. J. A., “A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.”, Human molecular genetics, vol. 18, no. 22, pp. 4405-14, 2009.

H. M. Moulton, Wu, B., Jearawiriyapaisarn, N., Sazani, P., Lu, Q. Long, and Kole, R., “Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.”, Annals of the New York Academy of Sciences, vol. 1175, pp. 55-60, 2009.

S. Svasti, Suwanmanee, T., Fucharoen, S., Moulton, H. M., Nelson, M. H., Maeda, N., Smithies, O., and Kole, R., “RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.”, Proceedings of the National Academy of Sciences of the United States of America, vol. 106, no. 4, pp. 1205-10, 2009.

S. Fletcher, Adams, A. M., Johnsen, R. D., Greer, K., Moulton, H. M., and Wilton, S. D., “Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.”, Molecular therapy : the journal of the American Society of Gene Therapy, vol. 18, no. 6, pp. 1218-23, 2010.

H. Yin, Moulton, H. M., Betts, C., Merritt, T., Seow, Y., Ashraf, S., Wang, Q., Boutilier, J., and Wood, M. J. A., “Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO.”, Molecular therapy : the journal of the American Society of Gene Therapy, vol. 18, no. 10, pp. 1822-9, 2010.

Q. Wang, Yin, H., Camelliti, P., Betts, C., Moulton, H. M., Lee, H., Saleh, A. F., Gait, M. J., and Wood, M. J. A., “In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.”, The journal of gene medicine, vol. 12, no. 4, pp. 354-64, 2010.

N. Jearawiriyapaisarn, Moulton, H. M., Sazani, P., Kole, R., and Willis, M. S., “Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.”, Cardiovascular research, vol. 85, no. 3, pp. 444-53, 2010.

H. M. Moulton and Moulton, J. D., “Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy.”, Biochimica et biophysica acta, vol. 1798, no. 12, pp. 2296-303, 2010.

H. Yin, Moulton, H. M., Betts, C., and Wood, M., “CPP-directed oligonucleotide exon skipping in animal models of Duchenne muscular dystrophy.”, Methods in molecular biology (Clifton, N.J.), vol. 683, pp. 321-38, 2011.

B. W. Neuman, Bederka, L. H., Stein, D. A., Ting, J. P. C., Moulton, H. M., and Buchmeier, M. J., “Development of peptide-conjugated morpholino oligomers as pan-arenavirus inhibitors.”, Antimicrobial agents and chemotherapy, vol. 55, no. 10, pp. 4631-8, 2011.

A. Crisp, Yin, H., Goyenvalle, A., Betts, C., Moulton, H. M., Seow, Y., Babbs, A., Merritt, T., Saleh, A. F., Gait, M. J., Stuckey, D. J., Clarke, K., Davies, K. E., and Wood, M. J. A., “Diaphragm rescue alone prevents heart dysfunction in dystrophic mice.”, Human molecular genetics, vol. 20, no. 3, pp. 413-21, 2011.

H. M. Moulton, “Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers.”, Methods in molecular biology (Clifton, N.J.), vol. 867, pp. 407-14, 2012.

B. Wu, Lu, P., Cloer, C., Shaban, M., Grewal, S., Milazi, S., Shah, S. N., Moulton, H. M., and Lu, Q. Long, “Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.”, The American journal of pathology, vol. 181, no. 2, pp. 392-400, 2012.

H. Yin, Boisguerin, P., Moulton, H. M., Betts, C., Seow, Y., Boutilier, J., Wang, Q., Walsh, A., Lebleu, B., and Wood, M. J. A., “Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency.”, Molecular therapy. Nucleic acids, vol. 2, p. e124, 2013.

H. M. Moulton, “In vivo delivery of morpholino oligos by cell-penetrating peptides.”, Current pharmaceutical design, vol. 19, no. 16, pp. 2963-9, 2013.