Eteplirsen for the treatment of Duchenne muscular dystrophy von Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., Kaye, Edward M.

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Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing von Sazani, Peter, Kole, Ryszard

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Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice von Jearawiriyapaisarn, Natee, Moulton, Hong M, Buckley, Brian, Roberts, Jennifer, Sazani, Peter, Fucharoen, Suthat, Iversen, Patrick L, Kole, Ryszard

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A Small Aptamer with Strong and Specific Recognition of the Triphosphate of ATP von Sazani, Peter L, Larralde, Rosa, Szostak, Jack W

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Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice von Roberts, Jennifer, Palma, Enzo, Sazani, Peter, Ørum, Henrik, Cho, Moo, Kole, Ryszard

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Polymeric Nanogels Produced via Inverse Microemulsion Polymerization as Potential Gene and Antisense Delivery Agents von McAllister, Karen, Sazani, Peter, Adam, Mirielle, Cho, Moo J, Rubinstein, Michael, Samulski, Richard Jude, DeSimone, Joseph M

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Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment von Ke, Qing, Zhao, Zheng-Yan, Griggs, Robert, Wiley, Veronica, Connolly, Anne, Kwon, Jennifer, Qi, Ming, Sheehan, Daniel, Ciafaloni, Emma, Howell, R. Rodney, Furu, Petra, Sazani, Peter, Narayana, Arvind, Gatheridge, Michele

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Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs von Sazani, P, Kang, S H, Maier, M A, Wei, C, Dillman, J, Summerton, J, Manoharan, M, Kole, R

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Peptide-Morpholino Conjugate: A Promising Therapeutic for Duchenne Muscular Dystrophy von Moulton, Hong M., Wu, Bo, Jearawiriyapaisarn, Natee, Sazani, Peter, Lu, Qi Long, Kole, Ryszard

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Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy von Sazani, Peter, Ness, Kirk P. Van, Weller, Doreen L., Poage, Duane W., Palyada, Kiran, Shrewsbury, Stephen B.

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Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice von Sazani, Peter, Ness, Kirk P. Van, Weller, Doreen L., Poage, Duane, Nelson, Keith, Shrewsbury, and Stephen B.

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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy von Echigoya, Yusuke, Nakamura, Akinori, Nagata, Tetsuya, Urasawa, Nobuyuki, Lim, Kenji Rowel Q., Trieu, Nhu, Panesar, Dharminder, Kuraoka, Mutsuki, Moulton, Hong M., Saito, Takashi, Aoki, Yoshitsugu, Iversen, Patrick, Sazani, Peter, Kole, Ryszard, Maruyama, Rika, Partridge, Terry, Takeda, Shin’ichi, Yokota, Toshifumi

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Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies von Heald, Alison E, Iversen, Patrick L, Saoud, Jay B, Sazani, Peter, Charleston, Jay S, Axtelle, Tim, Wong, Michael, Smith, William B, Vutikullird, Apinya, Kaye, Edward

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AVI-7288 for Marburg Virus in Nonhuman Primates and Humans von Heald, Alison E., Charleston, Jay S., Iversen, Patrick L., Warren, Travis K., Saoud, Jay B., Al-Ibrahim, Mohamed, Wells, Jay, Warfield, Kelly L., Swenson, Dana L., Welch, Lisa S., Sazani, Peter, Wong, Michael, Berry, Diane, Kaye, Edward M., Bavari, Sina

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Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers von Jearawiriyapaisarn, Natee, Moulton, Hong M., Sazani, Peter, Kole, Ryszard, Willis, Monte S.

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AVI-7288 for Marburg Virus in Nonhuman Primates and Humans von Heald, Alison E, Charleston, Jay S, Iversen, Patrick L, Warren, Travis K, Saoud, Jay B, Al-Ibrahim, Mohamed, Wells, Jay, Warfield, Kelly L, Swenson, Dana L, Welch, Lisa S, Sazani, Peter, Wong, Michael, Berry, Diane, Kaye, Edward M, Bavari, Sina

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Modification of HER2 pre‐mRNA alternative splicing and its effects on breast cancer cells von Wan, Jing, Sazani, Peter, Kole, Ryszard

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Systemically delivered antisense oligomers upregulate gene expression in mouse tissues von Sazani, Peter, Gemignani, Federica, Kang, Shin-Hong, Maier, Martin A., Manoharan, Muthiah, Persmark, Magnus, Bortner, Donna, Kole, Ryszard

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Safety Pharmacology and Genotoxicity Evaluation of AVI-4658 von Sazani, Peter, Weller, Doreen L., Shrewsbury, Stephen B.

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An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models von Graziewicz, Maria A, Tarrant, Teresa K, Buckley, Brian, Roberts, Jennifer, Fulton, LeShara, Hansen, Henrik, Ørum, Henrik, Kole, Ryszard, Sazani, Peter

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