Selective tissue targeting of synthetic nucleic acid drugs von Seth, Punit P, Tanowitz, Michael, Bennett, C Frank

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Overcoming the challenges of tissue delivery for oligonucleotide therapeutics von Gökirmak, Tufan, Nikan, Mehran, Wiechmann, Svenja, Prakash, Thazha P., Tanowitz, Michael, Seth, Punit P.

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Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides von Anderson, Brooke A, Freestone, Graeme C, Low, Audrey, De-Hoyos, Cheryl L, III, William J Drury, Østergaard, Michael E, Migawa, Michael T, Fazio, Michael, Wan, W Brad, Berdeja, Andres, Scandalis, Eli, Burel, Sebastien A, Vickers, Timothy A, Crooke, Stanley T, Swayze, Eric E, Liang, Xuehai, Seth, Punit P

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Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index von Shen, Wen, De Hoyos, Cheryl L., Migawa, Michael T., Vickers, Timothy A., Sun, Hong, Low, Audrey, Bell, Thomas A., Rahdar, Meghdad, Mukhopadhyay, Swagatam, Hart, Christopher E., Bell, Melanie, Riney, Stan, Murray, Susan F., Greenlee, Sarah, Crooke, Rosanne M., Liang, Xue-hai, Seth, Punit P., Crooke, Stanley T.

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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice von Prakash, Thazha P, Graham, Mark J, Yu, Jinghua, Carty, Rick, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Zhao, Chenguang, Aghajan, Mariam, Murray, Heather F, Riney, Stan, Booten, Sheri L, Murray, Susan F, Gaus, Hans, Crosby, Jeff, Lima, Walt F, Guo, Shuling, Monia, Brett P, Swayze, Eric E, Seth, Punit P

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Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle von Prakash, Thazha P, Mullick, Adam E, Lee, Richard G, Yu, Jinghua, Yeh, Steve T, Low, Audrey, Chappell, Alfred E, Østergaard, Michael E, Murray, Sue, Gaus, Hans J, Swayze, Eric E, Seth, Punit P

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Single-Stranded siRNAs Activate RNAi in Animals von Lima, Walt F., Prakash, Thazha P., Murray, Heather M., Kinberger, Garth A., Li, Wenyu, Chappell, Alfred E., Li, Cheryl S., Murray, Susan F., Gaus, Hans, Seth, Punit P., Swayze, Eric E., Crooke, Stanley T.

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Origins of the Increased Affinity of Phosphorothioate-Modified Therapeutic Nucleic Acids for Proteins von Hyjek-Składanowska, Malwina, Vickers, Timothy A, Napiórkowska, Agnieszka, Anderson, Brooke A, Tanowitz, Michael, Crooke, Stanley T, Liang, Xue-hai, Seth, Punit P, Nowotny, Marcin

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Site-specific incorporation of 5′-methyl DNA enhances the therapeutic profile of gapmer ASOs von Vasquez, Guillermo, Freestone, Graeme C, Wan, W Brad, Low, Audrey, De Hoyos, Cheryl Li, Yu, Jinghua, Prakash, Thazha P, Ǿstergaard, Michael E, Liang, Xue-hai, Crooke, Stanley T, Swayze, Eric E, Migawa, Michael T, Seth, Punit P

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Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver von Miller, Colton M, Donner, Aaron J, Blank, Emma E, Egger, Andrew W, Kellar, Brianna M, Østergaard, Michael E, Seth, Punit P, Harris, Edward N

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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides von Ostergaard, Michael E., De Hoyos, Cheryl L., Wan, W. Brad, Shen, Wen, Low, Audrey, Berdeja, Andres, Vasquez, Guillermo, Murray, Susan, Migawa, Michael T., Liang, Xue-hai, Swayze, Eric E., Crooke, Stanley T., Seth, Punit P.

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Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice von Chappell, Alfred E., Gaus, Hans J., Berdeja, Andres, Gupta, Ruchi, Jo, Minji, Prakash, Thazha P., Oestergaard, Michael, Swayze, Eric E., Seth, Punit P.

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Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts von Burel, Sebastien A, Hart, Christopher E, Cauntay, Patrick, Hsiao, Jill, Machemer, Todd, Katz, Melanie, Watt, Andy, Bui, Huynh-Hoa, Younis, Husam, Sabripour, Mahyar, Freier, Susan M, Hung, Gene, Dan, Amy, Prakash, T P, Seth, Punit P, Swayze, Eric E, Bennett, C Frank, Crooke, Stanley T, Henry, Scott P

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Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor von Schmidt, Karsten, Prakash, Thazha P, Donner, Aaron J, Kinberger, Garth A, Gaus, Hans J, Low, Audrey, Østergaard, Michael E, Bell, Melanie, Swayze, Eric E, Seth, Punit P

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Glucagon Like Peptide 1 Receptor Agonists for Targeted Delivery of Antisense Oligonucleotides to Pancreatic Beta Cell von Knerr, Laurent, Prakash, Thazha P, Lee, Richard, Drury III, William J, Nikan, Mehran, Fu, Wuxia, Pirie, Elaine, Maria, Leonardo De, Valeur, Eric, Hayen, Ahlke, Ölwegård-Halvarsson, Maria, Broddefalk, Johan, Ämmälä, Carina, Østergaard, Michael E, Meuller, Johan, Sundström, Linda, Andersson, Patrik, Janzén, David, Jansson-Löfmark, Rasmus, Seth, Punit P, Andersson, Shalini

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DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing von Nishina, Kazutaka, Piao, Wenying, Yoshida-Tanaka, Kie, Sujino, Yumiko, Nishina, Tomoko, Yamamoto, Tsuyoshi, Nitta, Keiko, Yoshioka, Kotaro, Kuwahara, Hiroya, Yasuhara, Hidenori, Baba, Takeshi, Ono, Fumiko, Miyata, Kanjiro, Miyake, Koichi, Seth, Punit P., Low, Audrey, Yoshida, Masayuki, Bennett, C. Frank, Kataoka, Kazunori, Mizusawa, Hidehiro, Obika, Satoshi, Yokota, Takanori

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Comprehensive Structure–Activity Relationship of Triantennary N‑Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes von Prakash, Thazha P, Yu, Jinghua, Migawa, Michael T, Kinberger, Garth A, Wan, W. Brad, Østergaard, Michael E, Carty, Recaldo L, Vasquez, Guillermo, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Aghajan, Mariam, Crosby, Jeff, Murray, Heather M, Booten, Sheri L, Hsiao, Jill, Soriano, Armand, Machemer, Todd, Cauntay, Patrick, Burel, Sebastien A, Murray, Susan F, Gaus, Hans, Graham, Mark J, Swayze, Eric E, Seth, Punit P

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Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics von Finicle, Brendan T, Eckenstein, Kazumi H, Revenko, Alexey S, Anderson, Brooke A, Wan, W Brad, McCracken, Alison N, Gil, Daniel, Fruman, David A, Hanessian, Stephen, Seth, Punit P, Edinger, Aimee L

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Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages von Wan, W Brad, Migawa, Michael T, Vasquez, Guillermo, Murray, Heather M, Nichols, Josh G, Gaus, Hans, Berdeja, Andres, Lee, Sam, Hart, Christopher E, Lima, Walt F, Swayze, Eric E, Seth, Punit P

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The Medicinal Chemistry of Therapeutic Oligonucleotides von Wan, W. Brad, Seth, Punit P

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