Search Results - "Kinberger, Garth A"

Single-Stranded siRNAs Activate RNAi in Animals by 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.

“…The therapeutic utility of siRNAs is limited by the requirement for complex formulations to deliver them to tissues. If potent single-stranded RNAs could be…”
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Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor by 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

“…Targeted delivery of antisense oligonucleotides (ASO) to hepatocytes via the asialoglycoprotein receptor (ASGR) has improved the potency of ASO drugs ∼30-fold…”
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Structural Analysis of Human Argonaute‑2 Bound to a Modified siRNA Guide by Schirle, Nicole T, Kinberger, Garth A, Murray, Heather F, Lima, Walt F, Prakash, Thazha P, MacRae, Ian J

“…Incorporation of chemical modifications into small interfering RNAs (siRNAs) increases their metabolic stability and improves their tissue distribution…”
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Comprehensive Structure–Activity Relationship of Triantennary N‑Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes by 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

“…The comprehensive structure–activity relationships of triantennary GalNAc conjugated ASOs for enhancing potency via ASGR mediated delivery to hepatocytes is…”
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Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides by Østergaard, Michael E, Yu, Jinghua, Kinberger, Garth A, Wan, W. Brad, Migawa, Michael T, Vasquez, Guillermo, Schmidt, Karsten, Gaus, Hans J, Murray, Heather M, Low, Audrey, Swayze, Eric E, Prakash, Thazha P, Seth, Punit P

“…Conjugation of triantennary N-acetyl galactosamine (GalNAc) to oligonucleotide therapeutics results in marked improvement in potency for reducing gene targets…”
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Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA by Prakash, Thazha P., Kinberger, Garth A., Murray, Heather M., Chappell, Alfred, Riney, Stan, Graham, Mark J., Lima, Walt F., Swayze, Eric E., Seth, Punit P.

“…[Display omitted] Chemical modifications are essential to improve metabolic stability and pharmacokinetic properties of siRNA to enable their systemic…”
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Dose-dependent reduction of somatic expansions but not Htt aggregates by di-valent siRNA-mediated silencing of MSH3 in HdhQ111 mice by Driscoll, Rachelle, Hampton, Lucas, Abraham, Neeta A., Larigan, J. Douglas, Joseph, Nadine F., Hernandez-Vega, Juan C., Geisler, Sarah, Yang, Fu-Chia, Deninger, Matthew, Tran, David T., Khatri, Natasha, Godinho, Bruno M. D. C., Kinberger, Garth A., Montagna, Daniel R., Hirst, Warren D., Guardado, Catherine L., Glajch, Kelly E., Arnold, H. Moore, Gallant-Behm, Corrie L., Weihofen, Andreas

“…Huntington's disease (HD) is a progressive neurodegenerative disorder caused by CAG trinucleotide repeat expansions in exon 1 of the HTT gene. In addition to…”
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Conjugation of mono and di-GalNAc sugars enhances the potency of antisense oligonucleotides via ASGR mediated delivery to hepatocytes by Kinberger, Garth A., Prakash, Thazha P., Yu, Jinghua, Vasquez, Guillermo, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Murray, Heather M., Gaus, Hans, Swayze, Eric E., Seth, Punit P.

“…[Display omitted] Antisense oligonucleotides (ASOs) conjugated to trivalent GalNAc ligands show 10-fold enhanced potency for suppressing gene targets expressed…”
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S -Acyl-2-Thioethyl: A Convenient Base-Labile Protecting Group for the Synthesis of siRNAs Containing 5'-Vinylphosphonate by Nikan, Mehran, Li, Wenyu, Kinberger, Garth A, Seth, Punit P, Swayze, Eric E, Prakash, Thazha P

“…We recently reported that ( )-5'-vinylphosphonate (5'-VP) is a metabolically-stable phosphate mimic for siRNA and demonstrated that 5'-VP improves the potency…”
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Solid-phase synthesis of 5′-triantennary N-acetylgalactosamine conjugated antisense oligonucleotides using phosphoramidite chemistry by Prakash, Thazha P., Brad Wan, W., Low, Audrey, Yu, Jinghua, Chappell, Alfred E., Gaus, Hans, Kinberger, Garth A., Østergaard, Michael E., Migawa, Michael T., Swayze, Eric E., Seth, Punit P.

“…[Display omitted] A convenient solid-phase synthetic method was developed for assembling a triantennary N-acetylgalactosamine (GalNAc) cluster on the 5′-end of…”
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A convenient synthesis of 5′-triantennary N-acetyl-galactosamine clusters based on nitromethanetrispropionic acid by Migawa, Michael T., Prakash, Thazha P., Vasquez, Guillermo, Wan, W. Brad, Yu, Jinghua, Kinberger, Garth A., Østergaard, Michael E., Swayze, Eric E., Seth, Punit P.

“…[Display omitted] A convenient method for the synthesis of several triantennary GalNAc clusters based on a nitromethanetrispropionic acid core was developed…”
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Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes by Tanowitz, Michael, Hettrick, Lisa, Revenko, Alexey, Kinberger, Garth A, Prakash, Thazha P, Seth, Punit P

“…Antisense oligonucleotide (ASO) therapeutics show tremendous promise for the treatment of previously intractable human diseases but to exert their effects on…”
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Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates by Østergaard, Michael E, Jackson, Michaela, Low, Audrey, E. Chappell, Alfred, G. Lee, Richard, Peralta, Rachel Q, Yu, Jinghua, Kinberger, Garth A, Dan, Amy, Carty, Rick, Tanowitz, Michael, Anderson, Patrick, Kim, Tae-Won, Fradkin, Linda, Mullick, Adam E, Murray, Sue, Rigo, Frank, Prakash, Thazha P, Bennett, C Frank, Swayze, Eric E, Gaus, Hans J, Seth, Punit P

“…Abstract We determined the effect of attaching palmitate, tocopherol or cholesterol to PS ASOs and their effects on plasma protein binding and on enhancing ASO…”
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Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2'-modifications and enhances antisense activity by Liang, Xue-Hai, Shen, Wen, Sun, Hong, Kinberger, Garth A, Prakash, Thazha P, Nichols, Joshua G, Crooke, Stanley T

“…RNase H1-dependent antisense oligonucleotides (ASOs) are chemically modified to enhance pharmacological properties. Major modifications include…”
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Identification of metabolically stable 5'-phosphate analogs that support single-stranded siRNA activity by Prakash, Thazha P, Lima, Walt F, Murray, Heather M, Li, Wenyu, Kinberger, Garth A, Chappell, Alfred E, Gaus, Hans, Seth, Punit P, Bhat, Balkrishen, Crooke, Stanley T, Swayze, Eric E

“…The ss-siRNA activity in vivo requires a metabolically stable 5'-phosphate analog. In this report we used crystal structure of the 5'-phosphate binding pocket…”
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Synthesis and Biophysical Evaluation of 2′,4′-Constrained 2′O-Methoxyethyl and 2′,4′-Constrained 2′O-Ethyl Nucleic Acid Analogues by Seth, Punit P, Vasquez, Guillermo, Allerson, Charles A, Berdeja, Andres, Gaus, Hans, Kinberger, Garth A, Prakash, Thazha P, Migawa, Michael T, Bhat, Balkrishen, Swayze, Eric E

“…We have recently shown that combining the structural elements of 2′O-methoxyethyl (MOE) and locked nucleic acid (LNA) nucleosides yielded a series of…”
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Evaluation of the effect of 2′-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice by Prakash, Thazha P., Yu, Jinghua, Kinberger, Garth A., Low, Audrey, Jackson, Michaela, Rigo, Frank, Swayze, Eric E., Seth, Punit P.

“…[Display omitted] •GalNAc conjugation improved potency of 2′-O-methyl Gapmer ASO 10-fold.•Potency of FHNA Gapmer ASO improved 14-fold by GalNAc…”
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Identification of metabolically stable 5΄-phosphate analogs that support single-stranded siRNA activity by Prakash, Thazha P, Lima, Walt F, Murray, Heather M, Li, Wenyu, Kinberger, Garth A, Chappell, Alfred E, Gaus, Hans, Seth, Punit P, Bhat, Balkrishen, Crooke, Stanley T, Swayze, Eric E

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The design, synthesis, and characterization of a PAMAM-based triple helical collagen mimetic dendrimer by Kinberger, Garth A., Taulane, Joseph P., Goodman, Murray

“…The synthesis and characterization of a collagen mimetic dendrimer composed of the Gly-Pro-Nleu sequence is described. The dendrimer is built on a ‘first…”
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Collagen Mimetic Dendrimers by Kinberger, Garth A, Cai, Weibo, Goodman, Murray

“…The synthesis of single-chain, scaffold (TRIS)- and dendrimer-assembled collagen mimetics (both Gly-Pro-Nleu and Gly-Nleu-Pro sequences) is reported. From the…”
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