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102 results for Oligonucleotides,+Antisense+-+pharmacokinetics

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ArticlesPeer ReviewedFull Text
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Author / Creator:Miller, Timothy ; Cudkowicz, Merit ; Shaw, Pamela J ; Andersen, Peter M ; Atassi, Nazem ; Bucelli, Robert C ; Genge, Angela ; Glass, Jonathan ; Ladha, Shafeeq ; Ludolph, Albert L ; Maragakis, Nicholas J ; McDermott, Christopher J ; Pestronk, Alan ; Ravits, John ; Salachas, François ; Trudell, Randall ; Van Damme, Philip ; Zinman, Lorne ; Bennett, C. Frank ; Lane, Roger ; Sandrock, Alfred ; Runz, Heiko ; Graham, Danielle ; Houshyar, Hani ; McCampbell, Alexander ; Nestorov, Ivan ; Chang, Ih ; McNeill, Manjit ; Fanning, Laura ; Fradette, Stephanie ; Ferguson, Toby A
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2020-07, Vol.383 (2), p.109-119
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Cellular uptake and trafficking of antisense oligonucleotides
Author / Creator:Crooke, Stanley T ; Wang, Shiyu ; Vickers, Timothy A ; Shen, Wen ; Liang, Xue-hai
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2017-03, Vol.35 (3), p.230-237
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Preclinical development of a miR-132 inhibitor for heart failure treatment
Author / Creator:Foinquinos, Ariana ; Batkai, Sandor ; Genschel, Celina ; Viereck, Janika ; Rump, Steffen ; Gyöngyösi, Mariann ; Traxler, Denise ; Riesenhuber, Martin ; Spannbauer, Andreas ; Lukovic, Dominika ; Weber, Natalie ; Zlabinger, Katrin ; Hašimbegović, Ena ; Winkler, Johannes ; Fiedler, Jan ; Dangwal, Seema ; Fischer, Martin ; de la Roche, Jeanne ; Wojciechowski, Daniel ; Kraft, Theresia ; Garamvölgyi, Rita ; Neitzel, Sonja ; Chatterjee, Shambhabi ; Yin, Xiaoke ; Bär, Christian ; Mayr, Manuel ; Xiao, Ke ; Thum, Thomas
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2020-01, Vol.11 (1), p.633-10, Article 633
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Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study
Author / Creator:Tsimikas, Sotirios, Prof ; Viney, Nicholas J, BSc ; Hughes, Steven G, MBBS ; Singleton, Walter, MD ; Graham, Mark J, MS ; Baker, Brenda F, PhD ; Burkey, Jennifer L, PhD ; Yang, Qingqing, MS ; Marcovina, Santica M, Prof ; Geary, Richard S, PhD ; Crooke, Rosanne M, PhD ; Witztum, Joseph L, Prof
Publisher:England: Elsevier Ltd
Journal title:The Lancet (British edition), 2015-10, Vol.386 (10002), p.1472-1483
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Nusinersen: First Global Approval
Author / Creator:Hoy, Sheridan M.
Publisher:Cham: Springer International Publishing
Journal title:Drugs (New York, N.Y.), 2017-03, Vol.77 (4), p.473-479
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Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
Author / Creator:Iwamoto, Naoki ; Butler, David C D ; Svrzikapa, Nenad ; Mohapatra, Susovan ; Zlatev, Ivan ; Sah, Dinah W Y ; Meena ; Standley, Stephany M ; Lu, Genliang ; Apponi, Luciano H ; Frank-Kamenetsky, Maria ; Zhang, Jason Jingxin ; Vargeese, Chandra ; Verdine, Gregory L
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2017-09, Vol.35 (9), p.845-851
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Selective tissue targeting of synthetic nucleic acid drugs
Author / Creator:Seth, Punit P ; Tanowitz, Michael ; Bennett, C Frank
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2019-03, Vol.129 (3), p.915-925
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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controll...
Author / Creator:van den Berg, Leonard H ; Rothstein, Jeffrey D ; Shaw, Pamela J ; Babu, Suma ; Benatar, Michael ; Bucelli, Robert C ; Genge, Angela ; Glass, Jonathan D ; Hardiman, Orla ; Libri, Vincenzo ; Mobach, Theodore ; Oskarsson, Björn ; Pattee, Gary L ; Ravits, John ; Shaw, Christopher E ; Weber, Markus ; Zinman, Lorne ; Jafar-nejad, Paymaan ; Rigo, Frank ; Lin, Luan ; Ferguson, Toby A ; Gotter, Anthony L ; Graham, Danielle ; Monine, Michael ; Inra, Jennifer ; Sinks, Susie ; Eraly, Satish ; Garafalo, Steve ; Fradette, Stephanie
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2024-09, Vol.23 (9), p.901-912
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Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency
Author / Creator:Wood, Mattew J. A. ; Altman, Sidney
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2020-12, Vol.117 (51), p.32370-32379
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Delivering Antisense Oligonucleotides across the Blood‐Brain Barrier by Tumor Cell‐Derived Small Apoptotic Bodies
Author / Creator:Wang, Yulian ; Pang, Jiayun ; Wang, Qingyun ; Yan, Luocheng ; Wang, Lintao ; Xing, Zhen ; Wang, Chunming ; Zhang, Junfeng ; Dong, Lei
Publisher:Germany: John Wiley & Sons, Inc
Journal title:Advanced science, 2021-07, Vol.8 (13), p.2004929-n/a
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Image-based detection and targeting of therapy resistance in pancreatic adenocarcinoma
Author / Creator:Fox, Raymond G. ; Lytle, Nikki K. ; Jaquish, Dawn V. ; Park, Frederick D. ; Ito, Takahiro ; Bajaj, Jeevisha ; Koechlein, Claire S. ; Zimdahl, Bryan ; Yano, Masato ; Kopp, Janel L. ; Kritzik, Marcie ; Sicklick, Jason K. ; Sander, Maike ; Grandgenett, Paul M. ; Hollingsworth, Michael A. ; Shibata, Shinsuke ; Pizzo, Donald ; Valasek, Mark A. ; Sasik, Roman ; Scadeng, Miriam ; Okano, Hideyuki ; Kim, Youngsoo ; MacLeod, A. Robert ; Lowy, Andrew M. ; Reya, Tannishtha
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2016-06, Vol.534 (7607), p.407-411
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Improving siRNA Delivery In Vivo Through Lipid Conjugation
Author / Creator:Osborn, Maire F. ; Khvorova, Anastasia
Publisher:United States: Mary Ann Liebert, Inc
Journal title:Nucleic acid therapeutics, 2018-06, Vol.28 (3), p.128-136
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A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration
Author / Creator:Monine, Michael ; Norris, Daniel ; Wang, Yanfeng ; Nestorov, Ivan
Publisher:New York: Springer US
Journal title:Journal of pharmacokinetics and pharmacodynamics, 2021-10, Vol.48 (5), p.639-654
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PK/PD modeling of FXI antisense oligonucleotides to bridge the dose‐FXI activity relation from healthy volunteers to end‐stage renal disease patients
Author / Creator:Willmann, Stefan ; Marostica, Eleonora ; Snelder, Nelleke ; Solms, Alexander ; Jensen, Markus ; Lobmeyer, Maximilian ; Lensing, Anthonie W. A. ; Bethune, Claudette ; Morgan, Erin ; Yu, Rosie Z. ; Wang, Yanfeng ; Jung, Shiangtung W. ; Geary, Richard ; Bhanot, Sanjay
Publisher:United States: John Wiley & Sons, Inc
Journal title:CPT: pharmacometrics and systems pharmacology, 2021-08, Vol.10 (8), p.890-901
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Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model
Author / Creator:MacCannell, Drew ; Berger, Zdenek ; Kirschner, Janbernd ; Mercuri, Eugenio ; Farrar, Michelle A. ; Iannaccone, Susan T. ; Kuntz, Nancy L. ; Finkel, Richard S. ; Valente, Marta ; Muntoni, Francesco
Publisher:Cham: Springer International Publishing
Journal title:CNS drugs, 2022-02, Vol.36 (2), p.181-190
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Antisense therapy for cancer
Author / Creator:Gleave, Martin E. ; Monia, Brett P.
Publisher:London: Nature Publishing Group UK
Journal title:Nature reviews. Cancer, 2005-06, Vol.5 (6), p.468-479
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Fatty Acid-Modified Gapmer Antisense Oligonucleotide and Serum Albumin Constructs for Pharmacokinetic Modulation
Author / Creator:Hvam, Michael Lykke ; Cai, Yunpeng ; Dagnæs-Hansen, Frederik ; Nielsen, Jesper Sejrup ; Wengel, Jesper ; Kjems, Jørgen ; Howard, Kenneth A.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2017-07, Vol.25 (7), p.1710-1717
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Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys
Author / Creator:Yu, Rosie Z. ; Gunawan, Rudy ; Post, Noah ; Zanardi, Thomas ; Hall, Shannon ; Burkey, Jennifer ; Kim, Tae-Won ; Graham, Mark J. ; Prakash, Thazha P. ; Seth, Punit P. ; Swayze, Eric E. ; Geary, Richard S. ; Henry, Scott P. ; Wang, Yanfeng
Publisher:United States: Mary Ann Liebert, Inc
Journal title:Nucleic acid therapeutics, 2016-12, Vol.26 (6), p.372-380
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First-in-human Phase I study of EZN-4176, a locked nucleic acid antisense oligonucleotide to exon 4 of the androgen receptor mRNA in patients with castration-resistant prostate cancer
Author / Creator:Bianchini, D ; Omlin, A ; Pezaro, C ; Lorente, D ; Ferraldeschi, R ; Mukherji, D ; Crespo, M ; Figueiredo, I ; Miranda, S ; Riisnaes, R ; Zivi, A ; Buchbinder, A ; Rathkopf, D E ; Attard, G ; Scher, H I ; de Bono, J ; Danila, D C
Publisher:London: Nature Publishing Group UK
Journal title:British journal of cancer, 2013-11, Vol.109 (10), p.2579-2586
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Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach
Author / Creator:Marchesi, Elena ; Bovolenta, Matteo ; Preti, Lorenzo ; Capobianco, Massimo L ; Mamchaoui, Kamel ; Bertoldo, Monica ; Perrone, Daniela
Publisher:Switzerland: MDPI AG
Journal title:Molecules (Basel, Switzerland), 2021-12, Vol.26 (24), p.7662
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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

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Cellular uptake and trafficking of antisense oligonucleotides

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Preclinical development of a miR-132 inhibitor for heart failure treatment

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Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study

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Nusinersen: First Global Approval

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Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides

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Selective tissue targeting of synthetic nucleic acid drugs

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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controll...

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Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency

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Delivering Antisense Oligonucleotides across the Blood‐Brain Barrier by Tumor Cell‐Derived Small Apoptotic Bodies

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Image-based detection and targeting of therapy resistance in pancreatic adenocarcinoma

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Improving siRNA Delivery In Vivo Through Lipid Conjugation

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A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration

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PK/PD modeling of FXI antisense oligonucleotides to bridge the dose‐FXI activity relation from healthy volunteers to end‐stage renal disease patients

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Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model

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Antisense therapy for cancer

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Fatty Acid-Modified Gapmer Antisense Oligonucleotide and Serum Albumin Constructs for Pharmacokinetic Modulation

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Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys

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First-in-human Phase I study of EZN-4176, a locked nucleic acid antisense oligonucleotide to exon 4 of the androgen receptor mRNA in patients with castration-resistant prostate cancer

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Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach

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