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Stéphanie Klein

ionis pharmaceuticals inc.

15H-index
74Paper Count
1.2KCitation Count
Published Papers 24
Publication Date
Discovery of long-acting APOC3 siRNA for treating patients with hypertriglyceridemia
err2025-11-11
err0
PREAI
errPrakash, Thazha P.; Mullick, Adam E.; Riney, Stan; Yu, Jinghua; Nikan, Mehran; Quirk, Clare; Crutchfield, Amanda; Damle, Sagar S.; Klein, Stephanie; Swayze, Eric E.
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A comparison of MALAT1 antisense oligonucleotide biodistribution following the intracerebroventricular and lumbar intrathecal routes of administration
err2025-10-13
err0
errOAAI
errMichelle M. Boyd; Curt Mazur; Kaylee Pribnow; Roger L. Zanon; Eric Adams; Jordan DuGal; Stephanie K. Klein; Lisa L. Shafer
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Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile
err2025-08-12
err0
PREAI
errNikan, Mehran; Li, Qingfeng; Tanowitz, Michael; Li, Hongda; Damle, Sagar; Annoual, Marie; Galindo-Murillo, Rodrigo; Low, Audrey; Klein, Stephanie; Quirk, Clare; Vasquez, Guillermo; Wan, W. Brad; Watt, Andrew T.; Migawa, Michael T.; Swayze, Eric E.; Prakash, Thazha P.
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Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles
err2025-04-10
err0
PREAI
errOstergaard, Michael E.; Carrer, Michele; Anderson, Brooke A.; Afetian, Megan; Bakooshli, Mohsen A.; Santos, Jinro A.; Klein, Stephanie K.; Capitanio, Juliana; Freestone, Graeme C.; Tanowitz, Michael; Galindo-Murillo, Rodrigo; Gaus, Hans J.; Dwyer, Chrissa A.; Jackson, Michaela; Jafar-nejad, Paymaan; Rigo, Frank; Seth, Punit P.; Gaynor, Katherine U.; Stanway, Steven J.; Urbonas, Liudvikas; St. Denis, Megan A.; Pellegrino, Simone; Bezerra, Gustavo A.; Rigby, Michael; Gowans, Ellen; Van Rietschoten, Katerine; Beswick, Paul; Skynner, Michael J.; Swayze, Eric E.
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Toxicologic Pathology Forum: Opinion on Interpretive Challenges for Procedure-Related Effects Associated With Direct Central Nervous System Delivery of Oligonucleotides to Rodents, Dogs, and Nonhuman Primates
err2024-01-05
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errOAAI
errBerman-Booty, Lisa D.; Klein, Stephanie K.; Mazur, Curt; Schroeder, Joseph; Korte, Sven; Ludwig, Florian T.; Romeike, Annette; Bolon, Brad; Grieves, Jessica L.
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Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome (vol 27, pg 526, 2021)
err2021-06-01
err0
errOAAI
errPuttaraju, Madaiah; Jackson, Michaela; Klein, Stephanie; Shilo, Asaf; Bennett, C. Frank; Gordon, Leslie; Rigo, Frank; Misteli, Tom
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Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome
err2021-03-11
err43
PREAI
errPuttaraju, Madaiah; Jackson, Michaela; Klein, Stephanie; Shilo, Asaf; Bennett, C. Frank; Gordon, Leslie; Rigo, Frank; Misteli, Tom
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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
err2019-10-07
err129
errOAAI
errRamos, Daniel M.; d'Ydewalle, Constantin; Gabbeta, Vijayalakshmi; Dakka, Amal; Klein, Stephanie K.; Norris, Daniel A.; Matson, John; Taylor, Shannon J.; Zaworski, Phillip G.; Prior, Thomas W.; Snyder, Pamela J.; Valdivia, David; Hatem, Christine L.; Waters, Ian; Gupte, Nikhil; Swoboda, Kathryn J.; Rigo, Frank; Bennett, C. Frank; Naryshkin, Nikolai; Paushkin, Sergey; Crawford, Thomas O.; Sumner, Charlotte J.
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Targeting castration-resistant prostate cancer with androgen receptor antisense oligonucleotide therapy
err2019-09-05
err26
errOAAI
errDe Velasco, Marco A.; Kura, Yurie; Sakai, Kazuko; Hatanaka, Yuji; Davies, Barry R.; Campbell, Hayley; Klein, Stephanie; Kim, Youngsoo; MacLeod, A. Robert; Sugimoto, Koichi; Yoshikawa, Kazuhiro; Nishio, Kazuto; Uemura, Hirotsugu
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Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models
err2019-09-01
err47
errOAAI
errKim, Youngsoo; Jo, Minji; Schmidt, Joanna; Luo, Xiaolin; Prakash, Thazha P.; Zhou, Tianyuan; Klein, Stephanie; Xiao, Xiaokun; Post, Noah; Yin, Zhengfeng; MacLeod, A. Robert
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Discovery and characterization of AZD8701, a high affinity antisense oligonucleotide targeting FOXP3 to relieve immunosuppression in cancer
err2019-07-01
err10
PREAI
errSinclair, Charles; Revenko, Alexey S.; Johnson, R. B.; Peter, Alison; Taylor, Molly A.; Hettrick, Lisa A.; Klein, Stephanie; Solanki, Anisha; Chapman, Melissa; Yates, James; Angell, Helen K.; Watt, Andrew; Gattis, Danielle; Monia, Brett P.; Barry, Simon T.; Lyne, Paul; Edbrooke, Mark; Goldberg, Frederick; Macleod, A. R.
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High-affinity antisense oligonucleotides targeting Foxp3 inhibit immunosuppressive function of regulatory T-cells and produce antitumor effects in syngeneic tumor models
err2018-07-01
err0
PREAI
errSinclair, Charles; Revenko, Alexey S.; Peter, Alison; Johnson, Robert B.; Hettrick, Lisa A.; Taylor, Molly; Staniszewska, Anna; Hughes, Adina; Sandin, Lisa; Klein, Stephanie; Watt, Andrew; Barry, Simon; Monia, Brett T.; Lyne, Paul; Macleod, A. Robert; Edbrooke, Mark
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Dynamic changes and molecular analysis of cell death in the spinal cord of SJL mice infected with the BeAn strain of Theiler's murine encephalomyelitis virus
err2018-02-12
err12
PREAI
errGerhauser, Ingo; Li, Lin; Li, Dandan; Klein, Stephanie; Elmarabet, Suliman Ahmed; Deschl, Ulrich; Kalkuhl, Arno; Baumgaertner, Wolfgang; Ulrich, Reiner; Beineke, Andreas
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High-affinity antisense oligonucleotides targeting Foxp3 inhibit immunosuppressive function of regulatory T cells and produce anti-tumor effects in syngeneic tumor models
err2018-01-01
err0
PREAI
errRevenko, Alexey S.; Sinclair, Charles; Ben Johnson; Hettrick, Lisa; Peter, Alison; Staniszewska, Anna; Hughes, Adina; Sandin, Linda; Taylor, Molly; Klein, Stephanie; Watt, Andy; Monia, Brett; Edbrooke, Mark; MacLeod, A. Robert
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Targeting KRAS-dependent tumors with AZD4785, a high-affinity therapeutic antisense oligonucleotide inhibitor of KRAS
err2017-06-14
err140
PREAI
errRoss, Sarah J.; Revenko, Alexey S.; Hanson, Lyndsey L.; Ellston, Rebecca; Staniszewska, Anna; Whalley, Nicky; Pandey, Sanjay K.; Revill, Mitchell; Rooney, Claire; Buckett, Linda K.; Klein, Stephanie K.; Hudson, Kevin; Monia, Brett P.; Zinda, Michael; Blakey, David C.; Lyne, Paul D.; Macleod, A. Robert
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Continuous, low intensity systemic dosing maximizes the therapeutic margin of Eg5/KSP inhibition in an orthotopic model of urothelial cell carcinoma
err2016-07-15
err0
PREAI
errWilliams, Leigh; Ellston, Rebecca; Trueman, Dawn; Musgrove, Helen; Ruston, Linette; Aquila, Brian; Pease, Elizabeth; Klein, Stephanie; Davies, Barry R.
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Establishment of an orthotopic bladder cancer model to evaluate continuous intravesical delivery of small molecule inhibitors in the nude rat
err2015-08-01
err1
PREAI
errWilliams, Leigh J.; Klein, Stephanie K.; Greenawalt, Danielle; Trueman, Dawn; Musgrove, Helen; Jones, Helen; Ruston, Linette; Davies, Barry R.
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Targeting the Immune System to Cancer
err2012-07-01
err0
errOAAI
errLevitzki, A.; Shir, A.; Berhani, O.; Langut, Y.; Zigler, M.; Ben-Ezri, E.; Pesach, N.; Klein, S.
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Periventricular Demyelination and Axonal Pathology Is Associated with Subependymal Virus Spread in a Murine Model for Multiple Sclerosis
err2012-04-25
err27
PREAI
errKummerfeld, Maren; Seehusen, Frauke; Klein, Stephanie; Ulrich, Reiner; Kreutzer, Robert; Gerhauser, Ingo; Herder, Vanessa; Baumgaertner, Wolfgang; Beineke, Andreas
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EFA6 Facilitates the Assembly of the Tight Junction by Coordinating an Arf6-dependent and -independent Pathway
err2008-10-01
err23
errOAAI
errKlein, Stephanie; Partisani, Mariagrazia; Franco, Michel; Luton, Frederic
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