Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
Rajeev, Kallanthottathil G., Nair, Jayaprakash K., Jayaraman, Muthusamy, Charisse, Klaus, Taneja, Nate, O'Shea, Jonathan, Willoughby, Jennifer L. S., Yucius, Kristina, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Milstein, Stuart, Liebow, Abigail, Querbes, William, Borodovsky, Anna, Fitzgerald, Kevin, Maier, Martin A., Manoharan, Muthiah
Published in Chembiochem : a European journal of chemical biology (13.04.2015)
Published in Chembiochem : a European journal of chemical biology (13.04.2015)
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Journal Article
Multivalent N‑Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Nair, Jayaprakash K., Willoughby, Jennifer L. S., Chan, Amy, Charisse, Klaus, Alam, Md. Rowshon, Wang, Qianfan, Hoekstra, Menno, Kandasamy, Pachamuthu, Kel’in, Alexander V., Milstein, Stuart, Taneja, Nate, O’Shea, Jonathan, Shaikh, Sarfraz, Zhang, Ligang, van der Sluis, Ronald J., Jung, Michael E., Akinc, Akin, Hutabarat, Renta, Kuchimanchi, Satya, Fitzgerald, Kevin, Zimmermann, Tracy, van Berkel, Theo J. C., Maier, Martin A., Rajeev, Kallanthottathil G., Manoharan, Muthiah
Published in Journal of the American Chemical Society (10.12.2014)
Published in Journal of the American Chemical Society (10.12.2014)
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Journal Article
5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates
Parmar, Rubina, Willoughby, Jennifer L. S., Liu, Jingxuan, Foster, Donald J., Brigham, Benjamin, Theile, Christopher S., Charisse, Klaus, Akinc, Akin, Guidry, Erin, Pei, Yi, Strapps, Walter, Cancilla, Mark, Stanton, Matthew G., Rajeev, Kallanthottathil G., Sepp-Lorenzino, Laura, Manoharan, Muthiah, Meyers, Rachel, Maier, Martin A., Jadhav, Vasant
Published in Chembiochem : a European journal of chemical biology (02.06.2016)
Published in Chembiochem : a European journal of chemical biology (02.06.2016)
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Journal Article
siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes
Matsuda, Shigeo, Keiser, Kristofer, Nair, Jayaprakash K, Charisse, Klaus, Manoharan, Rajar M, Kretschmer, Philip, Peng, Chang G, V. Kel’in, Alexander, Kandasamy, Pachamuthu, Willoughby, Jennifer L.S, Liebow, Abigail, Querbes, William, Yucius, Kristina, Nguyen, Tuyen, Milstein, Stuart, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah
Published in ACS chemical biology (15.05.2015)
Published in ACS chemical biology (15.05.2015)
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Journal Article
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates
Nair, Jayaprakash K, Attarwala, Husain, Sehgal, Alfica, Wang, Qianfan, Aluri, Krishna, Zhang, Xuemei, Gao, Minggeng, Liu, Ju, Indrakanti, Ramesh, Schofield, Sally, Kretschmer, Philip, Brown, Christopher R, Gupta, Swati, Willoughby, Jennifer L S, Boshar, Julie A, Jadhav, Vasant, Charisse, Klaus, Zimmermann, Tracy, Fitzgerald, Kevin, Manoharan, Muthiah, Rajeev, Kallanthottathil G, Akinc, Akin, Hutabarat, Renta, Maier, Martin A
Published in Nucleic acids research (02.11.2017)
Published in Nucleic acids research (02.11.2017)
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Journal Article
Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression
Willoughby, Jennifer L.S., Chan, Amy, Sehgal, Alfica, Butler, James S., Nair, Jayaprakash K., Racie, Tim, Shulga-Morskaya, Svetlana, Nguyen, Tuyen, Qian, Kun, Yucius, Kristina, Charisse, Klaus, van Berkel, Theo J.C., Manoharan, Muthiah, Rajeev, Kallanthottathil G., Maier, Martin A., Jadhav, Vasant, Zimmermann, Tracy S.
Published in Molecular therapy (03.01.2018)
Published in Molecular therapy (03.01.2018)
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Journal Article
Factor quinolinone inhibitors disrupt spindles and multiple LSF (TFCP2)-protein interactions in mitosis, including with microtubule-associated proteins
Yunes, Sarah A, Willoughby, Jennifer L S, Kwan, Julian H, Biagi, Jessica M, Pokharel, Niranjana, Chin, Hang Gyeong, York, Emily A, Su, Kuan-Chung, George, Kelly, Shah, Jagesh V, Emili, Andrew, Schaus, Scott E, Hansen, Ulla
Published in PloS one (15.06.2022)
Published in PloS one (15.06.2022)
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Journal Article
Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo
Jahns, Hartmut, Taneja, Nate, Willoughby, Jennifer L S, Akabane-Nakata, Masaaki, Brown, Christopher R, Nguyen, Tuyen, Bisbe, Anna, Matsuda, Shigeo, Hettinger, Matt, Manoharan, Rajar M, Rajeev, Kallanthottathil G, Maier, Martin A, Zlatev, Ivan, Charisse, Klaus, Egli, Martin, Manoharan, Muthiah
Published in Nucleic acids research (22.02.2022)
Published in Nucleic acids research (22.02.2022)
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Journal Article
Facile Synthesis, Geometry, and 2′-Substituent-Dependent in Vivo Activity of 5′‑(E)- and 5′‑(Z)‑Vinylphosphonate-Modified siRNA Conjugates
Parmar, Rubina Giare, Brown, Christopher R, Matsuda, Shigeo, Willoughby, Jennifer L. S, Theile, Christopher S, Charissé, Klaus, Foster, Donald J, Zlatev, Ivan, Jadhav, Vasant, Maier, Martin A, Egli, Martin, Manoharan, Muthiah, Rajeev, Kallanthottathil G
Published in Journal of medicinal chemistry (08.02.2018)
Published in Journal of medicinal chemistry (08.02.2018)
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Journal Article
5′-Morpholino modification of the sense strand of an siRNA makes it a more effective passenger
Kumar, Pawan, Parmar, Rubina G, Brown, Christopher R, Willoughby, Jennifer L. S, Foster, Donald J, Babu, I. Ramesh, Schofield, Sally, Jadhav, Vasant, Charisse, Klaus, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Maier, Martin A, Egli, Martin, Manoharan, Muthiah
Published in Chemical communications (Cambridge, England) (25.04.2019)
Published in Chemical communications (Cambridge, England) (25.04.2019)
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Journal Article
Divergent effects of glucose and fructose on hepatic lipogenesis and insulin signaling
Softic, Samir, Gupta, Manoj K, Wang, Guo-Xiao, Fujisaka, Shiho, O'Neill, Brian T, Rao, Tata Nageswara, Willoughby, Jennifer, Harbison, Carole, Fitzgerald, Kevin, Ilkayeva, Olga, Newgard, Christopher B, Cohen, David E, Kahn, C Ronald
Published in The Journal of clinical investigation (01.11.2017)
Published in The Journal of clinical investigation (01.11.2017)
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Journal Article
Targeting the oncogene LSF with either the small molecule inhibitor FQI1 or siRNA causes mitotic delays with unaligned chromosomes, resulting in cell death or senescence
Willoughby, Jennifer L S, George, Kelly, Roberto, Mark P, Chin, Hang Gyeong, Stoiber, Patrick, Shin, Hyunjin, Pedamallu, Chandra Sekhar, Schaus, Scott E, Fitzgerald, Kevin, Shah, Jagesh, Hansen, Ulla
Published in BMC cancer (15.06.2020)
Published in BMC cancer (15.06.2020)
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Journal Article
Metabolically Stable Anomeric Linkages Containing GalNAc–siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways
Kandasamy, Pachamuthu, Mori, Shohei, Matsuda, Shigeo, Erande, Namrata, Datta, Dhrubajyoti, Willoughby, Jennifer L. S., Taneja, Nate, O’Shea, Jonathan, Bisbe, Anna, Manoharan, Rajar M., Yucius, Kristina, Nguyen, Tuyen, Indrakanti, Ramesh, Gupta, Swati, Gilbert, Jason A., Racie, Tim, Chan, Amy, Liu, Ju, Hutabarat, Renta, Nair, Jayaprakash K., Charisse, Klaus, Maier, Martin A., Rajeev, Kallanthottathil G., Egli, Martin, Manoharan, Muthiah
Published in Journal of medicinal chemistry (23.02.2023)
Published in Journal of medicinal chemistry (23.02.2023)
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Journal Article
Dietary Sugars Alter Hepatic Fatty Acid Oxidation via Transcriptional and Post-translational Modifications of Mitochondrial Proteins
Softic, Samir, Meyer, Jesse G, Wang, Guo-Xiao, Gupta, Manoj K, Batista, Thiago M, Lauritzen, Hans P M M, Fujisaka, Shiho, Serra, Dolors, Herrero, Laura, Willoughby, Jennifer, Fitzgerald, Kevin, Ilkayeva, Olga, Newgard, Christopher B, Gibson, Bradford W, Schilling, Birgit, Cohen, David E, Kahn, C Ronald
Published in Cell metabolism (01.10.2019)
Published in Cell metabolism (01.10.2019)
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Journal Article
siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2
Elkayam, Elad, Parmar, Rubina, Brown, Christopher R, Willoughby, Jennifer L, Theile, Christopher S, Manoharan, Muthiah, Joshua-Tor, Leemor
Published in Nucleic acids research (07.04.2017)
Published in Nucleic acids research (07.04.2017)
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Journal Article
Fructose induced KHK-C can increase ER stress independent of its effect on lipogenesis to drive liver disease in diet-induced and genetic models of NAFLD
Park, Se-Hyung, Helsley, Robert N., Fadhul, Taghreed, Willoughby, Jennifer L.S., Noetzli, Leila, Tu, Ho-Chou, Solheim, Marie H., Fujisaka, Shiho, Pan, Hui, Dreyfuss, Jonathan M., Bons, Joanna, Rose, Jacob, King, Christina D., Schilling, Birgit, Lusis, Aldons J., Pan, Calvin, Gupta, Manoj, Kulkarni, Rohit N., Fitzgerald, Kevin, Kern, Philip A., Divanovic, Senad, Kahn, C. Ronald, Softic, Samir
Published in Metabolism, clinical and experimental (01.08.2023)
Published in Metabolism, clinical and experimental (01.08.2023)
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Journal Article
Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis
Butler, James S., Chan, Amy, Costelha, Susete, Fishman, Shannon, Willoughby, Jennifer L. S., Borland, Todd D., Milstein, Stuart, Foster, Donald J., Gonçalves, Paula, Chen, Qingmin, Qin, June, Bettencourt, Brian R., Sah, Dinah W., Alvarez, Rene, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Fitzgerald, Kevin, Meyers, Rachel E., Nochur, Saraswathy V., Saraiva, Maria J., Zimmermann, Tracy S.
Published in Amyloid (01.06.2016)
Published in Amyloid (01.06.2016)
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Journal Article
Role of a “Magic” Methyl: 2′-Deoxy-2′-α-F-2′-β‑C‑methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5′-Phosphate Mimics and Mitigation of Off-Target Effects
Guenther, Dale C., Mori, Shohei, Matsuda, Shigeo, Gilbert, Jason A., Willoughby, Jennifer L. S., Hyde, Sarah, Bisbe, Anna, Jiang, Yongfeng, Agarwal, Saket, Madaoui, Mimouna, Janas, Maja M., Charisse, Klaus, Maier, Martin A., Egli, Martin, Manoharan, Muthiah
Published in Journal of the American Chemical Society (17.08.2022)
Published in Journal of the American Chemical Society (17.08.2022)
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Journal Article
siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2
Elkayam, Elad, Parmar, Rubina, Brown, Christopher R, Willoughby, Jennifer L, Theile, Christopher S, Manoharan, Muthiah, Joshua-Tor, Leemor
Published in Nucleic acids research (05.05.2017)
Published in Nucleic acids research (05.05.2017)
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Journal Article