Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates
Foster, Donald J., Brown, Christopher R., Shaikh, Sarfraz, Trapp, Casey, Schlegel, Mark K., Qian, Kun, Sehgal, Alfica, Rajeev, Kallanthottathil G., Jadhav, Vasant, Manoharan, Muthiah, Kuchimanchi, Satya, Maier, Martin A., Milstein, Stuart
Published in Molecular therapy (07.03.2018)
Published in Molecular therapy (07.03.2018)
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Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity
Janas, Maja M., Schlegel, Mark K., Harbison, Carole E., Yilmaz, Vedat O., Jiang, Yongfeng, Parmar, Rubina, Zlatev, Ivan, Castoreno, Adam, Xu, Huilei, Shulga-Morskaya, Svetlana, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Keirstead, Natalie D., Maier, Martin A., Jadhav, Vasant
Published in Nature communications (19.02.2018)
Published in Nature communications (19.02.2018)
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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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Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo
Jayaraman, Muthusamy, Ansell, Steven M., Mui, Barbara L., Tam, Ying K., Chen, Jianxin, Du, Xinyao, Butler, David, Eltepu, Laxman, Matsuda, Shigeo, Narayanannair, Jayaprakash K., Rajeev, Kallanthottathil G., Hafez, Ismail M., Akinc, Akin, Maier, Martin A., Tracy, Mark A., Cullis, Pieter R., Madden, Thomas D., Manoharan, Muthiah, Hope, Michael J.
Published in Angewandte Chemie International Edition (20.08.2012)
Published in Angewandte Chemie International Edition (20.08.2012)
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GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy
Kasiewicz, Lisa N., Biswas, Souvik, Beach, Aaron, Ren, Huilan, Dutta, Chaitali, Mazzola, Anne Marie, Rohde, Ellen, Chadwick, Alexandra, Cheng, Christopher, Garcia, Sara P., Iyer, Sowmya, Matsumoto, Yuri, Khera, Amit V., Musunuru, Kiran, Kathiresan, Sekar, Malyala, Padma, Rajeev, Kallanthottathil G., Bellinger, Andrew M.
Published in Nature communications (15.05.2023)
Published in Nature communications (15.05.2023)
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An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
Sehgal, Alfica, Barros, Scott, Ivanciu, Lacramioara, Cooley, Brian, Qin, June, Racie, Tim, Hettinger, Julia, Carioto, Mary, Jiang, Yongfeng, Brodsky, Josh, Prabhala, Harsha, Zhang, Xuemei, Attarwala, Husain, Hutabarat, Renta, Foster, Don, Milstein, Stuart, Charisse, Klaus, Kuchimanchi, Satya, Maier, Martin A, Nechev, Lubo, Kandasamy, Pachamuthu, Kel'in, Alexander V, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Meyers, Rachel, Sorensen, Benny, Simon, Amy R, Dargaud, Yesim, Negrier, Claude, Camire, Rodney M, Akinc, Akin
Published in Nature medicine (01.05.2015)
Published in Nature medicine (01.05.2015)
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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
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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Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
Stoffel, Markus, Wolfrum, Christian, Shi, Shuanping, Jayaprakash, K Narayanannair, Jayaraman, Muthusamy, Wang, Gang, Pandey, Rajendra K, Rajeev, Kallanthottathil G, Nakayama, Tomoko, Charrise, Klaus, Ndungo, Esther M, Zimmermann, Tracy, Koteliansky, Victor, Manoharan, Muthiah
Published in Nature biotechnology (01.10.2007)
Published in Nature biotechnology (01.10.2007)
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Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA
Schlegel, Mark K, Foster, Donald J, Kel’in, Alexander V, Zlatev, Ivan, Bisbe, Anna, Jayaraman, Muthusamy, Lackey, Jeremy G, Rajeev, Kallanthottathil G, Charissé, Klaus, Harp, Joel, Pallan, Pradeep S, Maier, Martin A, Egli, Martin, Manoharan, Muthiah
Published in Journal of the American Chemical Society (28.06.2017)
Published in Journal of the American Chemical Society (28.06.2017)
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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
Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles
Mui, Barbara L, Tam, Ying K, Jayaraman, Muthusamy, Ansell, Steven M, Du, Xinyao, Tam, Yuen Yi C, Lin, Paulo JC, Chen, Sam, Narayanannair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Akinc, Akin, Maier, Martin A, Cullis, Pieter, Madden, Thomas D, Hope, Michael J
Published in Molecular therapy. Nucleic acids (01.12.2013)
Published in Molecular therapy. Nucleic acids (01.12.2013)
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4′‑C‑Methoxy-2′-deoxy-2′-fluoro Modified Ribonucleotides Improve Metabolic Stability and Elicit Efficient RNAi-Mediated Gene Silencing
Malek-Adamian, Elise, Guenther, Dale C, Matsuda, Shigeo, Martínez-Montero, Saúl, Zlatev, Ivan, Harp, Joel, Burai Patrascu, Mihai, Foster, Donald J, Fakhoury, Johans, Perkins, Lydia, Moitessier, Nicolas, Manoharan, Rajar M, Taneja, Nate, Bisbe, Anna, Charisse, Klaus, Maier, Martin, Rajeev, Kallanthottathil G, Egli, Martin, Manoharan, Muthiah, Damha, Masad J
Published in Journal of the American Chemical Society (18.10.2017)
Published in Journal of the American Chemical Society (18.10.2017)
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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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Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity
Harp, Joel M, Guenther, Dale C, Bisbe, Anna, Perkins, Lydia, Matsuda, Shigeo, Bommineni, Gopal R, Zlatev, Ivan, Foster, Donald J, Taneja, Nate, Charisse, Klaus, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Egli, Martin
Published in Nucleic acids research (19.09.2018)
Published in Nucleic acids research (19.09.2018)
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Branched Multifunctional Polyether Polyketals: Variation of Ketal Group Structure Enables Unprecedented Control over Polymer Degradation in Solution and within Cells
Shenoi, Rajesh A, Narayanannair, Jayaprakash K, Hamilton, Jasmine L, Lai, Benjamin F. L, Horte, Sonja, Kainthan, Rajesh K, Varghese, Jos P, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Kizhakkedathu, Jayachandran N
Published in Journal of the American Chemical Society (12.09.2012)
Published in Journal of the American Chemical Society (12.09.2012)
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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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Structural Basis of Duplex Thermodynamic Stability and Enhanced Nuclease Resistance of 5′‑C‑Methyl Pyrimidine-Modified Oligonucleotides
Kel′in, Alexander V, Zlatev, Ivan, Harp, Joel, Jayaraman, Muthusamy, Bisbe, Anna, O′Shea, Jonathan, Taneja, Nate, Manoharan, Rajar M, Khan, Saeed, Charisse, Klaus, Maier, Martin A, Egli, Martin, Rajeev, Kallanthottathil G, Manoharan, Muthiah
Published in Journal of organic chemistry (18.03.2016)
Published in Journal of organic chemistry (18.03.2016)
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Journal Article
Versatile Site-Specific Conjugation of Small Molecules to siRNA Using Click Chemistry
Yamada, Takeshi, Peng, Chang Geng, Matsuda, Shigeo, Addepalli, Haripriya, Jayaprakash, K. Narayanannair, Alam, Md. Rowshon, Mills, Kathy, Maier, Martin A, Charisse, Klaus, Sekine, Mitsuo, Manoharan, Muthiah, Rajeev, Kallanthottathil G
Published in Journal of organic chemistry (04.03.2011)
Published in Journal of organic chemistry (04.03.2011)
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Non-Nucleoside Building Blocks for Copper-Assisted and Copper-Free Click Chemistry for the Efficient Synthesis of RNA Conjugates
Jayaprakash, K. N, Peng, Chang Geng, Butler, David, Varghese, Jos P, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah
Published in Organic letters (03.12.2010)
Published in Organic letters (03.12.2010)
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