Opportunities and challenges in developing stearoyl-coenzyme A desaturase-1 inhibitors as novel therapeutics for human disease von Zhang, Zaihui, Dales, Natalie A, Winther, Michael D

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SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice von Palacino, James, Swalley, Susanne E, Song, Cheng, Cheung, Atwood K, Shu, Lei, Zhang, Xiaolu, Van Hoosear, Mailin, Shin, Youngah, Chin, Donovan N, Keller, Caroline Gubser, Beibel, Martin, Renaud, Nicole A, Smith, Thomas M, Salcius, Michael, Shi, Xiaoying, Hild, Marc, Servais, Rebecca, Jain, Monish, Deng, Lin, Bullock, Caroline, McLellan, Michael, Schuierer, Sven, Murphy, Leo, Blommers, Marcel J J, Blaustein, Cecile, Berenshteyn, Frada, Lacoste, Arnaud, Thomas, Jason R, Roma, Guglielmo, Michaud, Gregory A, Tseng, Brian S, Porter, Jeffery A, Myer, Vic E, Tallarico, John A, Hamann, Lawrence G, Curtis, Daniel, Fishman, Mark C, Dietrich, William F, Dales, Natalie A, Sivasankaran, Rajeev

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Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron‑2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA) von Cheung, Atwood K, Hurley, Brian, Kerrigan, Ryan, Shu, Lei, Chin, Donovan N, Shen, Yiping, O’Brien, Gary, Sung, Moo Je, Hou, Ying, Axford, Jake, Cody, Emma, Sun, Robert, Fazal, Aleem, Tomlinson, Ronald C, Jain, Monish, Deng, Lin, Hoffmaster, Keith, Song, Cheng, Van Hoosear, Mailin, Shin, Youngah, Servais, Rebecca, Towler, Christopher, Hild, Marc, Curtis, Daniel, Dietrich, William F, Hamann, Lawrence G, Briner, Karin, Chen, Karen S, Kobayashi, Dione, Sivasankaran, Rajeev, Dales, Natalie A

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An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion von Keller, Caroline Gubser, Shin, Youngah, Monteys, Alex Mas, Renaud, Nicole, Beibel, Martin, Teider, Natalia, Peters, Thomas, Faller, Thomas, St-Cyr, Sophie, Knehr, Judith, Roma, Guglielmo, Reyes, Alejandro, Hild, Marc, Lukashev, Dmitriy, Theil, Diethilde, Dales, Natalie, Cha, Jang-Ho, Borowsky, Beth, Dolmetsch, Ricardo, Davidson, Beverly L., Sivasankaran, Rajeev

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Discovery and characterization of a selective IKZF2 glue degrader for cancer immunotherapy von Bonazzi, Simone, d’Hennezel, Eva, Beckwith, Rohan E.J., Xu, Lei, Fazal, Aleem, Magracheva, Anna, Ramesh, Radha, Cernijenko, Artiom, Antonakos, Brandon, Bhang, Hyo-eun C., Caro, Roxana García, Cobb, Jennifer S., Ornelas, Elizabeth, Ma, Xiaolei, Wartchow, Charles A., Clifton, Matthew C., Forseth, Ry R., Fortnam, Bethany Hughes, Lu, Hongbo, Csibi, Alfredo, Tullai, Jennifer, Carbonneau, Seth, Thomsen, Noel M., Larrow, Jay, Chie-Leon, Barbara, Hainzl, Dominik, Gu, Yi, Lu, Darlene, Meyer, Matthew J., Alexander, Dylan, Kinyamu-Akunda, Jacqueline, Sabatos-Peyton, Catherine A., Dales, Natalie A., Zécri, Frédéric J., Jain, Rishi K., Shulok, Janine, Wang, Y. Karen, Briner, Karin, Porter, Jeffery A., Tallarico, John A., Engelman, Jeffrey A., Dranoff, Glenn, Bradner, James E., Visser, Michael, Solomon, Jonathan M.

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Use of Intramolecular 1,5-Sulfur–Oxygen and 1,5-Sulfur–Halogen Interactions in the Design of N‑Methyl-5-aryl‑N‑(2,2,6,6-tetramethylpiperidin-4-yl)-1,3,4-thiadiazol-2-amine SMN2 Spl... von Axford, Jake, Sung, Moo Je, Manchester, John, Chin, Donovan, Jain, Monish, Shin, Youngah, Dix, Ina, Hamann, Lawrence G, Cheung, Atwood K, Sivasankaran, Rajeev, Briner, Karin, Dales, Natalie A, Hurley, Brian

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A molecular glue degrader of the WIZ transcription factor for fetal hemoglobin induction von Ting, Pamela Y, Borikar, Sneha, Kerrigan, John Ryan, Thomsen, Noel M, Aghania, Eamon, Hinman, Amelia E, Reyes, Alejandro, Pizzato, Nicolas, Fodor, Barna D, Wu, Fabian, Belew, Muluken S, Mao, Xiaohong, Wang, Jian, Chitnis, Shripad, Niu, Wei, Hachey, Amanda, Cobb, Jennifer S, Savage, Nikolas A, Burke, Ashley, Paulk, Joshiawa, Dovala, Dustin, Lin, James, Clifton, Matthew C, Ornelas, Elizabeth, Ma, Xiaolei, Ware, Nathaniel F, Sanchez, Carina C, Taraszka, John, Terranova, Remi, Knehr, Judith, Altorfer, Marc, Barnes, S Whitney, Beckwith, Rohan E J, Solomon, Jonathan M, Dales, Natalie A, Patterson, Andrew W, Wagner, Jürgen, Bouwmeester, Tewis, Dranoff, Glenn, Stevenson, Susan C, Bradner, James E

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Discovery and Optimization of First-in-Class Molecular Glue Degraders of the WIZ Transcription Factor for Fetal Hemoglobin Induction to Treat Sickle Cell Disease von Kerrigan, John Ryan, Thomsen, Noel M., Cernijenko, Artiom, Kochanek, Sarah E., Dewhurst, Janetta, O’Brien, Gary, Ware, Nathaniel F., Sanchez, Carina C., Manning, James R., Ma, Xiaolei, Ornelas, Elizabeth, Savage, Nikolas A., Partridge, James R., Patterson, Andrew W., Lam, Philip, Dales, Natalie A., Bonazzi, Simone, Borikar, Sneha, Hinman, Amelia E., Ting, Pamela Y.

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ACE2 X-Ray Structures Reveal a Large Hinge-bending Motion Important for Inhibitor Binding and Catalysis von Towler, Paul, Staker, Bart, Prasad, Sridhar G., Menon, Saurabh, Tang, Jin, Parsons, Thomas, Ryan, Dominic, Fisher, Martin, Williams, David, Dales, Natalie A., Patane, Michael A., Pantoliano, Michael W.

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Systematic evaluation of amide bioisosteres leading to the discovery of novel and potent thiazolylimidazolidinone inhibitors of SCD1 for the treatment of metabolic diseases von Sun, Shaoyi, Zhang, Zaihui, Kodumuru, Vishnumurthy, Pokrovskaia, Natalia, Fonarev, Julia, Jia, Qi, Leung, Po-Yee, Tran, Jennifer, Ratkay, Leslie G., McLaren, David G., Radomski, Chris, Chowdhury, Sultan, Fu, Jianmin, Hubbard, Brian, Winther, Michael D., Dales, Natalie A.

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Opportunities and Challenges in Developing Stearoyl-Coenzyme A Desaturase‑1 Inhibitors as Novel Therapeutics for Human Disease: Miniperspective von Zhang, Zaihui, Dales, Natalie A, Winther, Michael D

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Pharmacological Inhibition of Stearoyl CoA Desaturase in the Skin Induces Atrophy of the Sebaceous Glands von Meingassner, Josef G., Aschauer, Heinrich, Winiski, Anthony P., Dales, Natalie, Yowe, David, Winther, Michael D., Zhang, Zaihui, Stütz, Anton, Billich, Andreas

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Substrate-Based Design of the First Class of Angiotensin-Converting Enzyme-Related Carboxypeptidase (ACE2) Inhibitors von Dales, Natalie A, Gould, Alexandra E, Brown, James A, Calderwood, Emily F, Guan, Bing, Minor, Charles A, Gavin, James M, Hales, Paul, Kaushik, Virendar K, Stewart, Michael, Tummino, Peter J, Vickers, Chad S, Ocain, Timothy D, Patane, Michael A

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Discovery of triazolone derivatives as novel, potent stearoyl-CoA desaturase-1 (SCD1) inhibitors von Sun, Shaoyi, Zhang, Zaihui, Pokrovskaia, Natalia, Chowdhury, Sultan, Jia, Qi, Chang, Elaine, Khakh, Kuldip, Kwan, Rainbow, McLaren, David G., Radomski, Chris C., Ratkay, Leslie G., Fu, Jianmin, Dales, Natalie A., Winther, Michael D.

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Enzymatic Characterization of the Pancreatic Islet-specific Glucose-6-Phosphatase-related Protein (IGRP) von Petrolonis, Anthony J, Yang, Qing, Tummino, Peter J, Fish, Susan M, Prack, Andrea E, Jain, Sadhana, Parsons, Thomas F, Li, Ping, Dales, Natalie A, Ge, Lin, Langston, Steven P, Schuller, Alwin G P, An, W Frank, Tartaglia, Louis A, Chen, Hong, Hong, Suk-Bong

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Targeted Degradation of the Wiz Transcription Factor for Gamma Globin De-Repression von Ting, Pamela Y, Borikar, Sneha, Kerrigan, John Ryan, Thomsen, Noel M, Aghania, Eamon, Hinman, Amelia E, Pizzato, Nicolas, Reyes, Alejandro, Fodor, Barna D, Wu, Fabian, Belew, Muluken S, Mao, Xiaohong, Wang, Jian, Chitnis, Shripad, Ashcroft, Peter, Niu, Wei, Hachey, Amanda, Cobb, Jennifer S, Savage, Nikolas A, Burke, Ashley, Paulk, Joshiawa, Dovala, Dustin, Lin, James, Clifton, Matthew C, Ornelas, Elizabeth, Ma, Xiaolei, Ware, Nathaniel F, Sanchez, Carina C, Terranova, Remi, Knehr, Judith, Altorfer, Marc, Barnes, S. Whitney, Taraszka, John, Lubicka, Danuta, Manning, James R, Miranda, Sonia R, Beckwith, Rohan EJ, Solomon, Jonathan M, Forrester, William, Hollingworth, Gregory, Dales, Natalie A, Patterson, Andrew W, Bouwmeester, Tewis, Wagner, Juergen, Dranoff, Glenn, Stevenson, Susan C, Bradner, James E

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Discovery of a novel indole pharmacophore for the irreversible inhibition of myeloperoxidase (MPO) von Patnaik, Anup, Axford, Laura, Deng, Lin, Cohick, Evan, Ren, Xianglin, Loi, Sally, Kecman, Sam, Hollis-Symynkywicz, Micah, Harrison, Tyler J., Papillon, Julien P.N., Dales, Natalie, Hamann, Lawrence G., Lee, Lac, Regard, Jean B., Marcinkeviciene, Jovita, Marro, Martin L., Patterson, Andrew W.

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Design and Synthesis of Unsymmetrical Peptidyl Urea Inhibitors of Aspartic Peptidases von Dales, Natalie A, Bohacek, Regine S, Satyshur, Kenneth A, Rich, Daniel H

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BET bromodomain inhibitors regulate keratinocyte plasticity von Schutzius, Gabi, Kolter, Christian, Bergling, Sebastian, Tortelli, Federico, Fuchs, Florian, Renner, Steffen, Guagnano, Vito, Cotesta, Simona, Rueeger, Heinrich, Faller, Michael, Bouchez, Laure, Salathe, Adrian, Nigsch, Florian, Richards, Shola M., Louis, Malvina, Gruber, Viktoria, Aebi, Alexandra, Turner, Jonathan, Grandjean, Frederic, Li, Jun, Dimitri, Chris, Thomas, Jason R., Schirle, Markus, Blank, Jutta, Drueckes, Peter, Vaupel, Andrea, Tiedt, Ralph, Manley, Paul W., Klopp, Julia, Hemmig, Rene, Zink, Florence, Leroy, Nelly, Carbone, Walter, Roma, Guglielmo, Keller, Caroline Gubser, Dales, Natalie, Beyerbach, Armin, Zimmerlin, Alfred, Bonenfant, Debora, Terranova, Remi, Berwick, Amy, Sahambi, Sukhdeep, Reynolds, Aimee, Jennings, Lori L., Ruffner, Heinz, Tarsa, Peter, Bouwmeester, Tewis, Driver, Vickie, Frederiksen, Mathias, Lohmann, Felix, Kirkland, Susan

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Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice von Palacino, James, Swalley, Susanne E, Song, Cheng, Cheung, Atwood K, Shu, Lei, Zhang, Xiaolu, Van Hoosear, Mailin, Shin, Youngah, Chin, Donovan N, Keller, Caroline Gubser, Beibel, Martin, Renaud, Nicole A, Smith, Thomas M, Salcius, Michael, Shi, Xiaoying, Hild, Marc, Servais, Rebecca, Jain, Monish, Deng, Lin, Bullock, Caroline, McLellan, Michael, Schuierer, Sven, Murphy, Leo, Blommers, Marcel J J, Blaustein, Cecile, Berenshteyn, Frada, Lacoste, Arnaud, Thomas, Jason R, Roma, Guglielmo, Michaud, Gregory A, Tseng, Brian S, Porter, Jeffery A, Myer, Vic E, Tallarico, John A, Hamann, Lawrence G, Curtis, Daniel, Fishman, Mark C, Dietrich, William F, Dales, Natalie A, Sivasankaran, Rajeev

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