SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
Naryshkin, Nikolai A., Weetall, Marla, Dakka, Amal, Narasimhan, Jana, Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Karp, Gary M., Qi, Hongyan, Woll, Matthew G., Chen, Guangming, Zhang, Nanjing, Gabbeta, Vijayalakshmi, Vazirani, Priya, Bhattacharyya, Anuradha, Furia, Bansri, Risher, Nicole, Sheedy, Josephine, Kong, Ronald, Ma, Jiyuan, Turpoff, Anthony, Lee, Chang-Sun, Zhang, Xiaoyan, Moon, Young-Choon, Trifillis, Panayiota, Welch, Ellen M., Colacino, Joseph M., Babiak, John, Almstead, Neil G., Peltz, Stuart W., Eng, Loren A., Chen, Karen S., Mull, Jesse L., Lynes, Maureen S., Rubin, Lee L., Fontoura, Paulo, Santarelli, Luca, Haehnke, Daniel, McCarthy, Kathleen D., Schmucki, Roland, Ebeling, Martin, Sivaramakrishnan, Manaswini, Ko, Chien-Ping, Paushkin, Sergey V., Ratni, Hasane, Gerlach, Irene, Ghosh, Anirvan, Metzger, Friedrich
Published in Science (American Association for the Advancement of Science) (08.08.2014)
Published in Science (American Association for the Advancement of Science) (08.08.2014)
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Small molecule splicing modifiers with systemic HTT-lowering activity
Bhattacharyya, Anuradha, Trotta, Christopher R., Narasimhan, Jana, Wiedinger, Kari J., Li, Wencheng, Effenberger, Kerstin A., Woll, Matthew G., Jani, Minakshi B., Risher, Nicole, Yeh, Shirley, Cheng, Yaofeng, Sydorenko, Nadiya, Moon, Young-Choon, Karp, Gary M., Weetall, Marla, Dakka, Amal, Gabbeta, Vijayalakshmi, Naryshkin, Nikolai A., Graci, Jason D., Tripodi, Thomas, Southwell, Amber, Hayden, Michael, Colacino, Joseph M., Peltz, Stuart W.
Published in Nature communications (15.12.2021)
Published in Nature communications (15.12.2021)
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Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy
Ratni, Hasane, Karp, Gary M, Weetall, Marla, Naryshkin, Nikolai A, Paushkin, Sergey V, Chen, Karen S, McCarthy, Kathleen D, Qi, Hongyan, Turpoff, Anthony, Woll, Matthew G, Zhang, Xiaoyan, Zhang, Nanjing, Yang, Tianle, Dakka, Amal, Vazirani, Priya, Zhao, Xin, Pinard, Emmanuel, Green, Luke, David-Pierson, Pascale, Tuerck, Dietrich, Poirier, Agnes, Muster, Wolfgang, Kirchner, Stephan, Mueller, Lutz, Gerlach, Irene, Metzger, Friedrich
Published in Journal of medicinal chemistry (14.07.2016)
Published in Journal of medicinal chemistry (14.07.2016)
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Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
Morini, Elisabetta, Chekuri, Anil, Logan, Emily M., Bolduc, Jessica M., Kirchner, Emily G., Salani, Monica, Krauson, Aram J., Narasimhan, Jana, Gabbeta, Vijayalakshmi, Grover, Shivani, Dakka, Amal, Mollin, Anna, Jung, Stephen P., Zhao, Xin, Zhang, Nanjing, Zhang, Sophie, Arnold, Michael, Woll, Matthew G., Naryshkin, Nikolai A., Weetall, Marla, Slaugenhaupt, Susan A.
Published in American journal of human genetics (02.03.2023)
Published in American journal of human genetics (02.03.2023)
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A deep learning approach to identify gene targets of a therapeutic for human splicing disorders
Gao, Dadi, Morini, Elisabetta, Salani, Monica, Krauson, Aram J., Chekuri, Anil, Sharma, Neeraj, Ragavendran, Ashok, Erdin, Serkan, Logan, Emily M., Li, Wencheng, Dakka, Amal, Narasimhan, Jana, Zhao, Xin, Naryshkin, Nikolai, Trotta, Christopher R., Effenberger, Kerstin A., Woll, Matthew G., Gabbeta, Vijayalakshmi, Karp, Gary, Yu, Yong, Johnson, Graham, Paquette, William D., Cutting, Garry R., Talkowski, Michael E., Slaugenhaupt, Susan A.
Published in Nature communications (07.06.2021)
Published in Nature communications (07.06.2021)
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Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
Woll, Matthew G, Qi, Hongyan, Turpoff, Anthony, Zhang, Nanjing, Zhang, Xiaoyan, Chen, Guangming, Li, Chunshi, Huang, Song, Yang, Tianle, Moon, Young-Choon, Lee, Chang-Sun, Choi, Soongyu, Almstead, Neil G, Naryshkin, Nikolai A, Dakka, Amal, Narasimhan, Jana, Gabbeta, Vijayalakshmi, Welch, Ellen, Zhao, Xin, Risher, Nicole, Sheedy, Josephine, Weetall, Marla, Karp, Gary M
Published in Journal of medicinal chemistry (14.07.2016)
Published in Journal of medicinal chemistry (14.07.2016)
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Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-Pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-negative Bacteria
Gerasyuto, Aleksey I, Arnold, Michael A, Wang, Jiashi, Chen, Guangming, Zhang, Xiaoyan, Smith, Sean, Woll, Matthew G, Baird, John, Zhang, Nanjing, Almstead, Neil G, Narasimhan, Jana, Peddi, Srinivasa, Dumble, Melissa, Sheedy, Josephine, Weetall, Marla, Branstrom, Arthur A, Prasad, J. V. N, Karp, Gary M
Published in Journal of medicinal chemistry (24.05.2018)
Published in Journal of medicinal chemistry (24.05.2018)
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Correction to Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-Negative Bacteria
Gerasyuto, Aleksey I, Arnold, Michael A, Wang, Jiashi, Chen, Guangming, Zhang, Xiaoyan, Smith, Sean, Woll, Matthew G, Baird, John, Zhang, Nanjing, Almstead, Neil G, Narasimhan, Jana, Peddi, Srinivasa, Dumble, Melissa, Sheedy, Josephine, Weetall, Marla, Branstrom, Arthur A, Prasad, J. V. N, Karp, Gary M
Published in Journal of medicinal chemistry (25.10.2018)
Published in Journal of medicinal chemistry (25.10.2018)
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Stereoselective Synthesis of 3-Substituted 2-Aminocyclopentanecarboxylic Acid Derivatives and Their Incorporation into Short 12-Helical β-Peptides That Fold in Water
Woll, Matthew G, Fisk, John D, LePlae, Paul R, Gellman, Samuel H
Published in Journal of the American Chemical Society (23.10.2002)
Published in Journal of the American Chemical Society (23.10.2002)
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Parallel Sheet Secondary Structure in γ-Peptides
Woll, Matthew G, Lai, Jonathan R, Guzei, Ilia A, Taylor, Stephen J. C, Smith, Mark E. B, Gellman, Samuel H
Published in Journal of the American Chemical Society (07.11.2001)
Published in Journal of the American Chemical Society (07.11.2001)
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Year of Publication 25.04.2024
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Year of Publication 25.04.2024
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Year of Publication 20.05.2024
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Year of Publication 20.05.2024
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Solution structure of a small protein containing a fluorinated side chain in the core
Cornilescu, Gabriel, Hadley, Erik B, Woll, Matthew G, Markley, John L, Gellman, Samuel H, Cornilescu, Claudia C
Published in Protein science (01.09.2007)
Published in Protein science (01.09.2007)
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SYDORENKO NADIYA, PATEL JIGAR S, CHIERCHIA MATTEO, REN HONGYU, CHEN GUANGMING, LUONG TOM TUAN, MOON YOUNG CHOON, KARP GARY MITCHELL, NARASIMHAN JANA, ZHANG XIAOYAN, ALAM RAUFUL, ZHANG NANJING, BABU SURESH, BHATTACHARYYA ANURADHA, MSZAR NICHOLAS WATER, WANG GANG, MAZZOTTI ANTHONY R, WOLL MATTHEW G
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헌팅턴병 치료를 위한 헤테로아릴 화합물
SYDORENKO NADIYA, TURPOFF ANTHONY, CHEN GUANGMING, MOON YOUNG CHOON, YAN WUMING, BARRAZA SCOTT J, KARP GARY MITCHELL, NARASIMHAN JANA, ZHANG NANJING, BABU SURESH, BHATTACHARYYA ANURADHA, MAZZOTTI ANTHONY R, WOLL MATTHEW G, KASSICK ANDREW J
Year of Publication 08.04.2021
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Year of Publication 08.04.2021
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