المؤلفون: Savitri Mandapati, Chunjuan Song, Alok K. Sharma, Mailin Van Hoosear, William W. Hauswirth, Guo-jie Ye, Jeffrey D. Chulay, Adrian M. Timmers, Cayrn Plummer, Ping Zhu, David R. Knop, Kari B. Green, Paulette M. Robinson, Peter Sonnentag, Wen-Tao Deng, Mark S. Shearman, Thomas J. Conlon, Kirsten E. Coleman

المصدر: Human Gene Therapy Clinical Development. 29:188-197

مصطلحات موضوعية: 0301 basic medicine, G-Protein-Coupled Receptor Kinase 1, Genetic enhancement, law.invention, Mice, 03 medical and health sciences, law, Complementary DNA, Retinitis pigmentosa, medicine, Animals, Codon, Eye Proteins, Gene, Research Articles, Genetics (clinical), medicine.diagnostic_test, HEK 293 cells, Genetic Therapy, Retinitis pigmentosa GTPase regulator, Dependovirus, medicine.disease, Molecular biology, eye diseases, 030104 developmental biology, Recombinant DNA, sense organs, Carrier Proteins, Retinitis Pigmentosa, Electroretinography

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3b489755bd2d1a5d95261f2a3fbdae5a
https://doi.org/10.1089/humc.2018.168

المؤلفون: Natalie Dales, Monish Jain, Sanchez Carina Cristina, Ryan Kerrigan, Atwood K. Cheung, Cary Fridrich, Donovan N. Chin, Brian Hurley, Keith Hoffmaster, Lin Deng, Ronald Tomlinson, Marc Hild, Rajeev Sivasankaran, Jake Axford, Aleem Fazal, Gary O’Brien, Christopher Towler, Mailin Van Hoosear, Youngah Shin, Dione Kobayashi, Shen Yiping, Lei Shu, William F. Dietrich, Karen S. Chen, Lawrence G. Hamann, Karin Briner, Rebecca Servais, Cheng Song, Emma Cody, Robert Sun, Daniel Curtis, Moo Je Sung, Ying Hou

المصدر: Journal of Medicinal Chemistry. 61:11021-11036

مصطلحات موضوعية: 0301 basic medicine, ERG1 Potassium Channel, Spliceosome, RNA Splicing, Phenotypic screening, Drug Evaluation, Preclinical, Administration, Oral, Quantitative Structure-Activity Relationship, SMN1, Crystallography, X-Ray, Cell Line, Muscular Atrophy, Spinal, Rats, Sprague-Dawley, 03 medical and health sciences, 0302 clinical medicine, Drug Discovery, medicine, Animals, Humans, Gene, Motor Neurons, Dose-Response Relationship, Drug, Chemistry, Brain, Spinal muscular atrophy, Motor neuron, medicine.disease, SMA, Survival of Motor Neuron 1 Protein, nervous system diseases, Mice, Inbred C57BL, Pyridazines, Survival of Motor Neuron 2 Protein, 030104 developmental biology, medicine.anatomical_structure, RNA splicing, Molecular Medicine, Neuroscience, 030217 neurology & neurosurgery

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a6a86b835f8a7bec6e7356af0b03dae4
https://doi.org/10.1021/acs.jmedchem.8b01291

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

المصدر: Nature Chemical Biology. 11:511-517

مصطلحات موضوعية: Models, Molecular, Gene Expression, Mice, Transgenic, SMN1, Biology, Ribonucleoprotein, U1 Small Nuclear, Muscular Atrophy, Spinal, Small Molecule Libraries, Mice, RNA Precursors, Animals, Humans, splice, snRNP, Enhancer, Molecular Biology, Gene, RNA, Double-Stranded, Binding Sites, Protein Stability, Cell Biology, SMA, Survival Analysis, Molecular biology, nervous system diseases, Survival of Motor Neuron 2 Protein, Alternative Splicing, Disease Models, Animal, Proteolysis, RNA splicing, Female, Precursor mRNA, Protein Binding

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8d75ec4fdc6df87f6744ae079777065c
https://doi.org/10.1038/nchembio.1837

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

المصدر: Nature Chemical Biology. 12:304-304

مصطلحات موضوعية: business.industry, Medicine, Physiology, splice, Cell Biology, Computational biology, business, SMA, Precursor mRNA, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ce99bc4efe7e0da691707c1b35b48fbd
https://doi.org/10.1038/nchembio0416-304c