recent publications
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academic article
- Haniff, H. S., Knerr, L., Chen, J. L., Disney, M. D., Lightfoot, H. L. Target-Directed Approaches for Screening Small Molecules against RNA Targets SLAS Discovery 2020 DOI:10.1177/2472555220922802 PMID:32419578
- Benhamou, R. I., Abe, M., Choudhary, S., Meyer, S. M., Angelbello, A. J., Disney, M. D. Optimization of the Linker Domain in a Dimeric Compound that Degrades an r(CUG) Repeat Expansion in Cells Journal of Medicinal Chemistry 2020 63:7827-7839 DOI:10.1021/acs.jmedchem.0c00558 PMID:32657583 PMCID:PMC7450518
- Angelbello, A. J., Disney, M. D. A Toxic RNA Templates the Synthesis of Its Own Fluorogenic Inhibitor by Using a Bio-orthogonal Tetrazine Ligation in Cells and Tissues ACS Chemical Biology 2020 15:1820-1825 DOI:10.1021/acschembio.0c00417 PMID:32551539 PMCID:PMC7392402
- Angelbello, A. J., Chen, J. L., Disney, M. D. Small molecule targeting of RNA structures in neurological disorders Annals of the New York Academy of Sciences 2020 1471:57-71 DOI:10.1111/nyas.14051 PMID:30964958 PMCID:PMC6785366
- Disney, M. D., Suresh, B. M., Benhamou, R. I., Childs-Disney, J. L. Progress toward the development of the small molecule equivalent of small interfering RNA Current Opinion in Chemical Biology 2020 56:63-71 DOI:10.1016/j.cbpa.2020.01.001 PMID:32036231 PMCID:PMC7311281
- Chen, J. L., Zhang, P., Abe, M., Aikawa, H., Zhang, L., Frank, A. J., Zembryski, T., Hubbs, C., Park, H., Withka, J., Steppan, C., Rogers, L., et al. Design, Optimization, and Study of Small Molecules That Target Tau Pre-mRNA and Affect Splicing Journal of the American Chemical Society 2020 142:8706-8727 DOI:10.1021/jacs.0c00768 PMID:32364710
- Todd, T. W., McEachin, Z. T., Chew, J., Burch, A. R., Jansen-West, K., Tong, J. M., Yue, M., Song, Y. P., Castanedes-Casey, M., Kurti, A., Dunmore, J. H., Fryer, J. D., et al. Hexanucleotide Repeat Expansions in c9FTD/ALS and SCA36 Confer Selective Patterns of Neurodegeneration In Vivo Cell Reports 2020 31 DOI:10.1016/j.celrep.2020.107616 PMID:32375043
- Angelbello, A. J., Defeo, M. E., Glinkerman, C. M., Boger, D. L., Disney, M. D. Precise Targeted Cleavage of a r(CUG) Repeat Expansion in Cells by Using a Small-Molecule-Deglycobleomycin Conjugate ACS Chemical Biology 2020 15:849-855 DOI:10.1021/acschembio.0c00036 PMCID:32186845
- Liu, X. H., Haniff, H. S., Childs-Disney, J. L., Shuster, A., Aikawa, H., Adibekian, A., Disney, M. D. Targeted Degradation of the Oncogenic MicroRNA 17-92 Cluster by Structure-Targeting Ligands Journal of the American Chemical Society 2020 142:6970-6982 DOI:10.1021/jacs.9b13159 PMID:32233464
- Benhamou, R. I., Angelbello, A. J., Wang, E. T., Disney, M. D. A toxic RNA catalyzes the cellular synthesis of its own inhibitor, shunting it to endogenous decay pathways Cell Chemical Biology 2020 27:223-231 DOI:10.1016/j.chembiol.2020.01.003 PMID:31981476 PMCID:PMC7081931
- Costales, M. G., Aikawa, H., Li, Y., Childs-Disney, J. L., Abegg, D., Hoch, D. G., Velagapudi, S. P., Nakai, Y., Khan, T., Wang, K. W., Yildirim, I., Adibekian, A., et al. Small-molecule targeted recruitment of a nuclease to cleave an oncogenic RNA in a mouse model of metastatic cancer Proceedings of the National Academy of Sciences of the United States of America 2020 117:2406-2411 DOI:10.1073/pnas.1914286117 PMID:31964809 PMCID:PMC7007575
- Benhamou, R. I., Angelbello, A. J., Andrews, R. J., Wang, E. T., Moss, W. N., Disney, M. D. Structure-specific cleavage of an RNA repeat expansion with a dimeric small molecule is advantageous over sequence-specific recognition by an oligonucleotide ACS Chemical Biology 2020 15:485-493 DOI:10.1021/acschembio.9b00958 PMID:31927948 PMCID:PMC7081929
- Zhang, P., Park, H. J., Zhang, J., Junn, E., Andrews, R. J., Velagapudi, S. P., Abegg, D., Vishnu, K., Costales, M. G., Childs-Disney, J. L., Adibekian, A., Moss, W. N., et al. Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA Proceedings of the National Academy of Sciences of the United States of America 2020 117:1457-1467 DOI:10.1073/pnas.1905057117 PMID:31900363 PMCID:PMC6983430
- Haniff, H. S., Knerr, L., Liu, X. H., Crynen, G., Bostrom, J., Abegg, D., Adibekian, A., Lekah, E., Wang, K. W., Cameron, M. D., Yildirim, I., Lemurell, M., et al. Design of a small molecule that stimulates vascular endothelial growth factor A enabled by screening RNA fold-small molecule interactions Nature Chemistry 2020 12:952-961 DOI:10.1038/s41557-020-0514-4 PMID:32839603
- Ursu, A., Childs-Disney, J. L., Andrews, R. J., O'Leary, C. A., Meyer, S. M., Angelbello, A. J., Moss, W. N., Disney, M. D. Design of small molecules targeting RNA structure from sequence Chemical Society Reviews 2020 49:7252-7270 DOI:10.1039/d0cs00455c PMID:32935689 PMCID:PMC7707016
- Ursu, A., Childs-Disney, J. L., Angelbello, A. J., Costales, M. G., Meyer, S. M., Disney, M. D. Gini Coefficients as a Single Value Metric to Define Chemical Probe Selectivity ACS Chemical Biology 2020 15:2031-2040 DOI:10.1021/acschembio.0c00486 PMID:32568503 PMCID:PMC7442733
- Costales, M. G., Childs-Disney, J. L., Haniff, H. S., Disney, M. D. How We Think about Targeting RNA with Small Molecules Journal of Medicinal Chemistry 2020 63:8880-8900 DOI:10.1021/acs.jmedchem.9b01927 PMID:32212706 PMCID:PMC7486258
- Benhamou, R. I., Vezina-Dawod, S., Choudhary, S., Wang, K. W., Meyer, S. M., Yildirim, I., Disney, M. D. Macrocyclization of a Ligand Targeting a Toxic RNA Dramatically Improves Potency Chembiochem 2020 DOI:10.1002/cbic.202000445 PMID:32649032
- Meyer, S. M., Williams, C. C., Akahori, Y., Tanaka, T., Aikawa, H., Tong, Y., Childs-Disney, J. L., Disney, M. D. Small molecule recognition of disease-relevant RNA structures Chemical Society Reviews 2020 49:7167-7199 DOI:10.1039/d0cs00560f PMID:32975549
- Haniff, H. S., Tong, Y., Liu, X. H., Chen, J. L., Suresh, B. M., Andrews, R. J., Peterson, J. M., O'Leary, C. A., Benhamou, R. I., Moss, W. N., Disney, M. D. Targeting the SARS-CoV-2 RNA Genome with Small Molecule Binders and Ribonuclease Targeting Chimera (RIBOTAC) Degraders ACS Central Science 2020 6:1713-1721 DOI:10.1021/acscentsci.0c00984 PMID:33140033 PMCID:PMC7553039
- Ursu, A., Vezina-Dawod, S., Disney, M. D. Methods to identify and optimize small molecules interacting with RNA (SMIRNAs) Drug Discovery Today 2019 24:2002-2016 DOI:10.1016/j.drudis.2019.06.019 PMID:31356880 PMCID:PMC6842402
- Costales, M. G., Suresh, B., Vishnu, K., Disney, M. D. Targeted degradation of a hypoxia-associated non-coding RNA enhances the selectivity of a small molecule interacting with RNA Cell Chemical Biology 2019 26:1180-1186 DOI:10.1016/j.chembiol.2019.04.008 PMID:31130520 PMCID:PMC6697612
- Chen, J. L., Moss, W. N., Spencer, A., Zhang, P., Childs-Disney, J. L., Disney, M. D. The RNA encoding the microtubule-associated protein tau has extensive structure that affects its biology PLoS One 2019 14 DOI:10.1371/journal.pone.0219210 PMID:31291322 PMCID:PMC6619747
- O'Leary, C. A., Andrews, R. J., Tompkins, V., Chen, J. L., Childs-Disney, J. L., Disney, M. D., Moss, W. N. RNA structural analysis of the MYC mRNA reveals conserved motifs that affect gene expression PLoS One 2019 14 DOI:10.1371/journal.pone.0213758 PMID:31206539 PMCID:PMC6576772
- Velagapudi, S. P., Li, Y., Disney, M. D. A cross-linking approach to map small molecule-RNA binding sites in cells Bioorganic & Medicinal Chemistry Letters 2019 29:1532-1536 DOI:10.1016/j.bmcl.2019.04.001 PMID:30987892
- Disney, M. D. Targeting RNA with small molecules to capture opportunities at the intersection of chemistry, biology, and medicine Journal of the American Chemical Society 2019 141:6776-6790 DOI:10.1021/jacs.8b13419 PMID:30896935
- Costales, M. G., Disney, M. D. Adding broccoli to the biosensor menu Cell Chemical Biology 2019 26:463-465 DOI:10.1016/j.chembiol.2019.04.004 PMID:31002799
- Angelbello, A. J., Rzuczek, S. G., McKee, K. K., Chen, J. L., Olafson, H., Cameron, M. D., Moss, W. N., Wang, E. T., Disney, M. D. Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model Proceedings of the National Academy of Sciences of the United States of America 2019 116:7799-7804 DOI:10.1073/pnas.1901484116 PMID:30926669 PMCID:PMC6475439
- Wang, Z. F., Ursu, A., Childs-Disney, J. L., Guertler, R., Yang, W. Y., Bernat, V., Rzuczek, S. G., Fuerst, R., Zhang, Y. J., Gendron, T. F., Yildirim, I., Dwyer, B. G., et al. The hairpin form of r(G(4)C(2))(exp) in c9ALS/FTD is repeat-associated non-ATG translated and a target for bioactive small molecules Cell Chemical Biology 2019 26:179-190 DOI:10.1016/j.chembiol.2018.10.018 PMID:30503283 PMCID:PMC6386614
- Costales, M. G., Hoch, D. G., Abegg, D., Childs-Disney, J. L., Velagapudi, S. P., Adibekian, A., Disney, M. D. A designed small molecule inhibitor of a non-coding RNA sensitizes HER2 negative cancers to herceptin Journal of the American Chemical Society 2019 141:2960-2974 DOI:10.1021/jacs.8b10558 PMID:30726072 PMCID:PMC6400281
- Chew, J., Cook, C., Gendron, T. F., Jansen-West, K., del Rosso, G., Daughrity, L. M., Castanedes-Casey, M., Kurti, A., Stankowski, J. N., Disney, M. D., Rothstein, J. D., Dickson, D. W., et al. Aberrant deposition of stress granule-resident proteins linked to C9orf72-associated TDP-43 proteinopathy Molecular Neurodegeneration 2019 14 DOI:10.1186/s13024-019-0310-z PMID:30767771 PMCID:PMC6377782
- Wales, D. J., Disney, M. D., Yildirim, I. Computational investigation of RNA A-bulges related to the microtubule-associated protein tau causing frontotemporal dementia and Parkinsonism Journal of Physical Chemistry B 2019 123:57-65 DOI:10.1021/acs.jpcb.8b09139 PMID:30517788
- Disney, M. D., Velagapudi, S. P., Li, Y., Costales, M. G., Childs-Disney, J. L. Identifying and validating small molecules interacting with RNA (SMIRNAs) Methods in Enzymology 2019 623:45-66 DOI:10.1016/bs.mie.2019.04.027 PMID:31239057 PMCID:PMC6628145
- Disney, M. D., Dwyer, B. G., Childs-Disney, J. L. Drugging the RNA world Cold Spring Harbor Perspectives in Biology 2018 10 DOI:10.1101/cshperspect.a034769 PMID:30385607
- Li, Y., Disney, M. D. Precise small molecule degradation of a noncoding RNA identifies cellular binding sites and modulates an oncogenic phenotype ACS Chemical Biology 2018 13:3065-3071 DOI:10.1021/acschembio.8b00827 PMID:30375843
- Disney, M. D. Introduction to the Symposium In Print for Laura Kiessling Bioorganic & Medicinal Chemistry 2018 26:III-III DOI:10.1016/j.bmc.2018.10.021 PMID:30420097
- Childs-Disney, J. L., Tran, T., Vummidi, B. R., Velagapudi, S. P., Haniff, H. S., Matsumoto, Y., Crynen, G., Southern, M. R., Biswas, A., Wang, Z., Tellinghuisen, T. L., Disney, M. D. A massively parallel selection of small molecule-RNA motif binding partners informs design of an antiviral from sequence Chem 2018 4:2384-2404 DOI:10.1016/j.chempr.2018.08.003
- Velagapudi, S. P., Costales, M. G., Vummidi, B. R., Nakai, Y., Angelbello, A. J., Tran, T., Haniff, H. S., Matsumoto, Y., Wang, Z. F., Chatterjee, A. K., Childs-Disney, J. L., Disney, M. D. Approved anti-cancer drugs target oncogenic non-coding RNAs Cell Chemical Biology 2018 25:1086-1094 DOI:10.1016/j.chembiol.2018.05.015 PMID:30251629
- Disney, M. D., Janda, K. D. Introduction Bioorganic & Medicinal Chemistry Letters 2018 28:2661-2662 DOI:10.1016/j.bmcl.2018.07.013 PMID:30029841
- Guan, L., Luo, Y., Ja, W. W., Disney, M. D. Small molecule alteration of RNA sequence in cells and animals Bioorganic & Medicinal Chemistry Letters 2018 28:2794-2796 DOI:10.1016/j.bmcl.2018.10.034 PMID:29079470 PMCID:PMC5906209
- Haniff, H. S., Graves, A., Disney, M. D. Selective small molecule recognition of RNA base pairs ACS Combinatorial Science 2018 20:482-491 DOI:10.1021/acscombsci.8b00049 PMID:29966095
- Costales, M. G., Matsumoto, Y., Velagapudi, S. P., Disney, M. D. Small molecule targeted recruitment of a nuclease to RNA Journal of the American Chemical Society 2018 140:6741-6744 DOI:10.1021/jacs.8b01233 PMID:29792692
- Angelbello, A. J., Chen, J. L., Childs-Disney, J. L., Zhang, P., Wang, Z. F., Disney, M. D. Using genome sequence to enable the design of medicines and chemical probes Chemical Reviews 2018 118:1599-1663 DOI:10.1021/acs.chemrev.7b00504 PMID:29322778
- Angelbello, A. J., Disney, M. D. Bleomycin can cleave an oncogenic noncoding RNA ChemBioChem 2018 19:43-47 DOI:10.1002/cbic.201700581 PMID:29084369
- Chen, J. L., VanEtten, D. M., Fountain, M. A., Yildirim, I., Disney, M. D. Structure and dynamics of RNA repeat expansions that cause Huntington's disease and myotonic dystrophy type 1 Biochemistry 2017 56:3463-3474 DOI:10.1021/acs.biochem.7b00252 PMID:28617590
- Disney, M. D. Inhibiting translation one protein at a time Trends in Biochemical Sciences 2017 42:412-413 DOI:10.1016/j.tibs.2017.04.008 PMID:28522328
- Gendron, T. F., Chew, J., Stankowski, J. N., Hayes, L. R., Zhang, Y. J., Prudencio, M., Carlomagno, Y., Daughrity, L. M., Jansen-West, K., Perkerson, E. A., O'Raw, A., Cook, C., et al. Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis Science Translational Medicine 2017 9 DOI:10.1126/scitranslmed.aai7866 PMID:28356511
- Velagapudi, S. P., Luo, Y., Tran, T., Haniff, H. S., Nakai, Y., Fallahi, M., Martinez, G. J., Childs-Disney, J. L., Disney, M. D. Defining RNA small molecule affinity landscapes enables design of a small molecule inhibitor of an oncogenic noncoding RNA ACS Central Science 2017 3:205-216 DOI:10.1021/acscentsci.7b00009 PMID:28386598 PMCID:PMC5364451
- Costales, M. G., Childs-Disney, J. L., Disney, M. D. Computational tools for design of selective small molecules targeting RNA: from small molecule microarrays to chemical similarity searching Topics in Medicinal Chemistry 2017 27:1-16
- Costales, M. G., Haga, C. L., Velagapudi, S. P., Childs-Disney, J. L., Phinney, D. G., Disney, M. D. Small molecule inhibition of microRNA-210 reprograms an oncogenic hypoxic circuit Journal of the American Chemical Society 2017 139:3446-3455 DOI:10.1021/jacs.61311273 PMID:28240549
- Rzuczek, S. G., Colgan, L. A., Nakai, Y., Cameron, M. D., Furling, D., Yasuda, R., Disney, M. D. Precise small-molecule recognition of a toxic CUG RNA repeat expansion Nature Chemical Biology 2017 13:188-193 DOI:10.1038/nchembio.2251 PMID:27941760 PMCID:PMC5290590
- Disney, M. D. Rational design of small molecules targeting oncogenic noncoding RNAs from sequence Accounts of Chemical Research 2016 49:2698-2704 DOI:10.1021/acs.accounts.6b00326 PMID:27993012
- Park, H., Tran, T., Lee, J. H., Park, H., Disney, M. D. Controlled dehydration improves the diffraction quality of two RNA crystals BMC Structural Biology 2016 16 DOI:10.1186/s12900-016-0069-1 PMID:27809904 PMCID:PMC5093936
- Disney, M. D. Chemistry and chemical biology of therapeutically important compounds [Preface] Bioorganic & Medicinal Chemistry 2016 24:3875-3875 DOI:10.1016/j.bmc.2016.06.049 PMID:27460698
- Yang, W. Y., He, F., Strack, R. L., Oh, S. Y., Frazer, M., Jaffrey, S. R., Todd, P. K., Disney, M. D. Small molecule recognition and tools to study modulation of r(CGG)(exp) in fragile X-associated tremor ataxia syndrome ACS Chemical Biology 2016 11:2456-2465 DOI:10.1021/acschembio.6b00147 PMID:27276216
- Costales, M. G., Rzuczek, S. G., Disney, M. D. Comparison of small molecules and oligonucleotides that target a toxic, non-coding RNA Bioorganic & Medicinal Chemistry Letters 2016 26:2605-2609 DOI:10.1016/j.bmcl.2016.04.025 PMID:27117425
- Yang, W. Y., Gao, R., Southern, M., Sarkar, P. S., Disney, M. D. Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10 Nature Communications 2016 7 DOI:10.1038/ncomms11647 PMID:27248057 PMCID:27248057
- Disney, M. D., Winkelsas, A. M., Velagapudi, S. P., Southern, M., Fallahi, M., Childs-Disney, J. L. Inforna 2.0: a platform for the sequence-based design of small molecules targeting structured RNAs ACS Chemical Biology 2016 11:1720-1728 DOI:10.1021/acschembio.6b00001 PMID:27097021 PMCID:PMC4912454
- Velagapudi, S. P., Cameron, M. D., Haga, C. L., Rosenberg, L. H., Lafitte, M., Duckett, D. R., Phinney, D. G., Disney, M. D. Design of a small molecule against an oncogenic noncoding RNA Proceedings of the National Academy of Sciences of the United States of America 2016 113:5898-5903 DOI:10.1073/pnas.1523975113 PMID:27170187 PMCID:PMC4889373
- Liu, B., Childs-Disney, J. L., Znosko, B. M., Wang, D., Fallahi, M., Gallo, S. M., Disney, M. D. Analysis of secondary structural elements in human microRNA hairpin precursors BMC Bioinformatics 2016 17 DOI:10.1186/s12859-016-0960-6 PMID:26928172 PMCID:PMC4772329
- Childs-Disney, J. L., Disney, M. D. Small molecule targeting of a microRNA associated with hepatocellular carcinoma ACS Chemical Biology 2016 11:375-380 DOI:10.1021/acschembio.5b00615 PMID:26551630 PMCID:PMC4856042
- Gonzalez, A. L., Rzuczek, S. G., Disney, M. D. Development of pharmacophore models for small molecules targeting RNA: Application to the RNA repeat expansion in myotonic dystrophy type 1 Bioorganic & Medicinal Chemistry Letters 2016 26:5792-5796 DOI:10.1016/j.bmcl.2016.10.037 PMID:27839685
- Rzuczek, S. G., Southern, M. R., Disney, M. D. Studying a drug-like, RNA-focused small molecule library identifies compounds that inhibit RNA toxicity in myotonic dystrophy ACS Chemical Biology 2015 10:2706-2715 DOI:10.1021/acschembio.5b00430 PMID:26414664 PMCID:PMC4903160
- Garbaccio, R. M., Johnson, D. S., Hong, J., Disney, M. D. Recent advances in medicinal chemistry and chemical biology. Preface. Bioorganic & Medicinal Chemistry Letters 2015 25:4714-4714 DOI:10.1016/j.bmcl.2015.08.083
- Yildirim, I., Chakraborty, D., Disney, M. D., Wales, D. J., Schatz, G. C. Computational investigation of RNA CUG repeats responsible for myotonic dystrophy 1 Journal of Chemical Theory and Computation 2015 11:4943-4958 DOI:10.1021/acs.jctc.5b00728 PMID:26500461 PMCID:PMC4606397
- Haga, C. L., Velagapudi, S. P., Strivelli, J. R., Yang, W. Y., Disney, M. D., Phinney, D. G. Small molecule inhibition of miR-544 biogenesis disrupts adaptive responses to hypoxia by modulating ATM-mTOR signaling ACS Chemical Biology 2015 10:2267-2276 DOI:10.1021/acschembio.5b00265 PMID:26181590 PMCID:PMC4876818
- Bernat, V., Disney, M. D. RNA structures as mediators of neurological diseases and as drug targets Neuron 2015 87:28-46 DOI:10.1016/j.neuron.2015.06.012 PMID:26139368 PMCID:PMC4508199
- Park, H., Gonzalez, A. L., Yildirim, I., Tran, T., Lohman, J. R., Fang, P., Guo, M., Disney, M. D. Crystallographic and computational analyses of AUUCU repeating RNA that causes spinocerebellar ataxia type 10 (SCA10) Biochemistry 2015 54:3851-3859 DOI:10.1021/acs.biochem.5b00551 PMID:26039897 PMCID:PMC4876817
- Yang, W. Y., Wilson, H. D., Velagapudi, S. P., Disney, M. D. Inhibition of non-ATG translational events in cells via covalent small molecules targeting RNA Journal of the American Chemical Society 2015 137:5336-5345 DOI:10.1021/ja507448y PMID:25825793 PMCID:PMC4856029
- Velagapudi, S. P., Vummidi, B. R., Disney, M. D. Small molecule chemical probes of microRNA function Current Opinion in Chemical Biology 2015 24:97-103 DOI:10.1016/j.cbpa.2014.10.024 PMID:25500006 PMCID:PMC4382669
- Rzuczek, S. G., Park, H., Disney, M. D. A toxic RNA catalyzes the in cellulo synthesis of its own inhibitor Angewandte Chemie-International Edition 2014 53:10956-10959 DOI:10.1002/anie.201406465 PMID:25164984
- Luo, Y., Disney, M. D. Bottom-up design of small molecules that stimulate exon 10 skipping in mutant MAPT pre-mRNA ChemBioChem 2014 15:2041-2044 DOI:10.1002/cbic.201402069 PMID:25115866 PMCID:PMC4334360
- Disney, M. D. Anti-cancer compound designed rationally TrAC Trends in Analytical Chemistry 2014 56:iv-v DOI:10.1016/j.trac.2014.02.001
- Velagapudi, S. P., Gallo, S. M., Disney, M. D. Sequence-based design of bioactive small molecules that target precursor microRNAs Nature Chemical Biology 2014 10:291-297 DOI:10.1038/nchembio.1452 PMID:24509821 PMCID:PMC3962094
- Tran, T., Childs-Disney, J. L., Liu, B., Guan, L. R., Rzuczek, S., Disney, M. D. Targeting the r(CGG) repeats that cause FXTAS with modularly assembled small molecules and oligonucleotides ACS Chemical Biology 2014 9:904-912 DOI:10.1021/cb400875u PMID:24506227 PMCID:PMC4287843
- Colak, D., Zaninovic, N., Cohen, M. S., Rosenwaks, Z., Yang, W. Y., Gerhardt, J., Disney, M. D., Jaffrey, S. R. Promoter-bound trinucleotide repeat mRNA drives epigenetic silencing in fragile X syndrome Science 2014 343:1002-1005 DOI:10.1126/science.1245831 PMID:24578575 PMCID:PMC4357282
- Childs-Disney, J. L., Yildirim, I., Park, H., Lohman, J. R., Guan, L., Tran, T., Sarkar, P., Schatz, G. C., Disney, M. D. Structure of the myotonic dystrophy type 2 RNA and designed small molecules that reduce toxicity ACS Chemical Biology 2014 9:538-550 DOI:10.1021/cb4007387 PMID:24341895 PMCID:PMC3944380
- Su, Z., Zhang, Y., Gendron, T. F., Bauer, P. O., Chew, J., Yang, W. Y., Fostvedt, E., Jansen-West, K., Belzil, V. V., Desaro, P., Johnston, A., Overstreet, K., et al. Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS Neuron 2014 83:1043-1050 DOI:10.1016/j.neuron.2014.07.041 PMID:25132468 PMCID:PMC4232217
- Disney, M. D., Yildirim, I., Childs-Disney, J. L. Methods to enable the design of bioactive small molecules targeting RNA Organic & Biomolecular Chemistry 2014 12:1029-1039 DOI:10.1039/c3ob42023j PMID:24357181 PMCID:PMC4020623
- Velagapudi, S. P., Disney, M. D. Two-dimensional combinatorial screening enables the bottom-up design of a microRNA-10b inhibitor Chemical Communications 2014 50:3027-3029 DOI:10.1039/c3cc00173c PMID:24503738 PMCID:PMC4040211
- Strack, R. L., Disney, M. D., Jaffrey, S. R. A superfolding Spinach2 reveals the dynamic nature of trinucleotide repeat-containing RNA Nature Methods 2013 10:1219-1224 DOI:10.1038/nmeth.2701 PMID:24162923 PMCID:PMC3852148
- Disney, M. D. Rational design of chemical genetic probes of RNA function and lead therapeutics targeting repeating transcripts Drug Discovery Today 2013 18:1228-1236 DOI:10.1016/j.drudis.2013.07.024 PMID:23939337 PMCID:PMC3849211
- Velagapudi, S. P., Disney, M. D. Defining RNA motif-aminoglycoside interactions via two-dimensional combinatorial screening and structure-activity relationships through sequencing Bioorganic & Medicinal Chemistry 2013 21:6132-6138 DOI:10.1016/j.bmc.2013.04.072 PMID:23719281 PMCID:PMC3789863
- Rzuczek, S. G., Gao, Y., Tang, Z. Z., Thornton, C. A., Kodadek, T., Disney, M. D. Features of modularly assembled compounds that impart bioactivity against an RNA target ACS Chemical Biology 2013 8:2312-2321 DOI:10.1021/cb400265y PMID:24032410 PMCID:PMC3876286
- Guan, L., Disney, M. D. Covalent small-molecule-RNA complex formation enables cellular profiling of small-molecule-RNA interactions Angewandte Chemie-International Edition 2013 52:10010-10013 DOI:10.1002/anie.201301639 PMID:23913698 PMCID:PMC3876275
- Childs-Disney, J. L., Stepniak-Konieczna, E., Tran, T., Yildirim, I., Park, H., Chen, C. Z., Hoskins, J., Southall, N., Marugan, J. J., Patnaik, S., Zheng, W., Austin, C. P., et al. Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules Nature Communications 2013 4:2044 DOI:10.1038/ncomms3044 PMID:23806903 PMCID:PMC3710115
- Yildirim, I., Park, H., Disney, M. D., Schatz, G. C. A dynamic structural model of expanded RNA CAG repeats: A refined X-ray structure and computational investigations using molecular dynamics and umbrella sampling simulations Journal of the American Chemical Society 2013 135:3528-3538 DOI:10.1021/ja3108627 PMID:23441937 PMCID:PMC3625063
- Sellier, C., Freyermuth, F., Tabet, R., Tran, T., He, F., Ruffenach, F., Alunni, V., Moine, H., Thibault, C., Page, A., Tassone, F., Willemsen, R., et al. Sequestration of DROSHA and DGCR8 by expanded CGG RNA repeats alters microRNA processing in Fragile X-associated tremor/ataxia syndrome Cell Reports 2013 3:869-880 DOI:10.1016/j.celrep.2013.02.004 PMID:23478018 PMCID:PMC3639429
- Guan, L. R., Disney, M. D. Small-molecule-mediated cleavage of RNA in living cells Angewandte Chemie-International Edition 2013 52:1462-1465 DOI:10.1002/anie.201206888 PMID:23280953 PMCID:PMC3711026
- Childs-Disney, J. L., Parkesh, R., Nakamori, M., Thornton, C. A., Disney, M. D. Rational design of bioactive, modularly assembled aminoglycosides targeting the RNA that causes myotonic dystrophy type 1 ACS Chemical Biology 2012 7:1984-1993 DOI:10.1021/cb3001606 PMID:23130637 PMCID:PMC3528830
- Velagapudi, S. P., Pushechnikov, A., Labuda, L. P., French, J. M., Disney, M. D. Probing a 2-aminobenzimidazole library for binding to RNA internal loops via two-dimensional combinatorial screening ACS Chemical Biology 2012 7:1902-1909 DOI:10.1021/cb300213g PMID:22958065 PMCID:PMC3500435
- Disney, M. D., Liu, B., Yang, W. Y., Sellier, C., Tran, T., Charlet-Berguerand, N., Childs-Disney, J. L. A small molecule that targets r(CGG)(exp) and improves defects in fragile X-associated tremor ataxia syndrome ACS Chemical Biology 2012 7:1711-1718 DOI:10.1021/cb300135h PMID:22948243 PMCID:PMC3477254
- Childs-Disney, J. L., Hoskins, J., Rzuczek, S. G., Thornton, C. A., Disney, M. D. Rationally designed small molecules targeting the RNA that causes myotonic dystrophy type 1 are potently bioactive ACS Chemical Biology 2012 7:856-862 DOI:10.1021/cb200408a PMID:22332923 PMCID:PMC3356481
- Parkesh, R., Childs-Disney, J. L., Nakamori, M., Kumar, A., Wang, E., Wang, T., Hoskins, J., Tran, T., Housman, D., Thornton, C. A., Disney, M. D. Design of a bioactive small molecule that targets the myotonic dystrophy type 1 RNA via an RNA motif-ligand database and chemical similarity searching Journal of the American Chemical Society 2012 134:4731-4742 DOI:10.1021/ja210088v PMID:22300544 PMCID:PMC3306011
- Kumar, A., Parkesh, R., Sznajder, L. J., Childs-Disney, J. L., Sobczak, K., Disney, M. D. Chemical correction of pre-mRNA splicing defects associated with sequestration of muscleblind-like 1 protein by expanded r(CAG)-containing transcripts ACS Chemical Biology 2012 7:496-505 DOI:10.1021/cb200413a PMID:22252896 PMCID:PMC3306454
- Guan, L. R., Disney, M. D. Recent advances in developing small molecules targeting RNA ACS Chemical Biology 2012 7:73-86 DOI:10.1021/cb200447r PMID:22185671
- Disney, M. D. Studying modification of aminoglycoside antibiotics by resistance-causing enzymes via microarray Methods in Molecular Biology 2012 808:303-320 DOI:10.1007/978-1-61779-373-8_21 PMID:22057534 PMCID:PMC3809005
- Kumar, A., Park, H., Fang, P. F., Parkesh, R., Guo, M., Nettles, K. W., Disney, M. D. Myotonic dystrophy type 1 RNA crystal structures reveal heterogeneous 1 x 1 nucleotide UU internal loop conformations Biochemistry 2011 50:9928-9935 DOI:10.1021/bi2013068 PMID:21988728 PMCID:PMC3218087
- Kumar, A., Fang, P. F., Park, H., Guo, M., Nettles, K. W., Disney, M. D. A crystal structure of a model of the repeating r(CGG) transcript found in fragile X syndrome ChemBioChem 2011 12:2140-2142 DOI:10.1002/cbic.201100337 PMID:21766409 PMCID:PMC3379549
- Childs-Disney, J. L., Tsitovich, P. B., Disney, M. D. Using modularly assembled ligands to bind RNA internal loops separated by different distances ChemBioChem 2011 12:2143-2146 DOI:10.1002/cbic.201100298 PMID:21830289 PMCID:PMC3378996
- Velagapudi, S. P., Seedhouse, S. J., French, J., Disney, M. D. Defining the rna internal loops preferred by benzimidazole derivatives via 2D combinatorial screening and computational analysis Journal of the American Chemical Society 2011 133:10111-10118 DOI:10.1021/ja200212b PMID:21604752 PMCID:PMC3126894
- Tran, T., Disney, M. D. Molecular recognition of 6 '-N-5-hexynoate kanamycin A and RNA 1x1 internal loops containing CA mismatches Biochemistry 2011 50:962-969 DOI:10.1021/bi101724h PMID:21207945 PMCID:PMC3106427
- Parkesh, R., Fountain, M., Disney, M. D. NMR spectroscopy and molecular dynamics simulation of r(CCGCUGCGG)(2) reveal a dynamic UU internal loop found in myotonic dystrophy type 1 Biochemistry 2011 50:599-601 DOI:10.1021/bi101896j PMID:21204525 PMCID:PMC3031998
- Lee, M. M., French, J. M., Disney, M. D. Influencing uptake and localization of aminoglycoside-functionalized peptoids Molecular Biosystems 2011 7:2441-2451 DOI:10.1039/c1mb05074e PMID:21611644 PMCID:PMC3135690
- Tsitovich, P. B., Pushechnikov, A., French, J. M., Disney, M. D. A chemoenzymatic route to diversify aminolgycosides enables a microarray-based method to probe acetyltransferase activity ChemBioChem 2010 11:1656-1660 DOI:10.1002/cbic.201000300 PMID:20629012 PMCID:PMC3016878
- Tran, T., Disney, M. D. Two-dimensional combinatorial screening of a bacterial rRNA A-site-like motif library: defining privileged asymmetric internal loops that bind aminoglycosides Biochemistry 2010 49:1833-1842 DOI:10.1021/bi901998m PMID:20108982 PMCID:PMC2846769
- Seedhouse, S. J., Labuda, L. P., Disney, M. D. The privileged chemical space predictor (PCSP): A computer program that identifies privileged chemical space from screens of modularly assembled chemical libraries Bioorganic & Medicinal Chemistry Letters 2010 20:1338-1343 DOI:10.1016/j.bmcl.2010.01.017 PMID:20097562 PMCID:PMC2825671
- Disney, M. D., Lee, M. M., Pushechnikov, A., Childs-Disney, J. L. The role of flexibility in the rational design of modularly assembled ligands targeting the RNAs that cause the myotonic dystrophies ChemBioChem 2010 11:375-382 DOI:10.1002/cbic.200900716 PMID:20058255 PMCID:PMC2848078
- Velagapudi, S. P., Seedhouse, S. J., Disney, M. D. Structure-activity relationships through sequencing (stARTS) defines optimal and suboptimal RNA motif targets for small molecules Angewandte Chemie-International Edition 2010 49:3816-3818 DOI:10.1002/anie.200907257 PMID:20397174 PMCID:PMC3021749
- Lee, M. M., Childs-Disney, J. L., Pushechnikov, A., French, J. M., Sobczak, K., Thornton, C. A., Disney, M. D. Controlling the specificity of modularly assembled small molecules for RNA via ligand module spacing: Targeting the RNAs that cause myotonic muscular dystrophy Journal of the American Chemical Society 2009 131:17464-17472 DOI:10.1021/ja906877y PMID:19904940 PMCID:PMC2801143
- Paul, D. J., Seedhouse, S. J., Disney, M. D. Two-dimensional combinatorial screening and the RNA Privileged Space Predictor program efficiently identify aminoglycoside-RNA hairpin loop interactions Nucleic Acids Research 2009 37:5894-5907 DOI:10.1093/nar/gkp594 PMID:19726586 PMCID:PMC2761267
- Pushechnikov, A., Lee, M. M., Childs-Disney, J. L., Sobczak, K., French, J. M., Thornton, C. A., Disney, M. D. Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: Application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3 Journal of the American Chemical Society 2009 131:9767-9779 DOI:10.1021/ja9020149 PMID:19552411 PMCID:PMC2731475
- Lee, M. M., Pushechnikov, A., Disney, M. D. Rational and modular design of potent ligands targeting the rna that causes myotonic dystrophy 2 ACS Chemical Biology 2009 4:345-355 DOI:10.1021/cb900025w PMID:19348464 PMCID:PMC2748256
- Labuda, L. P., Pushechnikov, A., Disney, M. D. Small molecule microarrays of RNA-focused peptoids help identify inhibitors of a pathogenic group I intron ACS Chemical Biology 2009 4:299-307 DOI:10.1021/cb800313m PMID:19278238 PMCID:PMC2777746
- Aminova, O., Paul, D. J., Childs-Disney, J. L., Disney, M. D. Two-dimensional combinatorial screening identifies specific 6 '-acylated kanamycin A- and 6 '-acylated neamine-RNA hairpin interactions Biochemistry 2008 47:12670-12679 DOI:10.1021/bi8012615 PMID:18991404 PMCID:PMC2681321
- Barrett, O. J., Pushechnikov, A., Wu, M. L., Disney, M. D. Studying aminoglycoside modification by the acetyltransferase class of resistance-causing enzymes via microarray Carbohydrate Research 2008 343:2924-2931 DOI:10.1016/j.carres.2008.08.018 PMID:18774127 PMCID:PMC2783256
- Disney, M. D., Labuda, L. P., Paul, D. J., Poplawski, S. G., Pushechnikov, A., Tran, T., Velagapudi, S. P., Wu, M., Childs-Disney, J. L. Two-dimensional combinatorial screening identifies specific aminoglycoside - RNA internal loop partners Journal of the American Chemical Society 2008 130:11185-11194 DOI:10.1021/ja803234t PMID:18652457
- Childs-Disney, J. L., Disney, M. D. A simple ligation-based method to increase the information density in sequencing reactions used to deconvolute nucleic acid selections RNA-a Publication of the RNA Society 2008 14:390-394 DOI:10.1261/ma.576908 PMID:18065718 PMCID:PMC2212251
- Childs-Disney, J. L., Wu, M. L., Pushechnikov, A., Aminova, O., Disney, M. D. A small molecule microarray platform to select RNA internal loop-ligand interactions ACS Chemical Biology 2007 2:745-754 DOI:10.1021/6700174r PMID:17975888
- Disney, M. D., Barrett, O. J. An aminoglycoside microarray platform for directly monitoring and studying antibiotic resistance Biochemistry 2007 46:11223-11230 DOI:10.1021/bi701071h PMID:17867707
- Disney, M. D., Childs-Disney, J. L. Using selection to identify and chemical microarray to study the rna internal loops recognized by 6 '-n-acylated kanamycin a ChemBioChem 2007 8:649-656 DOI:10.1002/cbic.200600569 PMID:17394189
- Barrett, O. J., Childs, J. L., Disney, M. D. Chemical microarrays to identify ligands that bind pathogenic cells ChemBioChem 2006 7:1882-1885 DOI:10.1002/cbic.200600260 PMID:17009274
- Brun, M. A., Disney, M. D., Seeberger, P. H. Miniaturization of microwave-assisted carbohydrate functionalization to create oligosaccharide microarrays ChemBioChem 2006 7:421-424 DOI:10.1002/cbic.200500361 PMID:16444768
- Kehr, J. C., Zilliges, Y., Springer, A., Disney, M. D., Ratner, D. D., Bouchier, C., Seeberger, P. H., de Marsac, N. T., Dittmann, E. A mannan binding lectin is involved in cell-cell attachment in a toxic strain of Microcystis aeruginosa Molecular Microbiology 2006 59:893-906 DOI:10.1111/j.1365-2958.2005.05001.x PMID:16420359
- Disney, M. D., Stephenson, R., Wright, T. W., Haidaris, C. G., Turner, D. H., Gigliotti, F. Activity of hoechst 33258 against Pneumocystis carinii f. sp muris, Candida albicans, and Candida dubliniensis Antimicrobial Agents and Chemotherapy 2005 49:1326-1330 DOI:10.1128/aac.49.4.1326-1330.2005 PMID:15793106
- Disney, M. D., Hook, D. F., Namoto, K., Seeberger, P. H., Seebach, D. N-linked glycosylated β-peptides are resistant to degradation by glycoamidase A Chemistry & Biodiversity 2005 2:1624-1634 DOI:10.1002/cbdv.200590132 PMID:17191959
- Disney, M. D., Childs, J. L., Turner, D. H. Hoechst 33258 selectively inhibits group I intron self-splicing by affecting RNA folding ChemBioChem 2004 5:1647-1652 DOI:10.1002/cbic.200400159 PMID:15532034
- Disney, M. D., Seeberger, P. H. The use of carbohydrate microarrays to study carbohydrate-cell interactions and to detect pathogens Chemistry & Biology 2004 11:1701-1707 DOI:10.1016/j.chembiol.2004.10.011 PMID:15610854
- Disney, M. D., Zheng, J., Swager, T. M., Seeberger, P. H. Detection of bacteria with carbohydrate-functionalized fluorescent polymers Journal of the American Chemical Society 2004 126:13343-13346 DOI:10.1021/ja047936i PMID:15479090
- Ratner, D. M., Adams, E. W., Disney, M. D., Seeberger, P. H. Tools for glycomics: mapping interactions of carbohydrates in biological systems ChemBioChem 2004 5:1375-1383 DOI:10.1002/cbic.200400106 PMID:15457538
- Disney, M. D., Seeberger, P. H. Aminoglycoside microarrays to explore interactions of antibiotics with RNAs and proteins Chemistry-a European Journal 2004 10:3308-3314 DOI:10.1002/chem.200306017 PMID:15224340
- Mathews, D. H., Disney, M. D., Childs, J. L., Schroeder, S. J., Zuker, M., Turner, D. H. Incorporating chemical modification constraints into a dynamic programming algorithm for prediction of RNA secondary structure Proceedings of the National Academy of Sciences of the United States of America 2004 101:7287-7292 DOI:10.1073/pnas.0401799101 PMID:15123812 PMCID:PMC409911
- Disney, M. D., Magnet, S., Blanchard, J. S., Seeberger, P. H. Aminoglycoside microarrays to study antibiotic resistance Angewandte Chemie-International Edition 2004 43:1591-1594 DOI:10.1002/anie.200353236 PMID:15022242
- Disney, M. D., Childs, J. L., Turner, D. H. New approaches to targeting RNA with oligonucleotides: inhibition of group I intron self-splicing Biopolymers 2004 73:151-161 DOI:10.1002/bip.10520 PMID:14691946
- Du, H., Disney, M. D., Miller, B. L., Krauss, T. D. Hybridization-based unquenching of DNA hairpins on au surfaces: prototypical "molecular beacon" biosensors Journal of the American Chemical Society 2003 125:4012-4013 DOI:10.1021/ja0290781 PMID:12670198
- Disney, M. D., Haidaris, C. G., Turner, D. H. Uptake and antifungal activity of oligonucleotides in Candida albicans Proceedings of the National Academy of Sciences of the United States of America 2003 100:1530-1534 DOI:10.1073/pnas.0337462100 PMID:12552085 PMCID:PMC149866
- Childs, J. L., Disney, M. D., Turner, D. H. Oligonucleotide directed misfolding of RNA inhibits Candida albicans group I intron splicing Proceedings of the National Academy of Sciences of the United States of America 2002 99:11091-11096 DOI:10.1073/pnas.172391199 PMID:12169671 PMCID:PMC123215
- Disney, M. D., Turner, D. H. Molecular recognition by the Candida albicans group I intron: tertiary interactions with an imino G.A pair facilitate binding of the 5' exon and lower the KM for guanosine Biochemistry 2002 41:8113-8119 DOI:10.1021/bi020102x PMID:12069604
- Disney, M. D., Matray, T., Gryaznov, S. M., Turner, D. H. Binding enhancement by tertiary interactions and suicide inhibition of a Candida albicans group I intron by phosphoramidate and 2 '-o-methyl hexanucleotides Biochemistry 2001 40:6520-6526 DOI:10.1021/bi002009j PMID:11371215
- Disney, M. D., Haidaris, C. G., Turner, D. H. Recognition elements for 5 ' exon substrate binding to the Candida albicans group I intron Biochemistry 2001 40:6507-6519 DOI:10.1021/bi002008r PMID:11371214
- Disney, M. D., Gryaznov, S. M., Turner, D. H. Contributions of individual nucleotides to tertiary binding of substrate by a Pneumocystis carinii group I intron Biochemistry 2000 39:14269-14278 DOI:10.1021/bi001345x PMID:11087376
- Disney, M. D., Testa, S. M., Turner, D. H. Targeting a pneumocystis carinii group i intron with methylphosphonate oligonucleotides: Backbone charge is not required for binding or reactivity Biochemistry 2000 39:6991-7000 DOI:10.1021/bi992937m PMID:10841781
- Testa, S. M., Disney, M. D., Turner, D. H., Kierzek, R. Thermodynamics of RNA-RNA duplexes with 2-or 4-thiouridines: implications for antisense design and targeting a group I intron Biochemistry 1999 38:16655-16662 DOI:10.1021/bi991187d PMID:10600128
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chapter
- Childs-Disney, J. L., Disney, M. D. Approaches to validate and manipulate RNA targets with small molecules in cells Annual Review of Pharmacology and Toxicology. 123-140. 2016 DOI:10.1146/annurev-pharmtox-010715-103910 PMID:26514201 PMCID:PMC4876813