Consortium, EP et al. Identification and analysis of functional elements in 1% of the human genome by the ENCODE pilot project. Nature 447799–816 (2007).
Santos, R. et al. A comprehensive map of molecular drug targets. Nat. Rev. Drug Discov. 1619–34 (2017).
Warner, KD et al. Principles for targeting RNA with drug-like small molecules. Nat. Rev. Drug Discov. 17547–558 (2018).
Zhang, F. & Lupski, JR Non-coding genetic variants in human disease. Hum. Mol. Genet. 24R102 – R110 (2015).
Lee, JT Epigenetic regulation by long noncoding RNAs. Science 3381435–1439 (2012).
Wutz, A. et al. Chromosomal silencing and localization are mediated by different domains of Xist RNA. Nat. Genet. 30167–174 (2002).
Rizvi, NF & Smith, GF RNA as a small molecule druggable target. Bioorg. Med. Chem. Lett. 275083–5088 (2017).
Disney, MD et al. Drugging the RNA world. Cold Spring Harb. Perspective. Biol. 10a034769 (2018).
Howe, JA et al. Selective small-molecule inhibition of an RNA structural element. Nature 526672–677 (2015).
Palacino, J. et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat. Chem. Biol. 11511–517 (2015).
Disney, MD et al. Inforna 2.0: a platform for the sequence-based design of small molecules targeting structured RNAs. ACS Chem. Biol. 111720–1728 (2016).
Allen Annis, D. et al. An affinity selection-mass spectrometry method for the identification of small molecule ligands from self-encoded combinatorial libraries — Discovery of a novel antagonist of E-coli dihydrofolate reductase. Int. J. Mass Spectrom. 23877–83 (2004).
Rizvi, NF et al. Discovery of selective RNA-binding small molecules by affinity-selection mass spectrometry. ACS Chem. Biol. 13820–831 (2018).
Rizvi, NF et al. Targeting RNA with small molecules: identification of selective, RNA-binding small molecules occupying drug-like chemical space. SLAS Discov. 25384–396 (2020).
Cifuentes-Rojas, C. et al. Regulatory interactions between RNA and polycomb repressive complex 2. Mol. Cell 55171–185 (2014).
Chillon, I. et al. Native purification and analysis of long RNAs. Enzymol Methods. 5583–37 (2015).
Lipinski, CA Lead- and drug-like compounds: the rule-of-five revolution. Drug Discov. Today Technol. 1337–341 (2004).
Monfort, A. et al. Identification of Spen as a crucial factor for Xist function through forward genetic screening in haploid embryonic stem cells. Cell Rep. 12554–561 (2015).
Lee, MK et al. A novel small-molecule binds to the influenza A virus RNA promoter and inhibits viral replication. Chem. Common. 50368–370 (2014).
Ogawa, Y. et al. Intersection of the RNA interference and X-inactivation pathways. Science 3201336–1341 (2008).
Zhao, J. et al. Genome-wide identification of polycomb-associated RNAs by RIP – seq. Mol. Cell 40939–953 (2010).
Patil, DP et al. m6RNA methylation promotes XIST-mediated transcriptional repression. Nature 537369–373 (2016).
Sunwoo, H. et al. Repeat E anchors Xist RNA to the inactive X chromosomal compartment through CDKN1A-interacting protein (CIZ1). Proc. Natl Acad. Ski. USA 11410654–10659 (2017).
Jeon, Y. & Lee, JT YY1 tethers Xist RNA to the inactive X nucleation center. Cell 146119–133 (2011).
Colognori, D. et al. Xist deletional analysis reveals an interdependence between Xist RNA and polycomb complexes for spreading along the inactive X. Mol. Cell 74101–117 and 110 (2019).
Liu, F. et al. Visualizing the secondary and tertiary architectural domains of lncRNA RepA. Nat. Chem. Biol. 13282–289 (2017).
Kikhney, AG & Svergun, DI A practical guide to small angle X-ray scattering (SAXS) of flexible and intrinsically disordered proteins. FEBS Lett. 5892570–2577 (2015).
Kim, DN et al. Zinc-finger protein CNBP alters the 3-D structure of lncRNA Braveheart in solution. Nat. Common. 11148 (2020).
Carrette, LLG et al. A mixed modality approach towards Xi reactivation for Rett syndrome and other X-linked disorders. Proc. Natl Acad. Ski. USA 1151715124115 (2017).
Stelzer, AC et al. Discovery of selective bioactive small molecules by targeting an RNA dynamic ensemble. Nat. Chem. Biol. 7553–559 (2011).