| [1] |
Warner KD, Hajdin CE, Weeks KM. 2018. Principles for targeting RNA with drug-like small molecules. |
| [2] |
The ENCODE Project Consortium. 2012. An integrated encyclopedia of DNA elements in the human genome. |
| [3] |
Saraswat A, Patki M, Fu Y, Barot S, Dukhande VV, et al. 2020. Nanoformulation of PROteolysis TArgeting Chimera targeting 'undruggable' c-Myc for the treatment of pancreatic cancer. |
| [4] |
ClinicalTrials.gov. 2026. Search of RNA-targeting therapeutics clinical trials. U.S. National Library of Medicine. https://clinicaltrials.gov/ (accessed on 17 May 2026) |
| [5] |
Khorkova O, Stahl J, Joji A, Volmar CH, Wahlestedt C. 2023. Amplifying gene expression with RNA-targeted therapeutics. |
| [6] |
Childs-Disney JL, Tsitovich PB, Disney MD. 2011. Using modularly assembled ligands to bind RNA internal loops separated by different distances. |
| [7] |
Menduti G, Rasà DM, Stanga S, Boido M. 2020. Drug screening and drug repositioning as promising therapeutic approaches for spinal muscular atrophy treatment. |
| [8] |
Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, et al. 2018. Discovery of risdiplam, a selective survival of motor neuron-2 (SMN2) gene splicing modifier for the treatment of spinal muscular atrophy (SMA). |
| [9] |
Dhillon S. 2020. Risdiplam: first approval. |
| [10] |
Xiao L, Kang S, Djordjevic D, Gonorazky H, Chiang J, et al. 2023. Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study. |
| [11] |
Childs-Disney JL, Yang X, Gibaut QMR, Tong Y, Batey RT, et al. 2022. Targeting RNA structures with small molecules. |
| [12] |
Tong Y, Lee Y, Liu X, Childs-Disney JL, Suresh BM, et al. 2023. Programming inactive RNA-binding small molecules into bioactive degraders. |
| [13] |
Tong Y, Gibaut QMR, Rouse W, Childs-Disney JL, Suresh BM, et al. 2022. Transcriptome-wide mapping of small-molecule RNA-binding sites in cells informs an isoform-specific degrader of QSOX1 mRNA. |
| [14] |
Xiong W, Liu X, Qi Q, Li Y, Huang S, et al. 2025. Bioorthogonal RNase L recruitment enables targeted inducible degradation of SARS-CoV-2 RNA. |
| [15] |
Bian T, Gao S, Sun X, Zhou S, Pei Y, et al. 2025. Single-molecule detection on intrastrand interactions among G4 clusters. |
| [16] |
Zhao L, Zhao J, Zhong K, Tong A, Jia D. 2022. Targeted protein degradation: mechanisms, strategies and application. |
| [17] |
Fang Y, Wang J, Zhao M, Zheng Q, Ren C, et al. 2022. Progress and challenges in targeted protein degradation for neurodegenerative disease therapy. |
| [18] |
Sakamoto KM, Kim KB, Kumagai A, Mercurio F, Crews CM, et al. 2001. Protacs: chimeric molecules that target proteins to the Skp1–Cullin–F box complex for ubiquitination and degradation. |
| [19] |
Domostegui A, Nieto-Barrado L, Perez-Lopez C, Mayor-Ruiz C. 2022. Chasing molecular glue degraders: screening approaches. |
| [20] |
Wu S, Xiao H, Sun Q. 2021. New approaches for small molecule-induced protein degradation. |
| [21] |
Henning NJ, Boike L, Spradlin JN, Ward CC, Liu G, et al. 2022. Deubiquitinase-targeting chimeras for targeted protein stabilization. |
| [22] |
Chen P-H, Hu Z, An E, Okeke I, Zheng S, et al. 2021. Modulation of phosphoprotein activity by phosphorylation targeting chimeras (PhosTACs). |
| [23] |
Costales MG, Matsumoto Y, Velagapudi SP, Disney MD. 2018. Small molecule targeted recruitment of a nuclease to RNA. |
| [24] |
Costales MG, Suresh B, Vishnu K, Disney MD. 2019. Targeted degradation of a hypoxia-associated non-coding RNA enhances the selectivity of a small molecule interacting with RNA. |
| [25] |
Costales MG, Aikawa H, Li Y, Childs-Disney JL, Abegg D, et al. 2020. Small-molecule targeted recruitment of a nuclease to cleave an oncogenic RNA in a mouse model of metastatic cancer. |
| [26] |
Guan L, Disney MD. 2013. Small-molecule-mediated cleavage of RNA in living cells. |
| [27] |
Haj-Yahia S, Nandi A, Benhamou RI. 2023. Targeted degradation of structured RNAs via ribonuclease-targeting chimeras (RiboTacs). |
| [28] |
Kubota K, Nakahara K, Ohtsuka T, Yoshida S, Kawaguchi J, et al. 2004. Identification of 2'-phosphodiesterase, which plays a role in the 2-5A system regulated by interferon. |
| [29] |
Kargbo RB. 2021. RIBOTACs: small molecules selectively destroy cancer-associated RNA. |
| [30] |
Liang X-H, Sun H, Shen W, Crooke ST. 2015. Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages. |
| [31] |
Wang W, He S, Dong G, Sheng C. 2022. Nucleic-acid-based targeted degradation in drug discovery. |
| [32] |
Meyer SM, Williams CC, Akahori Y, Tanaka T, Aikawa H, et al. 2020. Small molecule recognition of disease-relevant RNA structures. |
| [33] |
Gao X, Wu Z. 2025. IRES-mediated translation: expanding the toolkits of RNA therapy. |
| [34] |
Khaskia E, Dahatonde D, Benhamou RI. 2025. RNA G-quadruplex RIBOTAC-mediated targeted degradation of lncRNA TERRA. |
| [35] |
Cullen BR. 2004. Transcription and processing of human microRNA precursors. |
| [36] |
Ha M, Kim VN. 2014. Regulation of microRNA biogenesis. |
| [37] |
Bohnsack MT, Czaplinski K, Gorlich D. 2004. Exportin 5 is a RanGTP-dependent dsRNA-binding protein that mediates nuclear export of pre-miRNAs. |
| [38] |
Starega-Roslan J, Galka-Marciniak P, Krzyzosiak WJ. 2015. Nucleotide sequence of miRNA precursor contributes to cleavage site selection by Dicer. |
| [39] |
Childs-Disney JL, Disney MD. 2015. Small molecule targeting of a microRNA associated with hepatocellular carcinoma. |
| [40] |
Costales MG, Haga CL, Velagapudi SP, Childs-Disney JL, Phinney DG, et al. 2017. Small molecule inhibition of microRNA-210 reprograms an oncogenic hypoxic circuit. |
| [41] |
Gilam A, Conde J, Weissglas-Volkov D, Oliva N, Friedman E, et al. 2016. Local microRNA delivery targets Palladin and prevents metastatic breast cancer. |
| [42] |
Velagapudi SP, Gallo SM, Disney MD. 2014. Sequence-based design of bioactive small molecules that target precursor microRNAs. |
| [43] |
Velagapudi SP, Cameron MD, Haga CL, Rosenberg LH, Lafitte M, et al. 2016. Design of a small molecule against an oncogenic noncoding RNA. |
| [44] |
Li Y, Disney MD. 2018. Precise small molecule degradation of a noncoding RNA identifies cellular binding sites and modulates an oncogenic phenotype. |
| [45] |
Kwok CK, Merrick CJ. 2017. G-quadruplexes: Prediction, characterization, and biological application. |
| [46] |
Rosenberg M, Paterson BM. 1979. Efficient cap-dependent translation of polycistronic prokaryotic mRNAs is restricted to the first gene in the operon. |
| [47] |
Davila-Calderon J, Patwardhan NN, Chiu LY, Sugarman A, Cai Z, et al. 2020. IRES-targeting small molecule inhibits enterovirus 71 replication via allosteric stabilization of a ternary complex. |
| [48] |
Péladeau C, Jasmin BJ. 2021. Targeting IRES-dependent translation as a novel approach for treating Duchenne muscular dystrophy. |
| [49] |
Kozak M, Shatkin AJ. 1978. Migration of 40 S ribosomal subunits on messenger RNA in the presence of edeine. |
| [50] |
Nanbru C, Lafon I, Audigier S, Gensac MC, Vagner S, et al. 1997. Alternative translation of the proto-oncogene c-myc by an internal ribosome entry site. |
| [51] |
Lee ASY, Kranzusch PJ, Cate JHD. 2015. eIF3 targets cell-proliferation messenger RNAs for translational activation or repression. |
| [52] |
Lee AS, Kranzusch PJ, Doudna JA, Cate JHD. 2016. eIF3d is an mRNA cap-binding protein that is required for specialized translation initiation. |
| [53] |
Ding Y, Fei Y, Lu B. 2020. Emerging new concepts of degrader technologies. |
| [54] |
Lee Y, Rio DC. 2015. Mechanisms and regulation of alternative pre-mRNA splicing. |
| [55] |
Ule J, Blencowe BJ. 2019. Alternative splicing regulatory networks: functions, mechanisms, and evolution. |
| [56] |
Sazani P, Kole R. 2003. Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. |
| [57] |
Zheng S, Chen Y, Donahue CP, Wolfe MS, Varani G. 2009. Structural basis for stabilization of the tau pre-mRNA splicing regulatory element by novantrone (mitoxantrone). |
| [58] |
Messina S, Sframeli M. 2020. New treatments in spinal muscular atrophy: positive results and new challenges. |
| [59] |
Wang Z-F, Ursu A, Childs-Disney JL, Guertler R, Yang WY, et al. 2019. The hairpin form of r(G4C2) exp in c9ALS/FTD is repeat-associated non-ATG translated and a target for bioactive small molecules. |
| [60] |
Bush JA, Aikawa H, Fuerst R, Li Y, Ursu A, et al. 2021. Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models. |
| [61] |
Kumar V, Hasan GM, Hassan MI. 2017. Unraveling the role of RNA mediated toxicity of C9orf72 repeats in C9-FTD/ALS. |
| [62] |
Jahromi AH, Fu Y, Miller KA, Nguyen L, Luu LM, et al. 2013. Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1. |
| [63] |
Jog SP, Paul S, Dansithong W, Tring S, Comai L, et al. 2012. RNA splicing is responsive to MBNL1 dose. |
| [64] |
Angelbello AJ, DeFeo ME, Glinkerman CM, Boger DL, Disney MD. 2020. Precise targeted cleavage of a r(CUG) repeat expansion in cells by using a small-molecule-deglycobleomycin conjugate. |
| [65] |
La Spada AR, Taylor JP. 2010. Repeat expansion disease: progress and puzzles in disease pathogenesis. |
| [66] |
Gendron TF, Belzil VV, Zhang YJ, Petrucelli L. 2014. Mechanisms of toxicity in C9FTLD/ALS. |
| [67] |
Angelbello AJ, Disney MD. 2018. Bleomycin can cleave an oncogenic noncoding RNA. |
| [68] |
Benhamou RI, Abe M, Choudhary S, Meyer SM, Angelbello AJ, et al. 2020. Optimization of the linker domain in a dimeric compound that degrades an r(CUG) repeat expansion in cells. |
| [69] |
Disney MD, Angelbello AJ. 2016. Rational design of small molecules targeting oncogenic noncoding RNAs from sequence. |
| [70] |
Garner AL, Lorenz DA, Sandoval J, Gallagher EE, Kerk SA, et al. 2019. Tetracyclines as inhibitors of pre-microRNA maturation: a disconnection between RNA binding and inhibition. |
| [71] |
Pomplun S, Gates ZP, Zhang G, Quartararo AJ, Pentelute BL. 2020. Discovery of nucleic acid binding molecules from combinatorial biohybrid nucleobase peptide libraries. |
| [72] |
Vo DD, Tran TPA, Staedel C, Benhida R, Darfeuille F, et al. 2016. Oncogenic MicroRNAs biogenesis as a drug target: structure–activity relationship studies on new aminoglycoside conjugates. |
| [73] |
Disney MD, Childs JL, Turner DH. 2004. Hoechst 33258 selectively inhibits group I intron self-splicing by affecting RNA folding. |
| [74] |
Velagapudi SP, Seedhouse SJ, French J, Disney MD. 2011. Defining the RNA internal loops preferred by benzimidazole derivatives via 2D combinatorial screening and computational analysis. |
| [75] |
Tran T, Disney MD. 2012. Identifying the preferred RNA motifs and chemotypes that interact by probing millions of combinations. |
| [76] |
Parkesh R, Childs-Disney JL, Nakamori M, Kumar A, Wang E, et al. 2012. Design of a bioactive small molecule that targets the myotonic dystrophy type 1 RNA via an RNA motif-ligand database and chemical similarity searching. |
| [77] |
Guan L, Disney MD. 2013. Covalent small-molecule-RNA complex formation enables cellular profiling of small-molecule-RNA interactions. |
| [78] |
Disney MD, Liu B, Yang WY, Sellier C, Tran T, et al. 2012. A small molecule that targets r(CGG)(exp) and improves defects in fragile X-associated tremor ataxia syndrome. |
| [79] |
Su Z, Zhang Y, Gendron TF, Bauer PO, Chew J, et al. 2014. Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS. |
| [80] |
Rzuczek SG, Southern MR, Disney MD. 2015. Studying a drug-like, RNA-focused small molecule library identifies compounds that inhibit RNA toxicity in myotonic dystrophy. |
| [81] |
Costales MG, Hoch DG, Abegg D, Childs-Disney JL, Velagapudi SP, et al. 2019. A designed small molecule inhibitor of a non-coding RNA sensitizes HER2 negative cancers to Herceptin. |
| [82] |
Suresh BM, Li W, Zhang P, Wang KW, Yildirim I, et al. 2020. A general fragment-based approach to identify and optimize bioactive ligands targeting RNA. |
| [83] |
Haniff HS, Knerr L, Liu X, Crynen G, Boström J, et al. 2020. Design of a small molecule that stimulates vascular endothelial growth factor A enabled by screening RNA fold-small molecule interactions. |
| [84] |
Wagner-Griffin S, Abe M, Benhamou RI, Angelbello AJ, Vishnu K, et al. 2021. A druglike small molecule that targets r(CCUG) repeats in myotonic dystrophy type 2 facilitates degradation by RNA quality control pathways. |
| [85] |
Angelbello AJ, Benhamou RI, Rzuczek SG, Choudhary S, Tang Z, et al. 2021. A small molecule that binds an RNA repeat expansion stimulates its decay via the exosome complex. |
| [86] |
Tong Y, Zhang P, Yang X, Liu X, Zhang J, et al. 2024. Decreasing the intrinsically disordered protein α-synuclein levels by targeting its structured mRNA with a ribonuclease-targeting chimera. |
| [87] |
Meyer SM, Tanaka T, Zanon PRA, Baisden JT, Abegg D, et al. 2022. DNA-encoded library screening to inform design of a ribonuclease targeting chimera (RiboTAC). |
| [88] |
Liu X, Haniff HS, Childs-Disney JL, Shuster A, Aikawa H, et al. 2020. Targeted degradation of the oncogenic MicroRNA 17-92 cluster by structure-targeting ligands. |
| [89] |
Disney MD, Childs‐Disney JL. 2007. Using selection to identify and chemical microarray to study the RNA internal loops recognized by 6'-N-acylated Kanamycin A. |
| [90] |
Disney MD, Labuda LP, Paul DJ, Poplawski SG, Pushechnikov A, et al. 2008. Two-dimensional combinatorial screening identifies specific aminoglycoside–RNA internal loop partners. |
| [91] |
Lee MM, Pushechnikov A, Disney MD. 2009. Rational and modular design of potent ligands targeting the RNA that causes myotonic dystrophy 2. |
| [92] |
Kumar A, Parkesh R, Sznajder LJ, Childs-Disney JL, Sobczak K, et al. 2012. Chemical correction of pre-mRNA splicing defects associated with sequestration of muscleblind-like 1 protein by expanded r(CAG)-containing transcripts. |
| [93] |
Yang WY, Gao R, Southern M, Sarkar PS, Disney MD. 2016. Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10. |
| [94] |
Childs-Disney JL, Stepniak-Konieczna E, Tran T, Yildirim I, Park H, et al. 2013. Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules. |
| [95] |
Haga CL, Velagapudi SP, Strivelli JR, Yang W-Y, Disney MD, et al. 2015. Small molecule inhibition of miR-544 biogenesis disrupts adaptive responses to hypoxia by modulating ATM-mTOR signaling. |
| [96] |
Hoskins JW, Ofori LO, Chen CZ, Kumar A, Sobczak K, et al. 2014. Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects. |
| [97] |
Velagapudi SP, Costales MG, Vummidi BR, Nakai Y, Angelbello AJ, et al. 2018. Approved anti-cancer drugs target oncogenic non-coding RNAs. |
| [98] |
Haniff HS, Tong Y, Liu X, Chen JL, Suresh BM, et al. 2020. Targeting the SARS-CoV-2 RNA genome with small molecule binders and ribonuclease targeting chimera (RIBOTAC) degraders. |
| [99] |
Ursu A, Baisden JT, Bush JA, Taghavi A, Choudhary S, et al. 2021. A small molecule exploits hidden structural features within the RNA repeat expansion that causes c9ALS/FTD and rescues pathological hallmarks. |
| [100] |
Suresh BM, Akahori Y, Taghavi A, Crynen G, Gibaut QMR, et al. 2022. Low-molecular weight small molecules can potently bind RNA and affect oncogenic pathways in cells. |
| [101] |
Pernodet N, Hermetet F, Adami P, Vejux A, Descotes F, et al. 2012. High expression of QSOX1 reduces tumorogenesis, and is associated with a better outcome for breast cancer patients. |
| [102] |
Suresh BM, Tong Y, Abegg D, Adibekian A, Childs-Disney JL, et al. 2023. Altering the cleaving effector in chimeric molecules that target RNA enhances cellular selectivity. |
| [103] |
Wirth B. 2000. An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA). |
| [104] |
Palacino J, Swalley SE, Song C, Cheung AK, Shu L, et al. 2015. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. |
| [105] |
Mercure S, Montplaisir S, Lemay G. 1993. Correlation between the presence of a self-splicing intron in the 25S rDNA of C.albicans and strains susceptibility to 5-fluorocytosine. |
| [106] |
Bubenik JL, Scotti MM, Swanson MS. 2024. Therapeutic targeting of RNA for neurological and neuromuscular disease. |
| [107] |
Carter AP, Clemons WM, Brodersen DE, Morgan-Warren RJ, Wimberly BT, et al. 2000. Functional insights from the structure of the 30S ribosomal subunit and its interactions with antibiotics. |
| [108] |
Moazed D, Noller HF. 1987. Interaction of antibiotics with functional sites in 16S ribosomal RNA. |
| [109] |
Recht MI, Fourmy D, Blanchard SC, Dahlquist KD, Puglisi JD. 1996. RNA sequence determinants for aminoglycoside binding to an a-site rRNA model oligonucleotide. |
| [110] |
Lee MM, Childs-Disney JL, Pushechnikov A, French JM, Sobczak K, et al. 2009. Controlling the specificity of modularly assembled small molecules for RNA via ligand module spacing: targeting the RNAs that cause myotonic muscular dystrophy. |
| [111] |
Silva JG, Carvalho I. 2007. New insights into aminoglycoside antibiotics and derivatives. |
| [112] |
Labuda LP, Pushechnikov A, Disney MD. 2009. Small molecule microarrays of RNA-focused peptoids help identify inhibitors of a pathogenic group I intron. |
| [113] |
Li H, Dong J, Cai M, Xu Z, Cheng X-D, et al. 2021. Protein degradation technology: a strategic paradigm shift in drug discovery. |
| [114] |
Mikutis S, Rebelo M, Yankova E, Gu M, Tang C, et al. 2023. Proximity-induced nucleic acid degrader (PINAD) approach to targeted RNA degradation using small molecules. |
| [115] |
Bonet-Aleta J, Maehara T, Craig BA, Bernardes GJL. 2024. Small molecule RNA degraders. |
| [116] |
Ursu A, Childs-Disney JL, Andrews RJ, O’Leary CA, Meyer SM, et al. 2020. Design of small molecules targeting RNA structure from sequence. |
| [117] |
Disney MD. 2019. Targeting RNA with small molecules to capture opportunities at the intersection of chemistry, biology, and medicine. |
| [118] |
Childs-Disney JL, Tran T, Vummidi BR, Velagapudi SP, Haniff HS, et al. 2018. A massively parallel selection of small molecule-RNA motif binding partners informs design of an antiviral from sequence. |
| [119] |
Garner AL, Lorenz DA, Gallagher EE. 2019. A click chemistry assay to identify natural product ligands for pre-microRNAs. |
| [120] |
Borgelt L, Haacke N, Lampe P, Qiu X, Gasper R, et al. 2022. Small-molecule screening of ribonuclease L binders for RNA degradation. |
| [121] |
Malathi K, Paranjape JM, Ganapathi R, Silverman RH. 2004. HPC1/RNASEL mediates apoptosis of prostate cancer cells treated with 2', 5'-oligoadenylates, topoisomerase I inhibitors, and tumor necrosis factor-related apoptosis-inducing ligand. |
| [122] |
Loureiro RJ, Maiti S, Mondal K, Mukherjee S, Bujnicki JM. 2025. Modeling flexible RNA 3D structures and RNA-protein complexes. |
| [123] |
Dilliard SA, Cheng Q, Siegwart DJ. 2021. On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles. |
| [124] |
Rzuczek SG, Gao Y, Tang ZZ, Thornton CA, Kodadek T, et al. 2013. Features of modularly assembled compounds that impart bioactivity against an RNA target. |
| [125] |
Sadler AJ, Williams BRG. 2008. Interferon-inducible antiviral effectors. |
| [126] |
Rebouillat D, Hovanessian AG. 1999. The human 2', 5'-oligoadenylate synthetase family: Interferon-induced proteins with unique enzymatic properties. |
| [127] |
Takenaka Y, Yamada A, Tomioka Y, Akiyama Y, Ivanov P. 2025. RNase L produces tRNA-derived RNAs that contribute to translation inhibition. |
| [128] |
Tang Z, Hegde S, Hao S, Selvaraju M, Qiu J, et al. 2025. Chemical-guided SHAPE sequencing (cgSHAPE-seq) informs the binding site of RNA-degrading chimeras targeting SARS-CoV-2 5' untranslated region. |
| [129] |
Thakur CS, Jha BK, Dong B, Das Gupta J, Silverman KM, et al. 2007. Small-molecule activators of RNase L with broad-spectrum antiviral activity. |
| [130] |
Thakur CS, Xu Z, Wang Z, Novince Z, Silverman RH. 2005. A convenient and sensitive fluorescence resonance energy transfer assay for RNase L and 2',5' oligoadenylates. |
| [131] |
Zuker M. 2003. Mfold web server for nucleic acid folding and hybridization prediction. |
| [132] |
Rzuczek SG, Colgan LA, Nakai Y, Cameron MD, Furling D, et al. 2017. Precise small-molecule recognition of a toxic CUG RNA repeat expansion. |
| [133] |
Xu W, Biswas J, Singer RH, Rosbash M. 2022. Targeted RNA editing: Novel tools to study post-transcriptional regulation. |
| [134] |
Meyer SM, Kovachka S, Wang T, Cameron MD, Childs-Disney JL, et al. 2025. Linker optimization enhances the potency of ribonuclease-targeting chimeras in cancer models. |
| [135] |
Jiao Z, Song C, Sha X, Chen Y, Xing Y, et al. 2025. Targeted RNA degradation via LipoSM-RiboTAC nanoparticles: a versatile platform for cancer therapy. Journal of the American Chemical Society 147:42209−42220 |
| [136] |
Fang Y, Wu Q, Wang F, Liu Y, Zhang H, et al. 2025. Aptamer-RIBOTAC strategy enabling tumor-dpecific targeted degradation of microRNA for precise cancer therapy. |
| [137] |
Ellenbroek BD, Kahler JP, Evers SR, Pomplun SJ. 2024. Synthetic peptides: promising modalities for the targeting of disease-related nucleic acids. |
| [138] |
Morishita EC, Nakamura S. 2024. Recent applications of artificial intelligence in RNA-targeted small molecule drug discovery. |
| [139] |
Mullowney MW, Duncan KR, Elsayed SS, Garg N, van der Hooft JJJ, et al. 2023. Artificial intelligence for natural product drug discovery. |
| [140] |
Sato K, Hamada M. 2023. Recent trends in RNA informatics: a review of machine learning and deep learning for RNA secondary structure prediction and RNA drug discovery. |
| [141] |
Disney MD, Winkelsas AM, Velagapudi SP, Southern M, Fallahi M. 2016. Inforna 2.0: a platform for the sequence-based design of small molecules targeting structured RNAs. |
| [142] |
de Lajarte AA, Taillades Y, Aruda J, Bongrand P, Wightman FF, et al. 2026. Diverse database and machine learning model to narrow the generalization gap in RNA structure prediction. |
| [143] |
Wang Y, Tang S-C. 2022. The race to develop oral SERDs and other novel estrogen receptor inhibitors: recent clinical trial results and impact on treatment options. |
| [144] |
Ravegnini E, Trabocchi A, Lenci E. 2025. Small-molecule RNA ligands: a patent review (2018–2024). |
| [145] |
Su X, Tong Y, Zanon PRA, Wang J, Tanaka T, et al. 2024. Unbiased RNA degrader identification uncovers an LC3B-recruiting chimera for COL15A1 mRNA degradation. |
| [146] |
Su Y, Hammond MC. 2020. RNA-based fluorescent biosensors for live cell imaging of small molecules and RNAs. |
| [147] |
Seok H, Lee H, Jang E-S, Chi SW. 2018. Evaluation and control of miRNA-like off-target repression for RNA interference. |
| [148] |
Lee YT. 2024. Nexus between RNA conformational dynamics and functional versatility. |
| [149] |
Khaskia E, Benhamou RI. 2025. Leveraging RIBOTAC technology: fluorescent RNase L probes for live-cell imaging and function analysis. |