{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T13:30:24Z","timestamp":1792762224127,"version":"4.5.0"},"reference-count":205,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2023,11,23]],"date-time":"2023-11-23T00:00:00Z","timestamp":1700697600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Genet."],"abstract":"<jats:p>Awareness of RNA-based therapies has increased after the widespread adoption of mRNA vaccines against SARS-CoV-2 during the COVID-19 pandemic. These mRNA vaccines had a significant impact on reducing lung disease and mortality. They highlighted the potential for rapid development of RNA-based therapies and advances in nanoparticle delivery systems. Along with the rapid advancement in RNA biology, including the description of noncoding RNAs as major products of the genome, this success presents an opportunity to highlight the potential of RNA as a therapeutic modality. Here, we review the expanding compendium of RNA-based therapies, their mechanisms of action and examples of application in the lung. The airways provide a convenient conduit for drug delivery to the lungs with decreased systemic exposure. This review will also describe other delivery methods, including local delivery to the pleura and delivery vehicles that can target the lung after systemic administration, each providing access options that are advantageous for a specific application. We present clinical trials of RNA-based therapy in lung disease and potential areas for future directions. This review aims to provide an overview that will bring together researchers and clinicians to advance this burgeoning field.<\/jats:p>","DOI":"10.3389\/fgene.2023.1281538","type":"journal-article","created":{"date-parts":[[2023,11,23]],"date-time":"2023-11-23T11:17:14Z","timestamp":1700738234000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":13,"title":["Unlocking the potential of RNA-based therapeutics in the lung: current status and future directions"],"prefix":"10.3389","volume":"14","author":[{"given":"H. S. Jeffrey","family":"Man","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vaneeza A.","family":"Moosa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anand","family":"Singh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Licun","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John T.","family":"Granton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen C.","family":"Juvet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuong D.","family":"Hoang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"de Perrot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2023,11,23]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"761","DOI":"10.1172\/JCI84424","article-title":"Targeting noncoding RNAs in disease","volume":"127","author":"Adams","year":"2017","journal-title":"J. Clin. Invest."},{"key":"B2","doi-asserted-by":"publisher","first-page":"47","DOI":"10.1146\/annurev-physiol-032922-082826","article-title":"Lung cell atlases in health and disease","volume":"85","author":"Adams","year":"2023","journal-title":"Annu. Rev. Physiol."},{"key":"B3","doi-asserted-by":"publisher","first-page":"4438","DOI":"10.3390\/ijms24054438","article-title":"Novel biomarkers for diagnosis and monitoring of immune thrombocytopenia","volume":"24","author":"Allegra","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"B4","doi-asserted-by":"publisher","first-page":"3169","DOI":"10.1021\/acsnano.5b00147","article-title":"Elasticity of nanoparticles influences their blood circulation, phagocytosis, endocytosis, and targeting","volume":"9","author":"Anselmo","year":"2015","journal-title":"ACS Nano"},{"key":"B5","doi-asserted-by":"publisher","first-page":"e2120680119","DOI":"10.1073\/pnas.2120680119","article-title":"Single-cell RNA sequencing uncovers the nuclear decoy lincRNA PIRAT as a regulator of systemic monocyte immunity during COVID-19","volume":"119","author":"Aznaourova","year":"2022","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B6","doi-asserted-by":"publisher","first-page":"403","DOI":"10.1056\/NEJMoa2035389","article-title":"Efficacy and safety of the mRNA-1273 SARS-CoV-2 vaccine","volume":"384","author":"Baden","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"B7","doi-asserted-by":"publisher","first-page":"A4605","DOI":"10.1164\/ajrccm-conference.2019.199.1_MeetingAbstracts.A4605","article-title":"Pulmonary delivery of NLRP3 antisense oligonucleotides are effective at preventing but not reversing established bleomycin-induced pulmonary fibrosis in mice","volume":"199","author":"Bai","year":"2019","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"B8","doi-asserted-by":"publisher","first-page":"1374","DOI":"10.1016\/j.jtcvs.2014.10.128","article-title":"Updated patterns of failure after multimodality therapy for malignant pleural mesothelioma","volume":"149","author":"Baldini","year":"2015","journal-title":"J. Thorac. Cardiovasc. Surg."},{"key":"B9","doi-asserted-by":"publisher","first-page":"97","DOI":"10.2165\/00129785-200303020-00003","article-title":"Clinical potential of respirable antisense oligonucleotides (RASONs) in asthma","volume":"3","author":"Ball","year":"2003","journal-title":"Am. J. Pharmacogenomics"},{"key":"B10","doi-asserted-by":"publisher","first-page":"8598054","DOI":"10.1155\/2018\/8598054","article-title":"Safety and efficacy of extremely low LDL-cholesterol levels and its prospects in hyperlipidemia management","volume":"2018","author":"Bandyopadhyay","year":"2018","journal-title":"J. Lipids"},{"key":"B11","doi-asserted-by":"publisher","first-page":"281","DOI":"10.1016\/s0092-8674(04)00045-5","article-title":"MicroRNAs: genomics, biogenesis, mechanism, and function","volume":"116","author":"Bartel","year":"2004","journal-title":"Cell"},{"key":"B12","doi-asserted-by":"publisher","first-page":"215","DOI":"10.1016\/j.cell.2009.01.002","article-title":"MicroRNAs: target recognition and regulatory functions","volume":"136","author":"Bartel","year":"2009","journal-title":"Cell"},{"key":"B13","doi-asserted-by":"publisher","first-page":"20","DOI":"10.1016\/j.cell.2018.03.006","article-title":"Metazoan MicroRNAs","volume":"173","author":"Bartel","year":"2018","journal-title":"Cell"},{"key":"B14","doi-asserted-by":"publisher","first-page":"S540","DOI":"10.1016\/j.healun.2023.02.1475","article-title":"(1265) exploration of intragraft T cell phenotypes in minimal acute cellular rejection (ACR) using imaging mass cytometry (IMC)","volume":"42","author":"Beber","year":"2023","journal-title":"J. Heart Lung Transpl."},{"key":"B15","doi-asserted-by":"publisher","first-page":"81","DOI":"10.1146\/annurev-pharmtox-010716-104846","article-title":"Pharmacology of antisense drugs","volume":"57","author":"Bennett","year":"2017","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"B16","doi-asserted-by":"publisher","first-page":"259","DOI":"10.1146\/annurev.pharmtox.010909.105654","article-title":"RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform","volume":"50","author":"Bennett","year":"2010","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"B17","doi-asserted-by":"publisher","first-page":"199","DOI":"10.1038\/nmeth854","article-title":"3\u2019 UTR seed matches, but not overall identity, are associated with RNAi off-targets","volume":"3","author":"Birmingham","year":"2006","journal-title":"Nat. Methods"},{"key":"B18","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1111\/imm.13458","article-title":"Effector immune cells in chronic lung allograft dysfunction: a systematic review","volume":"166","author":"Bos","year":"2022","journal-title":"Immunology"},{"key":"B19","doi-asserted-by":"publisher","first-page":"e1010460","DOI":"10.1371\/journal.pgen.1010460","article-title":"Translational buffering by ribosome stalling in upstream open reading frames","volume":"18","author":"Bottorff","year":"2022","journal-title":"PLOS Genet."},{"key":"B20","doi-asserted-by":"publisher","first-page":"R21","DOI":"10.1093\/hmg\/ddr137","article-title":"RNAi medicine for the brain: progresses and challenges","volume":"20","author":"Boudreau","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"B21","doi-asserted-by":"publisher","first-page":"7507","DOI":"10.1073\/pnas.0810916106","article-title":"Upstream open reading frames cause widespread reduction of protein expression and are polymorphic among humans","volume":"106","author":"Calvo","year":"2009","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B22","doi-asserted-by":"publisher","first-page":"46","DOI":"10.1165\/rcmb.2019-0257OC","article-title":"Antisense oligonucleotides targeting jagged 1 reduce house dust mite-induced goblet cell metaplasia in the adult murine lung","volume":"63","author":"Carrer","year":"2020","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"B23","doi-asserted-by":"publisher","first-page":"642","DOI":"10.1016\/j.cell.2009.01.035","article-title":"Origins and Mechanisms of miRNAs and siRNAs","volume":"136","author":"Carthew","year":"2009","journal-title":"Cell"},{"key":"B24","doi-asserted-by":"publisher","first-page":"1272","DOI":"10.1016\/j.amjcard.2018.06.023","article-title":"Effectiveness and safety of inclisiran, A novel long-acting RNA therapeutic inhibitor of proprotein convertase subtilisin\/kexin 9","volume":"122","author":"Chandra Ghosh","year":"2018","journal-title":"Am. J. Cardiol."},{"key":"B25","doi-asserted-by":"publisher","first-page":"e1609","DOI":"10.1002\/wnan.1609","article-title":"Strategies for nonviral nanoparticle-based delivery of CRISPR\/Cas9 therapeutics","volume":"12","author":"Chen","year":"2020","journal-title":"Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol."},{"key":"B26","doi-asserted-by":"publisher","first-page":"601","DOI":"10.1038\/s41589-021-00763-0","article-title":"Hovlinc is a recently evolved class of ribozyme found in human lncRNA","volume":"17","author":"Chen","year":"2021","journal-title":"Nat. Chem. Biol."},{"key":"B205","doi-asserted-by":"crossref","first-page":"D127","DOI":"10.1093\/nar\/gkz757","article-title":"miRDB: an online database for prediction of functional microRNA targets","volume":"48","author":"Chen","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"B27","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1038\/nrd4503","article-title":"A holistic approach to targeting disease with polymeric nanoparticles","volume":"14","author":"Cheng","year":"2015","journal-title":"Nat. Rev. Drug Discov."},{"key":"B28","doi-asserted-by":"publisher","first-page":"313","DOI":"10.1038\/s41565-020-0669-6","article-title":"Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing","volume":"15","author":"Cheng","year":"2020","journal-title":"Nat. Nanotechnol."},{"key":"B29","doi-asserted-by":"publisher","first-page":"104304","DOI":"10.1016\/j.ebiom.2022.104304","article-title":"A lung targeted miR-29 mimic as a therapy for pulmonary fibrosis","volume":"85","author":"Chioccioli","year":"2022","journal-title":"eBioMedicine"},{"key":"B30","doi-asserted-by":"publisher","first-page":"190","DOI":"10.1016\/S1470-2045(20)30606-9","article-title":"Surgery for malignant pleural mesothelioma after radiotherapy (SMART): final results from a single-centre, phase 2 trial","volume":"22","author":"Cho","year":"2021","journal-title":"Lancet Oncol."},{"key":"B31","doi-asserted-by":"publisher","first-page":"886430","DOI":"10.3389\/fonc.2022.886430","article-title":"Current state of pleural-directed adjuncts against malignant pleural mesothelioma","volume":"12","author":"Choi","year":"2022","journal-title":"Front. Oncol."},{"key":"B32","doi-asserted-by":"publisher","first-page":"933","DOI":"10.1039\/c9nr06382j","article-title":"Targeted delivery of Chil3\/Chil4 siRNA to alveolar macrophages using ternary complexes composed of HMG and oligoarginine micelles","volume":"12","author":"Choi","year":"2020","journal-title":"Nanoscale"},{"key":"B33","doi-asserted-by":"publisher","first-page":"715","DOI":"10.1016\/j.tips.2020.08.002","article-title":"Inhaled RNA therapy: from promise to reality","volume":"41","author":"Chow","year":"2020","journal-title":"Trends Pharmacol. Sci."},{"key":"B34","doi-asserted-by":"publisher","first-page":"6393","DOI":"10.1038\/s41598-021-85549-z","article-title":"Assisting PNA transport through cystic fibrosis human airway epithelia with biodegradable hybrid lipid-polymer nanoparticles","volume":"11","author":"Comegna","year":"2021","journal-title":"Sci. Rep."},{"key":"B35","doi-asserted-by":"publisher","first-page":"2194","DOI":"10.1016\/s0959-8049(01)00286-6","article-title":"Phase II trial with ISIS 5132 in patients with small-cell (SCLC) and non-small cell (NSCLC) lung cancer. A European organization for research and treatment of cancer (eortc) early clinical studies group report","volume":"37","author":"Coudert","year":"2001","journal-title":"Eur. J. Cancer Oxf. Engl."},{"key":"B36","doi-asserted-by":"publisher","first-page":"561","DOI":"10.1038\/227561a0","article-title":"Central dogma of molecular biology","volume":"227","author":"Crick","year":"1970","journal-title":"Nature"},{"key":"B37","doi-asserted-by":"publisher","first-page":"100416","DOI":"10.1016\/j.jbc.2021.100416","article-title":"Antisense technology: a review","volume":"296","author":"Crooke","year":"2021","journal-title":"J. Biol. Chem."},{"key":"B38","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1038\/nbt.3779","article-title":"Cellular uptake and trafficking of antisense oligonucleotides","volume":"35","author":"Crooke","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"B39","doi-asserted-by":"publisher","first-page":"714","DOI":"10.1016\/j.cmet.2018.03.004","article-title":"RNA-targeted therapeutics","volume":"27","author":"Crooke","year":"2018","journal-title":"Cell Metab."},{"key":"B40","doi-asserted-by":"publisher","first-page":"397","DOI":"10.1165\/rcmb.2014-0232RT","article-title":"The roles of microRNAs and protein components of the microRNA pathway in lung development and diseases","volume":"52","author":"Cushing","year":"2015","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"B41","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1161\/CIRCRESAHA.122.321554","article-title":"BMPR2 mutation and metabolic reprogramming in pulmonary arterial hypertension","volume":"132","author":"Cuthbertson","year":"2023","journal-title":"Circ. Res."},{"key":"B42","doi-asserted-by":"publisher","first-page":"648","DOI":"10.1038\/nnano.2014.84","article-title":"In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight","volume":"9","author":"Dahlman","year":"2014","journal-title":"Nat. Nanotechnol."},{"key":"B43","doi-asserted-by":"publisher","first-page":"628137","DOI":"10.3389\/fbioe.2021.628137","article-title":"The limitless future of RNA therapeutics","volume":"9","author":"Damase","year":"2021","journal-title":"Front. Bioeng. Biotechnol."},{"key":"B44","doi-asserted-by":"publisher","first-page":"W375","DOI":"10.1093\/nar\/gkm216","article-title":"MolProbity: all-atom contacts and structure validation for proteins and nucleic acids","volume":"35","author":"Davis","year":"2007","journal-title":"Nucleic Acids Res."},{"key":"B45","doi-asserted-by":"publisher","first-page":"145839","DOI":"10.1016\/j.gene.2021.145839","article-title":"Therapeutic perceptions in antisense RNA-mediated gene regulation for COVID-19","volume":"800","author":"de Jesus","year":"2021","journal-title":"Gene"},{"key":"B46","doi-asserted-by":"publisher","first-page":"D262","DOI":"10.1093\/nar\/gkac840","article-title":"Ribocentre: a database of ribozymes","volume":"51","author":"Deng","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"B47","doi-asserted-by":"publisher","first-page":"1775","DOI":"10.1101\/gr.132159.111","article-title":"The GENCODE v7 catalog of human long noncoding RNAs: analysis of their gene structure, evolution, and expression","volume":"22","author":"Derrien","year":"2012","journal-title":"Genome Res."},{"key":"B48","doi-asserted-by":"publisher","first-page":"8800","DOI":"10.1073\/pnas.0912186107","article-title":"A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus","volume":"107","author":"DeVincenzo","year":"2010","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B49","doi-asserted-by":"publisher","first-page":"S2","DOI":"10.1038\/d41586-019-03068-4","article-title":"RNA therapies explained","volume":"574","author":"DeWeerdt","year":"2019","journal-title":"Nature"},{"key":"B50","doi-asserted-by":"publisher","first-page":"305","DOI":"10.3389\/fphar.2019.00305","article-title":"Antisense oligonucleotide: basic concepts and therapeutic application in inflammatory bowel disease","volume":"10","author":"Di Fusco","year":"2019","journal-title":"Front. Pharmacol."},{"key":"B51","doi-asserted-by":"publisher","first-page":"504","DOI":"10.1101\/gad.1184404","article-title":"Specificity of microRNA target selection in translational repression","volume":"18","author":"Doench","year":"2004","journal-title":"Genes Dev."},{"key":"B52","doi-asserted-by":"publisher","first-page":"1258096","DOI":"10.1126\/science.1258096","article-title":"Genome editing. The new frontier of genome engineering with CRISPR-Cas9","volume":"346","author":"Doudna","year":"2014","journal-title":"Science"},{"key":"B53","doi-asserted-by":"publisher","first-page":"99","DOI":"10.1016\/j.jcf.2019.05.014","article-title":"Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis","volume":"19","author":"Drevinek","year":"2020","journal-title":"J. Cyst. Fibros."},{"key":"B54","doi-asserted-by":"publisher","first-page":"721","DOI":"10.1038\/nmeth1079","article-title":"MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells","volume":"4","author":"Ebert","year":"2007","journal-title":"Nat. Methods"},{"key":"B55","doi-asserted-by":"publisher","first-page":"2529","DOI":"10.1093\/nar\/gkad067","article-title":"Chemistry, structure and function of approved oligonucleotide therapeutics","volume":"51","author":"Egli","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"B56","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1021\/nn900002m","article-title":"Impact of nanotechnology on drug delivery","volume":"3","author":"Farokhzad","year":"2009","journal-title":"ACS Nano"},{"key":"B57","doi-asserted-by":"publisher","first-page":"384","DOI":"10.1016\/j.omtn.2020.06.006","article-title":"Cell type impacts accessibility of mRNA to silencing by RNA interference","volume":"21","author":"Ferguson","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"B58","doi-asserted-by":"publisher","first-page":"1156","DOI":"10.1097\/JTO.0b013e3181b2793f","article-title":"Phase I study of the c-raf-1 antisense oligonucleotide ISIS 5132 in combination with carboplatin and paclitaxel in patients with previously untreated, advanced non-small cell lung cancer","volume":"4","author":"Fidias","year":"2009","journal-title":"J. Thorac. Oncol."},{"key":"B59","doi-asserted-by":"publisher","first-page":"806","DOI":"10.1038\/35888","article-title":"Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans","volume":"391","author":"Fire","year":"1998","journal-title":"Nature"},{"key":"B60","doi-asserted-by":"publisher","first-page":"1365","DOI":"10.1016\/j.ymthe.2021.03.005","article-title":"RNA-based vaccines and innate immune activation: not too hot and not too cold","volume":"29","author":"Frederickson","year":"2021","journal-title":"Mol. Ther."},{"key":"B61","doi-asserted-by":"publisher","first-page":"1110","DOI":"10.1093\/nar\/gky1260","article-title":"Characterization of the interactions of chemically-modified therapeutic nucleic acids with plasma proteins using a fluorescence polarization assay","volume":"47","author":"Gaus","year":"2019","journal-title":"Nucleic Acids Res."},{"key":"B62","doi-asserted-by":"publisher","first-page":"952","DOI":"10.1164\/rccm.200708-1251OC","article-title":"Antisense therapy against CCR3 and the common beta chain attenuates allergen-induced eosinophilic responses","volume":"177","author":"Gauvreau","year":"2008","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"B63","doi-asserted-by":"publisher","first-page":"1242","DOI":"10.1111\/j.1398-9995.2011.02638.x","article-title":"Dose\u2013response effects of TPI ASM8 in asthmatics after allergen","volume":"66","author":"Gauvreau","year":"2011","journal-title":"Allergy"},{"key":"B64","volume-title":"Human release 43","year":"2022"},{"key":"B65","doi-asserted-by":"publisher","first-page":"493","DOI":"10.1056\/NEJMoa2107454","article-title":"CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis","volume":"385","author":"Gillmore","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"B66","doi-asserted-by":"publisher","first-page":"199","DOI":"10.1038\/nature04917","article-title":"A germline-specific class of small RNAs binds mammalian Piwi proteins","volume":"442","author":"Girard","year":"2006","journal-title":"Nature"},{"key":"B67","doi-asserted-by":"publisher","first-page":"213","DOI":"10.1016\/j.healun.2015.08.012","article-title":"ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients","volume":"35","author":"Gottlieb","year":"2016","journal-title":"J. Heart Lung Transpl."},{"key":"B68","doi-asserted-by":"publisher","first-page":"537","DOI":"10.1038\/nature04791","article-title":"Fatality in mice due to oversaturation of cellular microRNA\/short hairpin RNA pathways","volume":"441","author":"Grimm","year":"2006","journal-title":"Nature"},{"key":"B69","doi-asserted-by":"publisher","first-page":"7312","DOI":"10.1158\/1078-0432.CCR-16-2186","article-title":"A combination RNAi-chemotherapy layer-by-layer nanoparticle for systemic targeting of KRAS\/P53 with cisplatin to treat non\u2013small cell lung cancer","volume":"23","author":"Gu","year":"2017","journal-title":"Clin. Cancer Res."},{"key":"B70","doi-asserted-by":"publisher","first-page":"287","DOI":"10.1038\/s41565-018-0358-x","article-title":"Self-assembled peptide\u2013poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis","volume":"14","author":"Guan","year":"2019","journal-title":"Nat. Nanotechnol."},{"key":"B71","doi-asserted-by":"publisher","first-page":"285","DOI":"10.1126\/science.6199841","article-title":"Catalytic activity of an RNA molecule prepared by transcription in vitro","volume":"223","author":"Guerrier-Takada","year":"1984","journal-title":"Science"},{"key":"B72","doi-asserted-by":"publisher","first-page":"849","DOI":"10.1016\/0092-8674(83)90117-4","article-title":"The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme","volume":"35","author":"Guerrier-Takada","year":"1983","journal-title":"Cell"},{"key":"B73","doi-asserted-by":"publisher","first-page":"503","DOI":"10.1038\/nbt.1633","article-title":"Ab initio reconstruction of cell type\u2013specific transcriptomes in mouse reveals the conserved multi-exonic structure of lincRNAs","volume":"28","author":"Guttman","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"B74","doi-asserted-by":"publisher","first-page":"1459","DOI":"10.1016\/S0140-6736(21)00675-9","article-title":"SARS-CoV-2 infection rates of antibody-positive compared with antibody-negative health-care workers in England: a large, multicentre, prospective cohort study (SIREN)","volume":"397","author":"Hall","year":"2021","journal-title":"Lancet"},{"key":"B75","doi-asserted-by":"publisher","first-page":"e13243","DOI":"10.15252\/emmm.202013243","article-title":"Delivery of oligonucleotide-based therapeutics: challenges and opportunities","volume":"13","author":"Hammond","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"B76","doi-asserted-by":"publisher","first-page":"4439","DOI":"10.1038\/s41467-019-12449-2","article-title":"High levels of AAV vector integration into CRISPR-induced DNA breaks","volume":"10","author":"Hanlon","year":"2019","journal-title":"Nat. Commun."},{"key":"B77","doi-asserted-by":"publisher","first-page":"502","DOI":"10.1242\/dev.098186","article-title":"Lung development: orchestrating the generation and regeneration of a complex organ","volume":"141","author":"Herriges","year":"2014","journal-title":"Dev. Camb. Engl."},{"key":"B78","doi-asserted-by":"publisher","first-page":"117","DOI":"10.1016\/j.ejpb.2015.02.014","article-title":"Barrier or carrier? Pulmonary surfactant and drug delivery","volume":"95","author":"Hidalgo","year":"2015","journal-title":"Eur. J. Pharm. Biopharm."},{"key":"B79","doi-asserted-by":"publisher","first-page":"e1647","DOI":"10.1002\/wrna.1647","article-title":"Translational remodeling by RNA-binding proteins and noncoding RNAs","volume":"12","author":"Ho","year":"2021","journal-title":"WIREs RNA"},{"key":"B80","doi-asserted-by":"publisher","first-page":"2029","DOI":"10.1128\/MCB.01257-12","article-title":"Active stabilization of human endothelial nitric oxide synthase mRNA by hnRNP E1 protects against antisense RNA and MicroRNAs","volume":"33","author":"Ho","year":"2013","journal-title":"Mol. Cell. Biol."},{"key":"B81","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1146\/annurev-med-042420-112725","article-title":"mRNA vaccines in the COVID-19 pandemic and beyond","volume":"73","author":"Hogan","year":"2022","journal-title":"Annu. Rev. Med."},{"key":"B82","doi-asserted-by":"publisher","first-page":"696","DOI":"10.1002\/jcb.29896","article-title":"The role of miR-29 family in disease","volume":"122","author":"Horita","year":"2021","journal-title":"J. Cell. Biochem."},{"key":"B83","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1038\/nm1191","article-title":"Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7","volume":"11","author":"Hornung","year":"2005","journal-title":"Nat. Med."},{"key":"B84","doi-asserted-by":"publisher","first-page":"1179","DOI":"10.1261\/rna.25706","article-title":"Widespread siRNA \u201coff-target\u201d transcript silencing mediated by seed region sequence complementarity","volume":"12","author":"Jackson","year":"2006","journal-title":"RNA"},{"key":"B85","doi-asserted-by":"publisher","first-page":"1920","DOI":"10.1056\/NEJMoa2022483","article-title":"An mRNA vaccine against SARS-CoV-2 \u2014 preliminary report","volume":"383","author":"Jackson","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"B86","doi-asserted-by":"publisher","first-page":"457","DOI":"10.1038\/nbt1081","article-title":"Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA","volume":"23","author":"Judge","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"B87","doi-asserted-by":"publisher","first-page":"6518","DOI":"10.1093\/nar\/gkw236","article-title":"The delivery of therapeutic oligonucleotides","volume":"44","author":"Juliano","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"B88","first-page":"153","article-title":"RNA aptamers: a review of recent trends and applications","volume-title":"Future trends in biotechnology advances in biochemical engineering\/biotechnology","author":"Kang","year":"2013"},{"key":"B89","doi-asserted-by":"publisher","first-page":"35835","DOI":"10.1021\/acsami.0c08268","article-title":"Surface-functionalized PEGylated nanoparticles deliver messenger RNA to pulmonary immune cells","volume":"12","author":"Ke","year":"2020","journal-title":"ACS Appl. Mat. Interfaces"},{"key":"B90","doi-asserted-by":"publisher","first-page":"852","DOI":"10.1016\/j.tig.2017.08.008","article-title":"Gene therapy with the sleeping beauty transposon system","volume":"33","author":"Kebriaei","year":"2017","journal-title":"Trends Genet. TIG"},{"key":"B91","doi-asserted-by":"publisher","first-page":"e1634","DOI":"10.1002\/wnan.1634","article-title":"T-cell targeted pulmonary siRNA delivery for the treatment of asthma","volume":"12","author":"Keil","year":"2020","journal-title":"WIREs Nanomedicine Nanobiotechnology"},{"key":"B92","doi-asserted-by":"publisher","first-page":"549","DOI":"10.1038\/nbt.1543","article-title":"Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs","volume":"27","author":"Khan","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"B93","doi-asserted-by":"publisher","first-page":"eaar8409","DOI":"10.1126\/sciadv.aar8409","article-title":"Endothelial siRNA delivery in nonhuman primates using ionizable low\u2013molecular weight polymeric nanoparticles","volume":"4","author":"Khan","year":"2018","journal-title":"Sci. Adv."},{"key":"B94","doi-asserted-by":"publisher","first-page":"108","DOI":"10.1016\/j.jconrel.2020.03.016","article-title":"Lung-targeted delivery of TGF-\u03b2 antisense oligonucleotides to treat pulmonary fibrosis","volume":"322","author":"Kim","year":"","journal-title":"J. Control. Release Off. J. Control. Release Soc."},{"key":"B95","doi-asserted-by":"publisher","first-page":"2285","DOI":"10.1038\/s41467-020-16164-1","article-title":"Single-cell RNA sequencing demonstrates the molecular and cellular reprogramming of metastatic lung adenocarcinoma","volume":"11","author":"Kim","year":"","journal-title":"Nat. Commun."},{"key":"B96","doi-asserted-by":"publisher","first-page":"455","DOI":"10.1038\/s12276-022-00757-5","article-title":"RNA therapy: rich history, various applications and unlimited future prospects","volume":"54","author":"Kim","year":"2022","journal-title":"Exp. Mol. Med."},{"key":"B97","doi-asserted-by":"publisher","first-page":"e3106","DOI":"10.1093\/cid\/ciaa1589","article-title":"Self-collected oral fluid and nasal swabs demonstrate comparable sensitivity to clinician collected nasopharyngeal swabs for coronavirus disease 2019 detection","volume":"73","author":"Kojima","year":"2021","journal-title":"Clin. Infect. Dis."},{"key":"B98","doi-asserted-by":"publisher","first-page":"665","DOI":"10.1016\/j.cell.2018.02.033","article-title":"Transcriptome engineering with RNA-targeting type VI-D CRISPR effectors","volume":"173","author":"Konermann","year":"2018","journal-title":"Cell"},{"key":"B99","doi-asserted-by":"publisher","first-page":"154","DOI":"10.1038\/nbt.1733","article-title":"Expression of therapeutic proteins after delivery of chemically modified mRNA in mice","volume":"29","author":"Kormann","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"B100","doi-asserted-by":"publisher","first-page":"396","DOI":"10.1038\/nature18300","article-title":"Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy","volume":"534","author":"Kranz","year":"2016","journal-title":"Nature"},{"key":"B101","doi-asserted-by":"publisher","first-page":"147","DOI":"10.1016\/0092-8674(82)90414-7","article-title":"Self-splicing RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of Tetrahymena","volume":"31","author":"Kruger","year":"1982","journal-title":"Cell"},{"key":"B102","doi-asserted-by":"publisher","first-page":"685","DOI":"10.1038\/nature04303","article-title":"Silencing of microRNAs in vivo with \u2018antagomirs","volume":"438","author":"Kr\u00fctzfeldt","year":"2005","journal-title":"\u2019 Nat."},{"key":"B103","doi-asserted-by":"publisher","first-page":"72","DOI":"10.1002\/jimd.12251","article-title":"Opportunities and challenges for antisense oligonucleotide therapies","volume":"44","author":"Kuijper","year":"2021","journal-title":"J. Inherit. Metab. Dis."},{"key":"B104","doi-asserted-by":"publisher","first-page":"e252","DOI":"10.1038\/mtna.2015.23","article-title":"siRNA versus miRNA as therapeutics for gene silencing","volume":"4","author":"Lam","year":"2015","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"B105","doi-asserted-by":"publisher","first-page":"797","DOI":"10.1038\/263797a0","article-title":"Polymers for the sustained release of proteins and other macromolecules","volume":"263","author":"Langer","year":"1976","journal-title":"Nature"},{"key":"B106","doi-asserted-by":"publisher","first-page":"579","DOI":"10.1097\/JTO.0b013e31823f459c","article-title":"Phase I\/II trial of custirsen (OGX-011), an inhibitor of clusterin, in combination with a gemcitabine and platinum regimen in patients with previously untreated advanced non-small cell lung cancer","volume":"7","author":"Laskin","year":"2012","journal-title":"J. Thorac. Oncol."},{"key":"B107","doi-asserted-by":"publisher","first-page":"843","DOI":"10.1016\/0092-8674(93)90529-y","article-title":"The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14","volume":"75","author":"Lee","year":"1993","journal-title":"Cell"},{"key":"B108","doi-asserted-by":"publisher","first-page":"662","DOI":"10.1016\/j.omtn.2023.02.021","article-title":"Inhalation of an RNA aptamer that selectively binds extracellular histones protects from acute lung injury","volume":"31","author":"Lei","year":"2023","journal-title":"Mol. Ther. - Nucleic Acids"},{"key":"B109","doi-asserted-by":"publisher","first-page":"H377-H391","DOI":"10.1152\/ajpheart.00717.2019","article-title":"LncRNA-SMILR modulates RhoA\/ROCK signaling by targeting miR-141 to regulate vascular remodeling in pulmonary arterial hypertension","volume":"319","author":"Lei","year":"2020","journal-title":"Am. J. Physiol.-Heart Circ. Physiol."},{"key":"B110","doi-asserted-by":"publisher","first-page":"1410","DOI":"10.1038\/s41587-023-01679-x","article-title":"Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing","volume":"41","author":"Li","year":"2023","journal-title":"Nat. Biotechnol."},{"key":"B111","doi-asserted-by":"publisher","first-page":"106216","DOI":"10.1016\/j.ijantimicag.2020.106216","article-title":"Safety and efficacy of artemisinin-piperaquine for treatment of COVID-19: an open-label, non-randomised and controlled trial","volume":"57","author":"Li","year":"2021","journal-title":"Int. J. Antimicrob. Agents"},{"key":"B112","doi-asserted-by":"publisher","first-page":"830","DOI":"10.1021\/acschembio.0c00003","article-title":"Engineering caveolae-targeted lipid nanoparticles to deliver mRNA to the lungs","volume":"15","author":"Li","year":"2020","journal-title":"ACS Chem. Biol."},{"key":"B113","doi-asserted-by":"publisher","first-page":"875","DOI":"10.1038\/nbt.3589","article-title":"Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames","volume":"34","author":"Liang","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"B114","doi-asserted-by":"publisher","first-page":"139","DOI":"10.3390\/molecules22010139","article-title":"Oligonucleotide therapy for obstructive and restrictive respiratory diseases","volume":"22","author":"Liao","year":"2017","journal-title":"Mol. Basel Switz."},{"key":"B115","doi-asserted-by":"publisher","first-page":"573","DOI":"10.1038\/s41576-023-00613-w","article-title":"Transitioning single-cell genomics into the clinic","volume":"24","author":"Lim","year":"2023","journal-title":"Nat. Rev. Genet."},{"key":"B116","doi-asserted-by":"publisher","first-page":"1675","DOI":"10.1261\/rna.7175704","article-title":"RNAi: finding the elusive endonuclease","volume":"10","author":"Lingel","year":"2004","journal-title":"RNA"},{"key":"B117","doi-asserted-by":"publisher","first-page":"701","DOI":"10.1038\/s41563-020-00886-0","article-title":"Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR\u2013Cas gene editing","volume":"20","author":"Liu","year":"2021","journal-title":"Nat. Mat."},{"key":"B118","doi-asserted-by":"publisher","first-page":"1059","DOI":"10.1038\/s41551-021-00786-x","article-title":"Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs","volume":"5","author":"Lokugamage","year":"2021","journal-title":"Nat. Biomed. Eng."},{"key":"B119","doi-asserted-by":"publisher","first-page":"1251","DOI":"10.1038\/s41565-021-00961-w","article-title":"Surface-fill hydrogel attenuates the oncogenic signature of complex anatomical surface cancer in a single application","volume":"16","author":"Majumder","year":"2021","journal-title":"Nat. Nanotechnol."},{"key":"B120","doi-asserted-by":"publisher","first-page":"103029","DOI":"10.1016\/j.jbc.2023.103029","article-title":"Long noncoding RNA GATA2-AS1 augments endothelial hypoxia inducible factor 1-\u03b1 induction and regulates hypoxic signaling","volume":"299","author":"Man","year":"2023","journal-title":"J. Biol. Chem."},{"key":"B121","doi-asserted-by":"publisher","first-page":"1399","DOI":"10.1038\/nbt1161","article-title":"Activation of the mammalian immune system by siRNAs","volume":"23","author":"Marques","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"B122","doi-asserted-by":"publisher","first-page":"1719","DOI":"10.1002\/eji.1830230749","article-title":"Induction of virus-specific cytotoxic T lymphocytes in vivo by liposome-entrapped mRNA","volume":"23","author":"Martinon","year":"1993","journal-title":"Eur. J. Immunol."},{"key":"B123","doi-asserted-by":"publisher","first-page":"8720","DOI":"10.1073\/pnas.1502281112","article-title":"Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy","volume":"112","author":"Mastorakos","year":"2015","journal-title":"Proc. Natl. Acad. Sci."},{"key":"B124","doi-asserted-by":"publisher","first-page":"430","DOI":"10.1038\/s41580-022-00566-8","article-title":"Long non-coding RNAs: definitions, functions, challenges and recommendations","volume":"24","author":"Mattick","year":"2023","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"B125","doi-asserted-by":"publisher","first-page":"6952","DOI":"10.1038\/ncomms7952","article-title":"Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium","volume":"6","author":"McNeer","year":"2015","journal-title":"Nat. Commun."},{"key":"B126","doi-asserted-by":"publisher","first-page":"338","DOI":"10.1038\/nature02872","article-title":"Revealing the world of RNA interference","volume":"431","author":"Mello","year":"2004","journal-title":"Nature"},{"key":"B127","doi-asserted-by":"publisher","first-page":"331","DOI":"10.3389\/fimmu.2017.00331","article-title":"RNA interference-induced innate immunity, off-target effect, or immune adjuvant?","volume":"8","author":"Meng","year":"2017","journal-title":"Front. Immunol."},{"key":"B128","doi-asserted-by":"publisher","first-page":"e64708","DOI":"10.1371\/journal.pone.0064708","article-title":"RNAi induces innate immunity through multiple cellular signaling pathways","volume":"8","author":"Meng","year":"2013","journal-title":"PLOS ONE"},{"key":"B129","doi-asserted-by":"publisher","first-page":"101","DOI":"10.1038\/s41573-020-0090-8","article-title":"Engineering precision nanoparticles for drug delivery","volume":"20","author":"Mitchell","year":"2021","journal-title":"Nat. Rev. Drug Discov."},{"key":"B130","doi-asserted-by":"publisher","first-page":"2931","DOI":"10.1534\/g3.117.044024","article-title":"ASPsiRNA: a resource of ASP-siRNAs having therapeutic potential for human genetic disorders and algorithm for prediction of their inhibitory efficacy","volume":"7","author":"Monga","year":"2017","journal-title":"G3 GenesGenomesGenetics"},{"key":"B131","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1007\/978-1-61779-439-1_10","article-title":"shRNA-induced interferon-stimulated gene analysis","volume-title":"Cytokine protocols methods in molecular biology","author":"Morral","year":"2012"},{"key":"B132","doi-asserted-by":"publisher","first-page":"1002","DOI":"10.1038\/nbt1122","article-title":"Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs","volume":"23","author":"Morrissey","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"B133","doi-asserted-by":"publisher","first-page":"429","DOI":"10.1038\/s41586-021-03534-y","article-title":"In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates","volume":"593","author":"Musunuru","year":"2021","journal-title":"Nature"},{"key":"B134","doi-asserted-by":"publisher","first-page":"1784","DOI":"10.1038\/s41593-018-0265-3","article-title":"Regulation of cell-type-specific transcriptomes by microRNA networks during human brain development","volume":"21","author":"Nowakowski","year":"2018","journal-title":"Nat. Neurosci."},{"key":"B135","doi-asserted-by":"publisher","first-page":"532","DOI":"10.2174\/156652311798192770","article-title":"Recent advances in understanding of the immunological off-target effects of siRNA","volume":"11","author":"Olejniczak","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"B136","doi-asserted-by":"publisher","first-page":"1859","DOI":"10.1001\/jama.2023.8120","article-title":"Cystic fibrosis: a review","volume":"329","author":"Ong","year":"2023","journal-title":"JAMA"},{"key":"B137","doi-asserted-by":"publisher","first-page":"1227","DOI":"10.1021\/cb500880f","article-title":"Biophysical and biological characterization of hairpin and molecular beacon RNase H active antisense oligonucleotides","volume":"10","author":"\u00d8stergaard","year":"2015","journal-title":"ACS Chem. Biol."},{"key":"B138","doi-asserted-by":"publisher","first-page":"345","DOI":"10.1016\/j.jconrel.2015.08.007","article-title":"Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes","volume":"217","author":"Pardi","year":"2015","journal-title":"J. Control. Release Off. J. Control. Release Soc."},{"key":"B139","doi-asserted-by":"publisher","first-page":"1805116","DOI":"10.1002\/adma.201805116","article-title":"Inhaled nanoformulated mRNA polyplexes for protein production in lung epithelium","volume":"31","author":"Patel","year":"2019","journal-title":"Adv. Mat."},{"key":"B140","doi-asserted-by":"publisher","first-page":"265","DOI":"10.1038\/s41576-021-00439-4","article-title":"Drug delivery systems for RNA therapeutics","volume":"23","author":"Paunovska","year":"2022","journal-title":"Nat. Rev. Genet."},{"key":"B141","doi-asserted-by":"publisher","first-page":"8341","DOI":"10.1021\/acsnano.8b03640","article-title":"Analyzing 2000 in vivo drug delivery data points reveals cholesterol structure impacts nanoparticle delivery","volume":"12","author":"Paunovska","year":"2018","journal-title":"ACS Nano"},{"key":"B142","doi-asserted-by":"publisher","first-page":"7020","DOI":"10.1200\/jco.2006.24.18_suppl.7020","article-title":"A prospective phase II trial of erlotinib in advanced non-small cell lung cancer (NSCLC) patients (p) with mutations in the tyrosine kinase (TK) domain of the epidermal growth factor receptor (EGFR)","volume":"24","author":"Paz-Ares","year":"2006","journal-title":"J. Clin. Oncol."},{"key":"B143","doi-asserted-by":"publisher","first-page":"12502","DOI":"10.1093\/nar\/gkab1096","article-title":"Inhibition of SARS-CoV-2 coronavirus proliferation by designer antisense-circRNAs","volume":"49","author":"Pfafenrot","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"B144","doi-asserted-by":"publisher","first-page":"2603","DOI":"10.1056\/NEJMoa2034577","article-title":"Safety and efficacy of the BNT162b2 mRNA covid-19 vaccine","volume":"383","author":"Polack","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"B145","doi-asserted-by":"publisher","first-page":"83","DOI":"10.1038\/s41577-020-00479-7","article-title":"A guide to vaccinology: from basic principles to new developments","volume":"21","author":"Pollard","year":"2021","journal-title":"Nat. Rev. Immunol."},{"key":"B146","doi-asserted-by":"publisher","first-page":"4606","DOI":"10.1021\/acs.molpharmaceut.7b00725","article-title":"From pulmonary surfactant, synthetic KL4 peptide as effective siRNA delivery vector for pulmonary delivery","volume":"14","author":"Qiu","year":"2017","journal-title":"Mol. Pharm."},{"key":"B147","doi-asserted-by":"publisher","first-page":"102","DOI":"10.1016\/j.jconrel.2019.10.026","article-title":"Effective mRNA pulmonary delivery by dry powder formulation of PEGylated synthetic KL4 peptide","volume":"314","author":"Qiu","year":"2019","journal-title":"J. Control. Release"},{"key":"B148","doi-asserted-by":"publisher","first-page":"998","DOI":"10.1016\/S0140-6736(10)60284-X","article-title":"Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial","volume":"375","author":"Raal","year":"2010","journal-title":"Lancet lond. Engl."},{"key":"B149","doi-asserted-by":"publisher","first-page":"1437","DOI":"10.1016\/S0140-6736(21)01961-9","article-title":"Disease pathology in fibrotic interstitial lung disease: is it all about usual interstitial pneumonia?","volume":"398","author":"Renzoni","year":"2021","journal-title":"Lancet lond. Engl."},{"key":"B150","doi-asserted-by":"publisher","first-page":"3548","DOI":"10.1021\/ja01595a086","article-title":"A new two stranded helical structure: polyadenylic acid and polyuridylic acid","volume":"78","author":"Rich","year":"1956","journal-title":"J. Am. Chem. Soc."},{"key":"B151","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1038\/nrneurol.2017.148","article-title":"Antisense oligonucleotides: the next frontier for treatment of neurological disorders","volume":"14","author":"Rinaldi","year":"2018","journal-title":"Nat. Rev. Neurol."},{"key":"B152","doi-asserted-by":"publisher","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"key":"B153","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1016\/j.lungcan.2005.12.012","article-title":"Phase II study of PKC-alpha antisense oligonucleotide aprinocarsen in combination with gemcitabine and carboplatin in patients with advanced non-small cell lung cancer","volume":"52","author":"Ritch","year":"2006","journal-title":"Lung Cancer"},{"key":"B154","doi-asserted-by":"publisher","first-page":"991","DOI":"10.1089\/hum.2008.131","article-title":"Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation","volume":"19","author":"Robbins","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"B155","first-page":"14","article-title":"Fomivirsen approved for CMV retinitis","volume":"4","author":"Roehr","year":"1998","journal-title":"J. Int. Assoc. Physicians AIDS Care"},{"key":"B156","doi-asserted-by":"publisher","first-page":"1586","DOI":"10.1038\/s41587-022-01491-z","article-title":"Unlocking the promise of mRNA therapeutics","volume":"40","author":"Rohner","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"B157","doi-asserted-by":"publisher","first-page":"116","DOI":"10.1056\/NEJMoa2116063","article-title":"Covid-19 vaccine effectiveness in New York state","volume":"386","author":"Rosenberg","year":"2022","journal-title":"N. Engl. J. Med."},{"key":"B158","doi-asserted-by":"publisher","first-page":"627","DOI":"10.1358\/dot.2019.55.10.3045038","article-title":"Viltolarsen for the treatment of Duchenne muscular dystrophy","volume":"55","author":"Roshmi","year":"2019","journal-title":"Drugs Today Barc. Spain"},{"key":"B159","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1038\/s41587-021-00933-4","article-title":"In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels","volume":"39","author":"Rothgangl","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"B160","doi-asserted-by":"publisher","first-page":"656","DOI":"10.1016\/j.jcf.2023.04.008","article-title":"Inhaled mRNA therapy for treatment of cystic fibrosis: interim results of a randomized, double-blind, placebo-controlled phase 1\/2 clinical study","author":"Rowe","year":"2023","journal-title":"J. Cyst. Fibros. Off. J. Eur. Cyst. Fibros. Soc."},{"key":"B161","doi-asserted-by":"publisher","first-page":"1110","DOI":"10.1200\/JCO.2004.10.148","article-title":"Phase I study of G3139, a bcl-2 antisense oligonucleotide, combined with carboplatin and etoposide in patients with small-cell lung cancer","volume":"22","author":"Rudin","year":"2004","journal-title":"J. Clin. Oncol."},{"key":"B162","doi-asserted-by":"publisher","first-page":"539","DOI":"10.1093\/annonc\/mdf124","article-title":"A pilot trial of G3139, a bcl-2 antisense oligonucleotide, and paclitaxel in patients with chemorefractory small-cell lung cancer","volume":"13","author":"Rudin","year":"2002","journal-title":"Ann. Oncol. Off. J. Eur. Soc. Med. Oncol."},{"key":"B163","doi-asserted-by":"publisher","first-page":"870","DOI":"10.1200\/JCO.2007.14.3461","article-title":"Randomized phase II study of carboplatin and etoposide with or without the bcl-2 antisense oligonucleotide oblimersen for extensive-stage small-cell lung cancer: CALGB 30103","volume":"26","author":"Rudin","year":"2008","journal-title":"J. Clin. Oncol."},{"key":"B164","doi-asserted-by":"publisher","first-page":"15","DOI":"10.3390\/ncrna7010015","article-title":"Endogenous double-stranded RNA","volume":"7","author":"Sadeq","year":"2021","journal-title":"Non-Coding RNA"},{"key":"B165","doi-asserted-by":"publisher","first-page":"222","DOI":"10.1038\/nature23003","article-title":"Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer","volume":"547","author":"Sahin","year":"2017","journal-title":"Nature"},{"key":"B166","doi-asserted-by":"publisher","first-page":"3852","DOI":"10.1073\/pnas.73.11.3852","article-title":"Viroids are single-stranded covalently closed circular RNA molecules existing as highly base-paired rod-like structures","volume":"73","author":"Sanger","year":"1976","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B167","doi-asserted-by":"publisher","first-page":"1319","DOI":"10.4161\/rna.22269","article-title":"Developing mRNA-vaccine technologies","volume":"9","author":"Schlake","year":"2012","journal-title":"RNA Biol."},{"key":"B168","doi-asserted-by":"publisher","first-page":"1513","DOI":"10.1038\/mt.2016.143","article-title":"Viral and synthetic RNA vector technologies and applications","volume":"24","author":"Schott","year":"2016","journal-title":"Mol. Ther."},{"key":"B169","doi-asserted-by":"publisher","first-page":"69","DOI":"10.1126\/science.aaa4971","article-title":"Neoantigens in cancer immunotherapy","volume":"348","author":"Schumacher","year":"2015","journal-title":"Science"},{"key":"B170","doi-asserted-by":"publisher","first-page":"799","DOI":"10.1007\/s00262-019-02315-x","article-title":"A phase I\/IIa study of the mRNA-based cancer immunotherapy CV9201 in patients with stage IIIB\/IV non-small cell lung cancer","volume":"68","author":"Sebastian","year":"2019","journal-title":"Cancer Immunol. Immunother."},{"key":"B171","doi-asserted-by":"publisher","first-page":"172","DOI":"10.1038\/nbt.1602","article-title":"Rational design of cationic lipids for siRNA delivery","volume":"28","author":"Semple","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"B172","doi-asserted-by":"publisher","first-page":"536","DOI":"10.1016\/j.jcf.2018.10.015","article-title":"Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis","volume":"18","author":"Sermet-Gaudelus","year":"2019","journal-title":"J. Cyst. Fibros."},{"key":"B173","first-page":"83","article-title":"MicroRNAs: processing, maturation, target recognition and regulatory functions","volume":"3","author":"Shukla","year":"2011","journal-title":"Mol. Cell. Pharmacol."},{"key":"B174","doi-asserted-by":"publisher","first-page":"1665","DOI":"10.1016\/j.ymthe.2019.05.020","article-title":"MicroRNA-215-5p treatment suppresses mesothelioma progression via the MDM2-p53-signaling Axis","volume":"27","author":"Singh","year":"2019","journal-title":"Mol. Ther."},{"key":"B175","doi-asserted-by":"publisher","first-page":"669","DOI":"10.1016\/j.omtn.2021.04.001","article-title":"MicroRNA-206 suppresses mesothelioma progression via the Ras signaling axis","volume":"24","author":"Singh","year":"2021","journal-title":"Mol. Ther. - Nucleic Acids"},{"key":"B176","doi-asserted-by":"publisher","first-page":"153","DOI":"10.1016\/j.addr.2007.03.006","article-title":"RNA interference and innate immunity","volume":"59","author":"Sioud","year":"2007","journal-title":"Adv. Drug Deliv. Rev."},{"key":"B177","doi-asserted-by":"publisher","first-page":"5478","DOI":"10.1073\/pnas.0508035103","article-title":"Gene expression patterns in human placenta","volume":"103","author":"Sood","year":"2006","journal-title":"Proc. Natl. Acad. Sci."},{"key":"B178","doi-asserted-by":"publisher","first-page":"e1409","DOI":"10.1634\/theoncologist.2018-0518","article-title":"A randomized, double-blinded, phase II trial of carboplatin and pemetrexed with or without apatorsen (OGX\u2010427) in patients with previously untreated stage IV non-squamous-non-small-cell lung cancer: the SPRUCE trial","volume":"24","author":"Spigel","year":"2019","journal-title":"Oncol."},{"key":"B179","doi-asserted-by":"publisher","first-page":"774","DOI":"10.1021\/mp100390w","article-title":"In vitro and in vivo mRNA delivery using lipid-enveloped pH-responsive polymer nanoparticles","volume":"8","author":"Su","year":"2011","journal-title":"Mol. Pharm."},{"key":"B180","doi-asserted-by":"publisher","first-page":"259","DOI":"10.1016\/j.ejps.2012.10.014","article-title":"Therapeutic RNA aptamers in clinical trials","volume":"48","author":"Sundaram","year":"2013","journal-title":"Eur. J. Pharm. Sci. Off. J. Eur. Fed. Pharm. Sci."},{"key":"B181","doi-asserted-by":"publisher","first-page":"82","DOI":"10.1016\/j.jconrel.2019.08.004","article-title":"Mechanistic profiling of the release kinetics of siRNA from lipidoid-polymer hybrid nanoparticles in vitro and in vivo after pulmonary administration","volume":"310","author":"Thanki","year":"2019","journal-title":"J. Control. Release"},{"key":"B182","doi-asserted-by":"publisher","first-page":"1761","DOI":"10.1056\/NEJMoa2110345","article-title":"Safety and efficacy of the BNT162b2 mRNA covid-19 vaccine through 6 months","volume":"385","author":"Thomas","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"B183","doi-asserted-by":"publisher","first-page":"1355","DOI":"10.1056\/NEJMoa2110362","article-title":"Effectiveness of covid-19 vaccines in ambulatory and inpatient care settings","volume":"385","author":"Thompson","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"B184","doi-asserted-by":"publisher","first-page":"644718","DOI":"10.3389\/fphar.2021.644718","article-title":"Insight into the prospects for RNAi therapy of cancer","volume":"12","author":"Tian","year":"2021","journal-title":"Front. Pharmacol."},{"key":"B185","doi-asserted-by":"publisher","first-page":"5712","DOI":"10.1038\/sj.onc.1206733","article-title":"Use of adeno-associated viral vector for delivery of small interfering RNA","volume":"22","author":"Tomar","year":"2003","journal-title":"Oncogene"},{"key":"B186","doi-asserted-by":"publisher","first-page":"505","DOI":"10.1126\/science.2200121","article-title":"Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase","volume":"249","author":"Tuerk","year":"1990","journal-title":"Science"},{"key":"B187","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1007\/s10637-005-6736-x","article-title":"Randomized phase II evaluation of aprinocarsen in combination with gemcitabine and cisplatin for patients with advanced\/metastatic non-small cell lung cancer","volume":"23","author":"Vansteenkiste","year":"2005","journal-title":"Invest. New Drugs"},{"key":"B188","doi-asserted-by":"publisher","first-page":"1386","DOI":"10.1016\/S1470-2045(17)30621-6","article-title":"Safety and activity of microRNA-loaded minicells in patients with recurrent malignant pleural mesothelioma: a first-in-man, phase 1, open-label, dose-escalation study","volume":"18","author":"van Zandwijk","year":"2017","journal-title":"Lancet Oncol."},{"key":"B189","doi-asserted-by":"publisher","first-page":"e2117198119","DOI":"10.1073\/pnas.2117198119","article-title":"Targeting stem-loop 1 of the SARS-CoV-2 5\u2032 UTR to suppress viral translation and Nsp1 evasion","volume":"119","author":"Vora","year":"2022","journal-title":"Proc. Natl. Acad. Sci."},{"key":"B190","doi-asserted-by":"publisher","first-page":"2439","DOI":"10.1056\/NEJMoa2027906","article-title":"Safety and immunogenicity of two RNA-based covid-19 vaccine candidates","volume":"383","author":"Walsh","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"B191","doi-asserted-by":"publisher","first-page":"645297","DOI":"10.3389\/fchem.2021.645297","article-title":"pH-Responsive amphiphilic carboxylate polymers: design and potential for endosomal escape","volume":"9","author":"Wang","year":"2021","journal-title":"Front. Chem."},{"key":"B192","doi-asserted-by":"publisher","first-page":"265","DOI":"10.1038\/s41596-022-00755-x","article-title":"Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery","volume":"18","author":"Wang","year":"2023","journal-title":"Nat. Protoc."},{"key":"B193","doi-asserted-by":"publisher","first-page":"4455","DOI":"10.1073\/pnas.96.8.4455","article-title":"A mouse model for achondroplasia produced by targeting fibroblast growth factor receptor 3","volume":"96","author":"Wang","year":"1999","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B194","doi-asserted-by":"publisher","first-page":"842","DOI":"10.1016\/j.drudis.2008.05.007","article-title":"Chemically modified siRNA: tools and applications","volume":"13","author":"Watts","year":"2008","journal-title":"Drug Discov. Today"},{"key":"B195","doi-asserted-by":"publisher","first-page":"3232","DOI":"10.1038\/s41467-020-17029-3","article-title":"Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing","volume":"11","author":"Wei","year":"2020","journal-title":"Nat. Commun."},{"key":"B196","doi-asserted-by":"publisher","first-page":"855","DOI":"10.1016\/0092-8674(93)90530-4","article-title":"Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans","volume":"75","author":"Wightman","year":"1993","journal-title":"Cell"},{"key":"B197","doi-asserted-by":"publisher","first-page":"e1478","DOI":"10.1002\/wrna.1478","article-title":"A 360\u00b0 view of circular RNAs: from biogenesis to functions","volume":"9","author":"Wilusz","year":"2018","journal-title":"Wiley Interdiscip. Rev. RNA"},{"key":"B198","doi-asserted-by":"publisher","first-page":"629","DOI":"10.1038\/s41573-021-00219-z","article-title":"Noncoding RNA therapeutics \u2014 challenges and potential solutions","volume":"20","author":"Winkle","year":"2021","journal-title":"Nat. Rev. Drug Discov."},{"key":"B199","doi-asserted-by":"publisher","first-page":"1465","DOI":"10.1126\/science.1690918","article-title":"Direct gene transfer into mouse muscle in vivo","volume":"247","author":"Wolff","year":"1990","journal-title":"Science"},{"key":"B200","doi-asserted-by":"publisher","first-page":"7723","DOI":"10.1073\/pnas.96.14.7723","article-title":"Transcriptional and posttranslational plasticity and the generation of inflammatory pain","volume":"96","author":"Woolf","year":"1999","journal-title":"Proc. Natl. Acad. Sci."},{"key":"B201","doi-asserted-by":"publisher","first-page":"280","DOI":"10.1073\/pnas.75.1.280","article-title":"Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide","volume":"75","author":"Zamecnik","year":"1978","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B202","doi-asserted-by":"publisher","first-page":"1445","DOI":"10.1164\/rccm.201910-2041OC","article-title":"Long noncoding RNA TYKRIL plays a role in pulmonary hypertension via the p53-mediated regulation of PDGFR\u03b2","volume":"202","author":"Zehendner","year":"2020","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"B203","doi-asserted-by":"publisher","first-page":"2434","DOI":"10.1016\/j.cell.2022.05.022","article-title":"Humoral and cellular immune memory to four COVID-19 vaccines","volume":"185","author":"Zhang","year":"2022","journal-title":"Cell"},{"key":"B204","doi-asserted-by":"publisher","first-page":"644","DOI":"10.1038\/s41419-022-05075-2","article-title":"RNA-based therapeutics: an overview and prospectus","volume":"13","author":"Zhu","year":"2022","journal-title":"Cell Death Dis."}],"container-title":["Frontiers in Genetics"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fgene.2023.1281538\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,23]],"date-time":"2023-11-23T11:17:21Z","timestamp":1700738241000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fgene.2023.1281538\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,11,23]]},"references-count":205,"alternative-id":["10.3389\/fgene.2023.1281538"],"URL":"https:\/\/doi.org\/10.3389\/fgene.2023.1281538","relation":{},"ISSN":["1664-8021"],"issn-type":[{"value":"1664-8021","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,11,23]]},"article-number":"1281538"}}