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ImmunoTherapy Cancer"},{"key":"10.1016\/j.ymthe.2025.03.034_bib68","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1056\/NEJMoa2412309","article-title":"CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy","volume":"391","author":"Fontana","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.03.034_bib69","doi-asserted-by":"crossref","first-page":"5357","DOI":"10.1007\/s12274-022-5254-x","article-title":"A lipid-based LMP2-mRNA vaccine to treat nasopharyngeal carcinoma","volume":"16","author":"Guo","year":"2023","journal-title":"Nano Res."},{"key":"10.1016\/j.ymthe.2025.03.034_bib70","article-title":"134 mNTX-250, a novel multimodal HPV-16 mRNA-based therapeutic, induces potent anti-tumor responses and establishes HPV-16 specific immune memory","volume":"11","author":"Li","year":"2023","journal-title":"J. ImmunoTherapy Cancer"},{"key":"10.1016\/j.ymthe.2025.03.034_bib71","doi-asserted-by":"crossref","first-page":"2521","DOI":"10.1016\/j.cell.2024.04.003","article-title":"RNA aggregates harness the danger response for potent cancer immunotherapy","volume":"187","author":"Mendez-Gomez","year":"2024","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.034_bib72","doi-asserted-by":"crossref","DOI":"10.1002\/jmv.28924","article-title":"Advances in mRNA vaccines for viral diseases","volume":"95","author":"Huang","year":"2023","journal-title":"J. Med. Virol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib73","doi-asserted-by":"crossref","DOI":"10.1002\/jmv.28572","article-title":"mRNA vaccines: The future of prevention of viral infections?","volume":"95","author":"Rzymski","year":"2023","journal-title":"J. Med. Virol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib74","article-title":"New Kids on the Block: RNA-Based Influenza Virus Vaccines","volume":"6","author":"Scorza","year":"2018","journal-title":"Vaccines (Basel)"},{"key":"10.1016\/j.ymthe.2025.03.034_bib75","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1016\/S1473-3099(23)00650-3","article-title":"Immunogenicity and safety of a booster dose of a self-amplifying RNA COVID-19 vaccine (ARCT-154) versus BNT162b2 mRNA COVID-19 vaccine: a double-blind, multicentre, randomised, controlled, phase 3, non-inferiority trial","volume":"24","author":"Oda","year":"2024","journal-title":"Lancet Infect. Dis."},{"key":"10.1016\/j.ymthe.2025.03.034_bib76","doi-asserted-by":"crossref","first-page":"e729","DOI":"10.1016\/S1473-3099(24)00615-7","article-title":"12-month persistence of immune responses to self-amplifying mRNA COVID-19 vaccines: ARCT-154 versus BNT162b2 vaccine","volume":"24","author":"Oda","year":"2024","journal-title":"Lancet Infect. Dis."},{"key":"10.1016\/j.ymthe.2025.03.034_bib77","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1038\/s41434-020-00204-y","article-title":"Self-amplifying RNA vaccines for infectious diseases","volume":"28","author":"Bloom","year":"2021","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.034_bib78","doi-asserted-by":"crossref","DOI":"10.1016\/j.vaccine.2024.126408","article-title":"Advancing influenza vaccines: A review of next-generation candidates and their potential for global health impact","volume":"42","author":"Taaffe","year":"2024","journal-title":"Vaccine"},{"key":"10.1016\/j.ymthe.2025.03.034_bib79","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1186\/s43141-023-00581-y","article-title":"Influenza vaccine: a review on current scenario and future prospects","volume":"21","author":"Gupta","year":"2023","journal-title":"J. Genet. Eng. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib80","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1089\/vim.2017.0129","article-title":"Antibody Immunodominance: The Key to Understanding Influenza Virus Antigenic Drift","volume":"31","author":"Altman","year":"2018","journal-title":"Viral Immunol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib81","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1038\/s41541-023-00752-5","article-title":"Seasonal quadrivalent mRNA vaccine prevents and mitigates influenza infection","volume":"8","author":"Kackos","year":"2023","journal-title":"NPJ Vaccin."},{"key":"10.1016\/j.ymthe.2025.03.034_bib82","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0297833","article-title":"mRNA vaccines encoding influenza virus hemagglutinin (HA) elicits immunity in mice from influenza A virus challenge","volume":"19","author":"Reneer","year":"2024","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2025.03.034_bib83","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2024.1381508","article-title":"Trivalent mRNA vaccine-candidate against seasonal flu with cross-specific humoral immune response","volume":"15","author":"Mazunina","year":"2024","journal-title":"Front. Immunol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib84","doi-asserted-by":"crossref","first-page":"10435","DOI":"10.1128\/JVI.01715-13","article-title":"Hemagglutinin stalk-based universal vaccine constructs protect against group 2 influenza A viruses","volume":"87","author":"Margine","year":"2013","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib85","first-page":"301","article-title":"Advances in universal influenza virus vaccine design and antibody mediated therapies based on conserved regions of the hemagglutinin","volume":"386","author":"Krammer","year":"2015","journal-title":"Curr. Top. Microbiol. 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Commun."},{"key":"10.1016\/j.ymthe.2025.03.034_bib93","doi-asserted-by":"crossref","first-page":"446","DOI":"10.1016\/j.ymthe.2017.11.017","article-title":"Self-Amplifying RNA Vaccines Give Equivalent Protection against Influenza to mRNA Vaccines but at Much Lower Doses","volume":"26","author":"Vogel","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.034_bib94","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/j.omtm.2022.09.013","article-title":"Self-amplifying mRNA bicistronic influenza vaccines raise cross-reactive immune responses in mice and prevent infection in ferrets","volume":"27","author":"Chang","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.034_bib95","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1038\/s41541-023-00747-2","article-title":"Self-amplifying mRNA seasonal influenza vaccines elicit mouse neutralizing antibody and cell-mediated immunity and protect ferrets","volume":"8","author":"Cheung","year":"2023","journal-title":"NPJ Vaccin."},{"key":"10.1016\/j.ymthe.2025.03.034_bib96","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1126\/science.aav9033","article-title":"A proof of concept for structure-based vaccine design targeting RSV in humans","volume":"365","author":"Crank","year":"2019","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.034_bib97","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/j.cytogfr.2022.10.001","article-title":"Development of mRNA vaccines against respiratory syncytial virus (RSV)","volume":"68","author":"Qiu","year":"2022","journal-title":"Cytokine Growth Factor Rev."},{"key":"10.1016\/j.ymthe.2025.03.034_bib98","unstructured":"WHO. WHO Director-General declares mpox outbreak a public health emergency of international concern. https:\/\/www.who.int\/news\/item\/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern. 2024. Accessed 11\/2024."},{"key":"10.1016\/j.ymthe.2025.03.034_bib99","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1038\/s41392-024-02058-x","article-title":"Next-generation mpox vaccines: efficacy of mRNA-1769 compared to modified vaccinia virus Ankara in non-human primates","volume":"9","author":"Mayer","year":"2024","journal-title":"Signal Transduct. Target. 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Dis."},{"key":"10.1016\/j.ymthe.2025.03.034_bib107","doi-asserted-by":"crossref","first-page":"1330","DOI":"10.1016\/S1473-3099(23)00361-4","article-title":"The quest for a cytomegalovirus vaccine continues","volume":"23","author":"Chiuppesi","year":"2023","journal-title":"Lancet Infect. Dis."},{"key":"10.1016\/j.ymthe.2025.03.034_bib108","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1038\/nature21428","article-title":"Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination","volume":"543","author":"Pardi","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.034_bib109","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1038\/s41541-023-00656-4","article-title":"An optimized messenger RNA vaccine candidate protects non-human primates from Zika virus infection","volume":"8","author":"Bollman","year":"2023","journal-title":"NPJ Vaccin."},{"key":"10.1016\/j.ymthe.2025.03.034_bib110","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1093\/infdis\/jiad593","article-title":"Human Cytomegalovirus mRNA-1647 Vaccine Candidate Elicits Potent and Broad Neutralization and Higher Antibody-Dependent Cellular Cytotoxicity Responses Than the gB\/MF59 Vaccine","volume":"230","author":"Hu","year":"2024","journal-title":"J. Infect. Dis."},{"key":"10.1016\/j.ymthe.2025.03.034_bib111","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1186\/2042-4280-3-5","article-title":"Herpes simplex virus and varicella zoster virus, the house guests who never leave","volume":"3","author":"Kinchington","year":"2012","journal-title":"Herpesviridae"},{"key":"10.1016\/j.ymthe.2025.03.034_bib112","doi-asserted-by":"crossref","first-page":"1015","DOI":"10.1016\/S1474-4422(07)70267-3","article-title":"The neurotropic herpes viruses: herpes simplex and varicella-zoster","volume":"6","author":"Steiner","year":"2007","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2025.03.034_bib113","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1111\/ajt.12003","article-title":"Varicella zoster virus (VZV) and herpes simplex virus (HSV) in solid organ transplant patients","volume":"13","author":"Zuckerman","year":"2013","journal-title":"Am. J. 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Accessed 11\/2024."},{"key":"10.1016\/j.ymthe.2025.03.034_bib127","unstructured":"Moderna Announces Positive Phase 3 Data for Combination Vaccine Against Influenza and COVID-19. https:\/\/investors.modernatx.com\/news\/news-details\/2024\/Moderna-Announces-Positive-Phase-3-Data-for-Combination-Vaccine-Against-Influenza-and-COVID-19-\/default.aspx. Accessed 11\/2024."},{"key":"10.1016\/j.ymthe.2025.03.034_bib128","unstructured":"WHO. Glob. tuberculosis Rep. 2022. https:\/\/www.who.int\/teams\/global-tuberculosis-programme\/tb-reports\/global-tuberculosis-report-2022\/tb-disease-burden\/2-2-tb-mortality.2022. Accessed 11\/2024."},{"key":"10.1016\/j.ymthe.2025.03.034_bib129","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1038\/s41585-021-00481-1","article-title":"100 years of Bacillus Calmette-Guerin immunotherapy: from cattle to COVID-19","volume":"18","author":"Lobo","year":"2021","journal-title":"Nat. Rev. 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