001449749 000__ 06317cam\a2200589\i\4500 001449749 001__ 1449749 001449749 003__ OCoLC 001449749 005__ 20230310004416.0 001449749 006__ m\\\\\o\\d\\\\\\\\ 001449749 007__ cr\cn\nnnunnun 001449749 008__ 220925s2022\\\\sz\a\\\\o\\\\\000\0\eng\d 001449749 019__ $$a1345589457 001449749 020__ $$a9783031084157$$q(electronic bk.) 001449749 020__ $$a3031084152$$q(electronic bk.) 001449749 020__ $$z9783031084140 001449749 020__ $$z3031084144 001449749 0247_ $$a10.1007/978-3-031-08415-7$$2doi 001449749 035__ $$aSP(OCoLC)1345580679 001449749 040__ $$aYDX$$beng$$erda$$epn$$cYDX$$dGW5XE$$dEBLCP$$dN$T$$dOCLCQ 001449749 049__ $$aISEA 001449749 050_4 $$aRM263 001449749 08204 $$a615.5/8$$223/eng/20221004 001449749 24500 $$aMessenger RNA therapeutics /$$cStefan Jurga, Jan Barciszewski, editors. 001449749 264_1 $$aCham :$$bSpringer,$$c[2022] 001449749 264_4 $$c©2022 001449749 300__ $$a1 online resource (xii, 446 pages) :$$billustrations (chiefly color). 001449749 336__ $$atext$$btxt$$2rdacontent 001449749 337__ $$acomputer$$bc$$2rdamedia 001449749 338__ $$aonline resource$$bcr$$2rdacarrier 001449749 4901_ $$aRNA technologies ;$$vvolume 13 001449749 5050_ $$aIntro -- Introduction: IVT Messenger RNA in the Syringe -- Contents -- Roadmap to the Development of mRNA Therapeutics: From Molecule Design and Delivery Strategies to Manufacturing, Quality Control, and Regulatory Considerations -- 1 Introduction -- 2 mRNA Design Strategies -- 2.1 Reducing mRNA Immunogenicity -- 2.2 Optimizing Protein Expression -- 2.3 Optimizing mRNA Stability -- 3 Delivery Strategies for mRNA Therapeutics -- 3.1 Lipid Nanoparticles for mRNA Delivery -- 3.2 Other Materials for mRNA Delivery -- 3.3 mRNA Drug Product Route of Administration 001449749 5058_ $$a4 Manufacturing of mRNA Drug Products -- 4.1 mRNA Drug Substance Manufacturing -- 4.2 mRNA Drug Product Manufacturing -- 5 Quality Control of mRNA Therapeutics -- 5.1 Drug Substance (mRNA) CQAs and Quality Control Strategy -- 5.2 Drug Product (mRNA-LNPs) CQAs and Quality Control Strategy -- 6 Conclusion and Future Directions -- References -- Messenger RNA for Prophylaxis -- 1 Introduction -- 2 mRNA Vaccines: Categories and Biological Function -- 3 Advances in mRNA Constructs -- 3.1 5' Capping -- 3.2 Untranslated Regions (UTRs) -- 3.3 Poly(A)-Tail 001449749 5058_ $$a3.4 Nucleotide Modification and Codon Optimization -- 3.5 Purity -- 3.6 Self-Amplifying Specific Features -- 4 mRNA Carrier Technologies -- 4.1 Tropism and Uptake Efficiency -- 4.2 The Art of Endosomal Escape -- 4.3 Formulation -- 4.4 Stability -- 5 Conclusions -- References -- Messenger RNA Therapeutics: Start of a New Era in Medicine -- 1 Introduction -- 2 Production of IVT mRNA -- 3 Immunogenicity of IVT mRNA -- 4 Strategies to Increase the Stability and Reduce the Immunogenicity of IVT mRNA -- 4.1 Capping (m7GpppN or m7Gp3N) -- 4.2 Tailing -- 4.3 Untranslated Regions (UTRs) 001449749 5058_ $$a4.4 Coding Region -- 5 Purification of Synthetic mRNA -- 6 Synthetic mRNA Platforms and their Features -- 6.1 Unmodified mRNA -- 6.2 Modified mRNA -- 6.3 Sequence-Optimized Unmodified mRNA -- 6.4 Replicon RNA -- 7 In Vivo Delivery Strategies of Exogenous mRNA -- 7.1 Delivery of Naked mRNA -- 7.2 Cationic Liposome-Mediated or Cationic Nanoemulsion (CNE)-based RNA Transfection -- 7.3 Peptide-based Delivery -- 7.4 Electroporation and Nucleoporation -- 7.5 Gene Gun-Mediated Delivery of mRNA -- 7.6 Use of Polymer Nanomaterials -- 7.7 Virus-like Replicon Particle (VRP)-based Delivery of mRNA 001449749 5058_ $$a7.8 Other Lesser-Known Methods -- 8 Applications of mRNA Therapeutics -- 8.1 mRNA as a Therapeutic Agent for Replacement of Defective Protein within the Cell -- 8.2 mRNA as Vaccines Against Cancer -- 8.3 Dendritic Cell (DC) Vaccines -- 8.4 mRNA Vaccines in Prevention of Diseases -- 8.5 mRNA-Mediated Genome Editing -- 8.6 Generation of Induced Pluripotent Stem Cells (iPSCs) using mRNA -- 9 Safety of mRNA Therapeutics -- 9.1 The Safety Concern Over the Use of Non-Natural Nucleotides/Nucleosides in IVT mRNA -- 9.2 Safety Considerations Regarding the Encoded Protein -- 10 Conclusions -- References 001449749 506__ $$aAccess limited to authorized users. 001449749 520__ $$aThis book focuses on the fundamentals and applications of messenger RNA (mRNA)-based therapeutics and discusses the strengths and key challenges of this emerging class of drugs. In the past 30 years, extensive research and technological development in many areas have contributed to the emergence of in vitro transcribed mRNA as a therapeutic that has now reached clinical testing. Formulations that protect the mRNA from nucleases and accelerate its cellular uptake, combined with improvements to the mRNA molecules themselves, have been critical advancements for mRNAs to become viable therapeutics. Though once regarded as a serious impediment, the transient nature of mRNA technology is now considered a major advantage in making mRNA therapies safe and, ultimately, a potential game changer in the field of medicine. This new book in the RNA Technologies series provides a state-of-the-art overview on the emerging field of mRNA therapeutics covering essential strategies for formulation, delivery, and application. It also reviews the promising role in cancer immunotherapy, respiratory diseases, and chronic HBV infection and discusses RNA vaccines in light of the current COVID-19 pandemic. mRNA-based approaches have great potential to revolutionize molecular biology, cell biology, biomedical research, and medicine. Thus, this handbook is an essential resource for researchers in academia and industry contributing to the development of this new area of therapeutics. 001449749 588__ $$aOnline resource; title from PDF title page (SpringerLink, viewed October 4, 2022). 001449749 650_0 $$aChemotherapy. 001449749 650_0 $$amRNA vaccines. 001449749 650_0 $$aMessenger RNA. 001449749 655_0 $$aElectronic books. 001449749 7001_ $$aJurga, Stefan,$$eeditor.$$1https://isni.org/isni/0000000071964270 001449749 7001_ $$aBarciszewski, Jan,$$eeditor.$$1https://isni.org/isni/0000000081568570 001449749 77608 $$iPrint version:$$z3031084144$$z9783031084140$$w(OCoLC)1317834019 001449749 830_0 $$aRNA technologies ;$$vv. 13. 001449749 852__ $$bebk 001449749 85640 $$3Springer Nature$$uhttps://univsouthin.idm.oclc.org/login?url=https://link.springer.com/10.1007/978-3-031-08415-7$$zOnline Access$$91397441.1 001449749 909CO $$ooai:library.usi.edu:1449749$$pGLOBAL_SET 001449749 980__ $$aBIB 001449749 980__ $$aEBOOK 001449749 982__ $$aEbook 001449749 983__ $$aOnline 001449749 994__ $$a92$$bISE