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|a mRNA-based therapeutics /
|c Edited by Fernando Aranda, Pedro Berraondo, Lorenzo Galluzzi.
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|a Cambridge, MA :
|b Academic Press,
|c 2022.
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|a International review of cell and molecular biology ;
|v Volume 372
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|a Includes bibliographical references.
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|a Intro -- mRNA-Based Therapeutics -- Copyright -- Contents -- Contributors -- Chapter One: mRNA-based therapies: Preclinical and clinical applications -- 1. Introduction -- 2. Chemical modifications -- 2.1. Improvement of IVT mRNA stability and translation efficiency -- 2.1.1. 5-cap and 3-poly(A) tail -- 2.1.2. 5- and 3-UTRs -- 2.1.3. The coding region -- 2.2. Immunostimulatory properties of IVT mRNA -- 3. Preclinical and clinical applications of IVT mRNA -- 3.1. Vaccines against infectious diseases -- 3.2. Cancer immunotherapy -- 3.2.1. Cancer vaccines -- 3.2.2. mRNA-encoded immunomodulators -- 3.2.3. mRNA-engineered immune cells -- 3.2.4. mRNA-encoded antibodies -- 3.3. Autoimmunity -- 3.3.1. Non-inflammatory autoantigen vaccines -- 3.3.2. mRNA-encoded regulatory factors -- 3.4. mRNA-encoded protein replacement -- 3.5. Gene editing -- 3.6. Cell reprogramming and therapeutic cell generation -- 4. Conclusions and future perspectives -- Conflict of interest disclosure -- References -- Chapter Two: Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases -- 1. Introduction -- 1.1. mRNA therapy development -- 2. mRNA therapeutics vs other advanced therapies for liver metabolic diseases -- 2.1. Acute intermittent porphyria -- 2.2. Phenylketonuria -- 2.3. Classic galactosemia -- 2.4. Liver arginase deficiency -- 2.5. Glycogen storage diseases -- 2.5.1. Glycogen storage disease type Ia -- 2.5.2. Glycogen storage disease type 3 -- 2.6. AAT deficiency -- 2.7. Crigler-Najjar syndrome type 1 -- 2.8. Propionic acidemia -- 2.9. Ornithine transcarbamylase deficiency -- 2.10. Methylmalonic acidemia -- 2.11. Primary hyperoxaluria -- 2.12. Fabry disease -- 3. Conclusions and perspectives -- Acknowledgment -- Funding -- Competing interest -- References -- Chapter Three: Applications of self-replicating RNA -- 1. Introduction.
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8 |
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|a 2. Self-replicating RNA vectors -- 2.1. Viral vectors -- 2.2. RNA vectors -- 2.3. DNA vectors -- 3. Self-replicating RNA for infectious diseases -- 3.1. Alphaviruses -- 3.2. Arenaviruses -- 3.3. Filoviruses -- 3.4. Flaviviruses -- 3.5. Hepatocytic viruses -- 3.6. Lentiviruses -- 3.7. Influenza virus and orthopneumoviruses -- 3.8. Coronaviruses -- 3.9. Bacterial and parasitic targets -- 4. Self-replicating RNA for cancer therapy -- 4.1. Brain cancer -- 4.2. Breast cancer -- 4.3. Cervical cancer -- 4.4. Colon cancer -- 4.5. Lung cancer -- 4.6. Melanoma -- 4.7. Ovarian cancer -- 4.8. Pancreatic cancer -- 4.9. Prostate cancer -- 5. Conclusions -- References -- Chapter Four: Present and future of lipid nanoparticle-mRNA technology in phenylketonuria disease treatment -- 1. Introduction -- 2. Replacement therapies for PKU -- 3. Emerging strategy: Genome editing on PKU -- 4. Future perspective -- Acknowledgments and declaration of interests -- References -- Further reading -- Chapter Five: RNA gene editing in the eye and beyond: The neglected tool of the gene editing armatorium? -- 1. Introduction -- 2. The advent of ADAR -- 2.1. ADAR: A natural RNA editor -- 2.2. Developing ADAR editing for therapeutics -- 2.2.1. ADAR-SNAP -- 2.2.2. GluR2-ADAR (GRIA2, GluRB) -- 2.2.2.1. Endogeneous ADAR -- 2.2.2.2. Exogeneous ADAR -- 2.2.3. ADAR bacteriophage effectors -- 2.2.3.1. ADAR BoxB -- 2.2.3.2. ADAR MS2-MCP -- 2.2.4. Cas based systems -- 2.2.4.1. Cas13-ADAR2-DD -- 2.2.4.2. A programmable cytidine deaminase -- 2.2.4.3. CIRTS -- 2.2.4.4. Cas7-11 -- 2.2.5. Leaper -- 3. RNA editing in the eye -- 3.1. The eye as a site for RNA editing -- 3.2. Potential targets -- 4. Challenges and pitfalls -- 4.1. Off-targets -- 4.1.1. Tumorigenesis -- 4.1.2. Immunogenicity -- 4.1.3. Tackling off-targets -- 4.2. Editing limitations -- 4.3. Delivery challenges.
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| 505 |
8 |
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|a 4.3.1. Packaging limitations -- 4.3.2. The need for retreatment -- 4.3.2.1. Safety concerns -- 4.3.2.2. Costs -- 5. Conclusion -- Acknowledgments -- References -- Chapter Six: mRNA delivery technologies: Toward clinical translation -- 1. Introduction -- 2. Synthetic mRNA -- 2.1. Synthetic mRNA structure -- 2.2. Synthetic mRNA modifications -- 3. mRNA delivery systems -- 3.1. Physical administration methods -- 3.2. Nucleic acid delivery systems -- 3.2.1. Polymeric delivery systems -- 3.2.2. Polypeptidic delivery systems -- 3.2.3. Dendrimers -- 3.2.4. Gold nanoparticles -- 3.2.5. Lipidic systems -- 3.2.5.1. Components of lipidic systems -- 3.2.5.2. Lipid-based nanocarriers -- 4. Clinical applications of mRNA -- 4.1. Gene editing -- 4.2. Immunotherapy -- 4.2.1. mRNA vaccines against infectious diseases -- 4.2.2. Cancer immunotherapy -- 4.3. Protein replacement therapy -- 5. Conclusions -- Acknowledgments -- References -- Chapter Seven: Advances in mRNA vaccines -- 1. Messenger RNA synthesis and modification for vaccine -- 1.1. 5Cap structure -- 1.2. UTRs -- 1.3. Poly(A) tail -- 1.4. Nucleoside modification -- 1.5. Self-amplifying RNA -- 2. Carrier-mediated mRNA vaccine delivery -- 2.1. Lipid-based delivery -- 2.2. Polymer-based delivery -- 2.3. Peptide-based delivery -- 2.4. Pseudovirus-based particles -- 3. mRNA vaccines for clinical applications -- References.
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|t MRNA-BASED THERAPEUTICS.
|d [S.l.] : ELSEVIER ACADEMIC PRESS, 2022
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|a International review of cell and molecular biology ;
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