mRNA-based therapeutics /

Detalles Bibliográficos
Clasificación:Libro Electrónico
Otros Autores: Aranda, Fernando, Berraondo, Pedro, Galluzzi, Lorenzo
Formato: Electrónico eBook
Idioma:Inglés
Publicado: Cambridge, MA : Academic Press, 2022.
Colección:International review of cell and molecular biology ; Volume 372.
Temas:
Messenger RNA.
Therapeutics.
Th�erapeutique.
treating (health care function)
Messenger RNA
Therapeutics
Acceso en línea:Texto completo
Tabla de Contenidos:
  • 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.
  • 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.
  • 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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