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mRNA-based therapeutics /

MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New er...

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Bibliographic Details
Corporate Author: ScienceDirect (Online service)
Other Authors: Aranda, Fernando, Berraondo, Pedro, Galluzzi, Lorenzo
Format: eBook
Language:English
Published: Cambridge, MA : Academic Press, 2022.
Series:International review of cell and molecular biology ; Volume 372.
Subjects:
Messenger RNA.
Therapeutics.
Thérapeutique.
treating (health care function)
Messenger RNA
Therapeutics
Electronic books.
Online Access:Connect to the full text of this electronic book
Table of Contents:
  • 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.