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Author: Publisher: Academic Press ISBN: 0323994091 Category : Science Languages : en Pages : 328
Book Description
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 era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics
Author: Publisher: Academic Press ISBN: 0323994091 Category : Science Languages : en Pages : 328
Book Description
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 era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics
Author: Sarfaraz K. Niazi Publisher: CRC Press ISBN: 1000788067 Category : Medical Languages : en Pages : 327
Book Description
This book is the most comprehensive and complete treatise on nucleic acid therapeutic products, including mRNA vaccines, their manufacturing, formulations, and testing for safety and efficacy. Details include cGMP-compliant manufacturing and regulatory filing steps. A new concept of “biosimilar” mRNA vaccine is presented to secure fast approval of copies of mRNA vaccines. Projections of financial plans to establish RNA manufacturing facilities are provided, along with details of supply chain management. Finally, the future of nucleic acid products in gene therapy and other newer applications is presented, along with a perspective that all new vaccines will be the nucleic acid type that will further provide first-time prevention of autoimmune disorders. It is projected that both big pharma and start-ups will enter this field, and we can expect significant additions to our drug armamentarium soon.
Author: Stefan Jurga Publisher: Springer Nature ISBN: 3031084152 Category : Medical Languages : en Pages : 451
Book Description
This 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.
Author: Paloma H. Giangrande Publisher: Academic Press ISBN: 0128217391 Category : Science Languages : en Pages : 474
Book Description
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies. Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development. Features leading experts in the field of RNA therapeutics, spanning all classes of RNA therapies Provides a detailed examination of approved RNA therapies and lessons learned from failed therapeutics Covers all aspects of therapeutic discovery and preclinical development, as well as clinical translation, manufacturing and regulatory aspects
Author: Anya Hillery Publisher: Elsevier ISBN: 0443289352 Category : Medical Languages : en Pages : 318
Book Description
mRNA Therapeutics: Foundations, Innovations and Clinical Applications aims to provide a comprehensive text that covers all aspects of mRNA therapeutics, from the foundational science that underpins this disruptive new drug class, through the scientific and technological breakthroughs crucial for therapeutic success, to the current clinical applications and the innovative advances driving future directions. The book begins with foundational knowledge covering mRNA biology, the immune system, and vaccines. The second section addresses the major challenges associated with mRNA as a therapeutic modality, and the molecular engineering innovations and delivery technologies that have allowed these hurdles to be largely overcome. The third section describes the current and future clinical applications of mRNA therapeutics that are transforming, or are poised to transform, medicine and health. This includes the use of mRNA vaccines for COVID-19 and other infectious diseases, as well as mRNA's role in revolutionizing cancer immunotherapy, covering immunostimulants, cancer vaccines including personalized neoantigen vaccines, and CAR T cell technologies. Additional chapters describe the use of mRNA therapeutics for protein replacement therapy and gene-editing, as well as newer mRNA constructs, including self-amplifying mRNA. The final section addresses the safety and regulatory considerations of mRNA therapeutics, along with broader cultural issues including vaccine hesitancy, global vaccine inequality, and pandemic preparedness. Currently, mRNA texts either provide personal accounts from key players involved in COVID-19 vaccine development, with limited scientific depth, or focus on highly specialized, more esoteric applications of mRNA in advanced molecular biology. This book aims to bridge this gap by providing a scientifically rigorous and wide-ranging exploration of mRNA's role in therapeutics. This pioneering textbook serves as a vital addition to the academic canon, providing an essential tool for the current and next generation of students, scientists, researchers and professionals in a wide variety of related disciplines including molecular biology, biomedical engineering, pharmaceutical science, oncology, and the health sciences. - Focuses on the science of mRNA, covering the development, modus operandi, platform manufacturing technology, safety, and efficacy of this treatment modality - Provides the mRNA technology fit with the wider context of vaccinology, virology, oncology, biotechnology, as well as manufacturing and regulatory science - Offers an understanding of the recent paradigm shift in the way we treat disease
Author: Robert E. Rhoads Publisher: Humana ISBN: 9781493936236 Category : Medical Languages : en Pages : 0
Book Description
This volume presents detailed laboratory protocols for in vitro synthesis of mRNA with favorable properties, its introduction into cells by a variety of techniques, and the measurement of physiological and clinical consequences such as protein replacement and cancer immunotherapy. Synthetic techniques are described for structural features in mRNA that provide investigational tools such as fluorescence emission, click chemistry, photo-chemical crosslinking, and that produce mRNA with increased stability in the cell, increased translational efficiency, and reduced activation of the innate immune response. Protocols are described for clinical applications such as large-scale transfection of dendritic cells, production of GMP-grade mRNA, redirecting T cell specificity, and use of molecular adjuvants for RNA vaccines. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Synthetic mRNA: Production, Introduction into Cells, and Physiological Consequences is a valuable and cutting-edge resource for both laboratory investigators and clinicians interested in this powerful and rapidly evolving technology.
Author: Mouldy Sioud Publisher: Humana Press ISBN: 9781607616641 Category : Science Languages : en Pages : 527
Book Description
Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.
Author: Mouldy Sioud Publisher: Humana Press ISBN: 9781607616641 Category : Science Languages : en Pages : 527
Book Description
Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.
Author: Lara Scheherazade Milane Publisher: CRC Press ISBN: 1000505936 Category : Science Languages : en Pages : 511
Book Description
We have surpassed the omics era and are truly in the Age of Molecular Therapeutics. The fast-paced development of SARS-CoV-2 vaccines, such as the mRNA vaccines encoding the viral spike protein, demonstrated the need for and capability of molecular therapy and nanotechnology-based solutions for drug delivery. In record speed, the SARS-CoV-2 viral RNA genome was sequenced and shared with the scientific community, allowing the rapid design of molecular therapeutics. The mRNA vaccines exploit the host cell endoplasmic reticulum to produce viral spike proteins for antigen presentation and recognition by the innate and adaptive immune system. Lipid nanoparticles enable the delivery of the fragile, degradation-sensitive nucleic acid payloads. Molecular-based therapeutics and nanotechnology solutions continue to drive the scientific and medical response to the COVID-19 pandemic as new mRNA, DNA, and protein-based vaccines are developed and approved and the emergency use approved vaccines are rapidly manufactured and distributed throughout the globe. The need for molecular therapies and drug delivery solutions is clear, and as these therapies progress and become more specialized there will be important advancements in organelle targeting. For example, using organelle targeting to direct lipid nanoparticles with mRNA payloads to the endoplasmic reticulum would increase the efficacy of mRNA vaccines, reducing the required dose and therefore the biomanufacturing demand. Likewise, improving the delivery of DNA therapeutics to the nucleus would improve efficacy. Organelles and molecules have always been drug targets, but until recently we have not had the tools or capability to design and develop such highly specific therapeutics. Organelle targeting has far-reaching implications. For example, mitochondria are central to both energy production and intrinsic apoptosis. Effectively targeting and manipulating mitochondria has therapeutic applications for diseases such as myopathies, cancer, neurodegeneration, progerias, diabetes, and the natural aging process. The SARS-CoV-2 vaccines that exploit the endoplasmic reticulum (for mRNA vaccines) and the nucleic translational process (DNA vaccines) attest to the need for organelle and molecular therapeutics. This book covers the status, demand, and future of organelle- and molecularly targeted therapeutics that are critical to the advancement of modern medicine. Organelle and molecular targeting is the drug design and drug delivery approach of today and the future; understanding this approach is essential for students, scientists, and clinicians contributing to modern medicine.
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Author: Sarfaraz K. Niazi
Author: Stefan Jurga
Author: Paloma H. Giangrande
Author: Anya Hillery
Author: Robert E. Rhoads
Author: Mouldy Sioud
Author: Jens Kurreck
Author: Lara Scheherazade Milane