Download or read book mRNA Therapeutics written by Sarfaraz K. Niazi and published by CRC Press. This book was released on 2022-12-06 with total page 327 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book mRNA Therapeutics written by Anya Hillery and published by Elsevier. This book was released on 2024-09-09 with total page 318 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Download or read book MRNA Based Therapeutics written by Fernando Aranda Vega and published by Elsevier. This book was released on 2022-09-07 with total page 326 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Download or read book MRNA Based Therapeutics written by and published by Academic Press. This book was released on 2022-09-03 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Download or read book Messenger RNA Therapeutics written by Stefan Jurga and published by Springer Nature. This book was released on 2022-09-20 with total page 451 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Synthetic mRNA written by Robert E. Rhoads and published by Humana. This book was released on 2016-05-29 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book RNA Therapeutics Part A written by and published by Elsevier. This book was released on 2024-02-13 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA Therapeutics, Part A, Volume 203 presents the latest on a new class of medication based on RNA molecules. The book includes timely chapters that focus on An introduction to RNA therapeutics and potentials, The development and technologies of RNA therapeutics, Types of RNA therapeutics, Molecular mechanisms of RNA therapeutics, IT, and AI applications in RNA therapeutics, The application of data sciences and bioinformatics in RNA therapeutics, RNA therapeutics history and future perspectives, Recent applications of RNA therapeutic in clinics, Advantages and disadvantages of RNA therapeutics, RNA therapeutics for neurological disease, RNA therapeutics for metabolic disorders, and RNA therapeutics for metabolic disorders. - Discusses the history of RNA therapeutics, along with future perspectives - Presents the processes of RNA therapeutical developments - Covers recent applications of RNA therapeutics in clinics - Provides the advantages and disadvantages of RNA therapeutics

Download or read book RNA Therapeutics written by Mouldy Sioud and published by Humana Press. This book was released on 2011-03-04 with total page 527 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book RNA Therapeutics written by Amanda L. Garner and published by Springer. This book was released on 2017-09-26 with total page 263 pages. Available in PDF, EPUB and Kindle. Book excerpt: Medicinal chemistry is both science and art. The science of medicinal chemistry offers mankind one of its best hopes for improving the quality of life. The art of medicinal chemistry continues to challenge its practitioners with the need for both intuition and experience to discover new drugs. Hence sharing the experience of drug research is uniquely beneficial to the field of medicinal chemistry. Drug research requires interdisciplinary team-work at the interface between chemistry, biology and medicine. Therefore, the topic-related series Topics in Medicinal Chemistry covers all relevant aspects of drug research, e.g. pathobiochemistry of diseases, identification and validation of (emerging) drug targets, structural biology, drugability of targets, drug design approaches, chemogenomics, synthetic chemistry including combinatorial methods, bioorganic chemistry, natural compounds, high-throughput screening, pharmacological in vitro and in vivo investigations, drug-receptor interactions on the molecular level, structure-activity relationships, drug absorption, distribution, metabolism, elimination, toxicology and pharmacogenomics. In general, special volumes are edited by well known guest editors.

Download or read book RNA Therapeutics written by Paloma H. Giangrande and published by Academic Press. This book was released on 2022-04-10 with total page 474 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Download or read book RNA Therapeutics Part B written by and published by Elsevier. This book was released on 2024-03-06 with total page 360 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA Therapeutics, Part B, Volume 204 updates on a new class of medication based on RNA molecules, with chapters on a variety of topics, including RNA therapeutics for cardiovascular disorders, RNA therapeutics for kidney injury, RNA therapeutics for beta-thalassemia, RNA therapeutics for infectious diseases, RNA therapeutics for reproductive diseases, Potential of RNA and transcriptomic data to drive drug repurposing for various diseases, RNA therapeutics for glioma/glioblastoma, RNA therapeutics for regenerative medicine, Advances in the liposome-based delivery systems for RNA therapeutics in cancer, Advances in the polymeric nanoparticulate delivery systems for RNA therapeutics in cancer, and more. - Covers RNA therapeutics' history and future perspectives - Discusses the processes of RNA therapeutical development - Presents recent applications of RNA therapeutic in clinics - Provides the advantages and disadvantages of RNA therapeutics

Download or read book mRNA Vaccines written by Dong Yu and published by Springer Nature. This book was released on 2023-01-03 with total page 215 pages. Available in PDF, EPUB and Kindle. Book excerpt: The formulation and the technological advancements in RNA biology, chemistry, stability, and encapsulated delivery systems that have enabled the development of fully synthetic mRNA vaccines are discussed in this volume. The applications of the mRNA technology is covered, focusing on infectious diseases but also touching on other indications, such as immunotherapies and molecular therapies. Potent and long-lasting immune responses observed in animal models, encouraging data from early human clinical studies, together with the success of two mRNA-based COVID-19 vaccines support the use of mRNA-based vaccination as an attractive alternative to conventional vaccine approaches. Consequently, the development progress of the technology, particularly on production, capabilities, and clinical development is reviewed. Topics on safety, regulatory issues, and possible challenges to the mRNA vaccination approach round off this book. Thanks to their high potency, the prospect for generic, low-cost manufacturing processes, and entirely synthetic nature, the future for mRNA vaccines is highly promising. Importantly, mRNA vaccines have the potential to minimize the time between pathogen identification and vaccine release with a huge impact on public health. As the mRNA-based vaccination technology has been progressing rapidly, the book is intended to be an end-to-end review series, covering everything from basic RNA biology and preclinical studies to the manufacturing strategy, clinical development and regulatory approval. It provides established RNA researchers and developers with updates on the latest advancements in the field and allows for a quick but comprehensive overview of this transformative technology, its application, and future potential.

Download or read book Novel insights in RNA modifications From basic to translational research written by Huilin Huang and published by Frontiers Media SA. This book was released on 2023-04-06 with total page 279 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Organelle and Molecular Targeting written by Lara Scheherazade Milane and published by CRC Press. This book was released on 2021-12-27 with total page 511 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Pharmaceutics written by Alekha Dash and published by Elsevier. This book was released on 2023-09-13 with total page 588 pages. Available in PDF, EPUB and Kindle. Book excerpt: Pharmaceutics: Basic Principles and Application to Pharmacy Practice, Second Edition is a valuable textbook covering the role and application of pharmaceutics within pharmacy practice. This updated resource is geared toward meeting and incorporating the current curricular guidelines on pharmaceutics and laboratory skills mandated by the American Council for Pharmacy Education. It includes a number of student-friendly features, including chapter objectives and summaries, practical examples, case studies, numerous images and key-concept text boxes. Two new chapters are included, as well as a new end of chapter section covering "critical reflections and practice applications". Divided into three sections – Physical Principles and Properties of Pharmaceutics; Practical Aspects of Pharmaceutics; and Biological Applications of Pharmaceutics – this new edition covers all aspects of pharmaceutics and providing a single and compelling source for students. - Facilitates an integrated and extensive coverage of the study of pharmaceutics due to the clear and engaging language used by the authors - Includes chapter objectives and summaries to illustrate and reinforce key ideas - Meets curricular guidelines for pharmaceutics and laboratory skills mandated by the Accreditation Council for Pharmacy Education (ACPE) - Includes new practice questions, answers, and case studies for experiential learning

Download or read book Aging written by Michael Fossel and published by Elsevier. This book was released on 2024-02-29 with total page 274 pages. Available in PDF, EPUB and Kindle. Book excerpt: Aging: How Aging Works, How We Reverse Aging, and Prospects for Curing Aging Diseases explains the process of aging beyond mere entropy, exposing it as a complicated and dynamic process that undercuts maintenance and permits age-related disease. With a deeper understanding of the aging process, intervention becomes both easy to understand and clinically feasible. With a solid academic approach, this proposed book builds upon the substantial work published over the past 20 years, citing the newest data, up-to-date models based upon that data, and the implications for improved clinical intervention, including recent developments in gene and cell therapy. Coverage of age-related diseases includes neurodegenerative, cardiovascular, bone and joint, immune system, renal, pulmonary, and skin aging. Future directions of the field focus on interventions, including a summary of previous attempts to intervene in aging and age-related disease, the status of current research, and proposed biotech interventions, as well as their potential obstacles, risks, and benefits. This is the perfect reference for scientists, clinicians, and researchers interested in the translational research opportunities such as drug discovery, pharmacogenetics, and experimental therapeutics, not only summarizing where the field stands, but giving a clear and cogent view of where clinical medicine is going in the next decade. - Provides a sophisticated, accurate, and clear explanation of aging - Gives a clear explanation of the fundamental role of cell aging in age-related disease - Offers a unified model for the role of epigenetic and telomere changes in cell aging - Outlines effective approaches to intervention in the fundamental aging process - Introduces upcoming interventions intended to both cure and prevent age-related diseases

Download or read book Goldman Cecil Medicine E Book written by Lee Goldman and published by Elsevier Health Sciences. This book was released on 2023-07-15 with total page 4183 pages. Available in PDF, EPUB and Kindle. Book excerpt: For more than 95 years, Goldman-Cecil Medicine has been the authoritative source for internal medicine and the care of adult patients. Every chapter is written by acclaimed experts who, with the oversight of our editors, provide definitive, unbiased advice on the diagnosis and treatment of thousands of common and uncommon conditions, always guided by an understanding of the epidemiology and pathobiology, as well as the latest medical literature. But Goldman-Cecil Medicine is not just a textbook. It is designed to optimize electronic searches that will rapidly take you to exactly the information you are seeking. Throughout the lifetime of each edition, periodic updates continually include the newest information from a wide range of journals. Furthermore, Goldman-Cecil Medicine is available for all users of ClinicalKey, Elsevier's full library of subspecialty textbooks that can be accessed by readers who may want even more in-depth information. - More than 400 chapters authored by a veritable "Who's Who" of modern medicine - A practical, templated organization with an emphasis on up-to-date, evidence-based references - New chapters on Population Health, Effects of Climate Change on Health, Bradycardias, Transgender Medicine, Whipple Disease, COVID-19 Virology and Pathobiology, COVID-19 Epidemiology/Clinical Manifestations/Diagnosis/Community Prevention, COVID-19 Treatment and Vaccination, Polyomaviruses, and more - Thousands of algorithms, figures, and tables that make its information readily accessible - Over 100 supplementary videos, heart sounds, and key references - Available in print and on a variety of electronic devices - Continuously updated by Lee Goldman, MD - An eBook version is included with purchase. The eBook allows you to access all of the text, figures, and references, with the ability to search, customize your content, make notes and highlights, and have content read aloud.