Synthetic mRNA

Synthetic mRNA

Author: Robert E. Rhoads

Publisher: Humana

Published: 2016-05-29

Total Pages: 0

ISBN-13: 9781493936236

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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.


Molecular Biology: A Very Short Introduction

Molecular Biology: A Very Short Introduction

Author: Aysha Divan

Publisher: Oxford University Press

Published: 2016-08-18

Total Pages: 195

ISBN-13: 0191035815

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Molecular Biology is the story of the molecules of life, their relationships, and how these interactions are controlled. It is an expanding field in life sciences, and its applications are wide and growing. We can now harness the power of molecular biology to treat diseases, solve crimes, map human history, and produce genetically modified organisms and crops, and these applications have sparked a multitude of fascinating legal and ethical debates. In this Very Short Introduction, Aysha Divan and Janice Royds examine the history, present, and future of Molecular Biology. Starting with the building blocks established by Darwin, Wallace and Mendel, and the discovery of the structure of DNA in 1953, they consider the wide range of applications for Molecular Biology today, including the development of new drugs, and forensic science. They also look forward to two key areas of evolving research such as personalised medicine and synthetic biology. ABOUT THE SERIES: The Very Short Introductions series from Oxford University Press contains hundreds of titles in almost every subject area. These pocket-sized books are the perfect way to get ahead in a new subject quickly. Our expert authors combine facts, analysis, perspective, new ideas, and enthusiasm to make interesting and challenging topics highly readable.


Messenger RNA Therapeutics

Messenger RNA Therapeutics

Author: Stefan Jurga

Publisher: Springer Nature

Published: 2022-09-20

Total Pages: 451

ISBN-13: 3031084152

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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.


Gene Therapy of the Central Nervous System: From Bench to Bedside

Gene Therapy of the Central Nervous System: From Bench to Bedside

Author: Michael G. Kaplitt

Publisher: Gulf Professional Publishing

Published: 2006

Total Pages: 380

ISBN-13: 9780123976321

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Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.


The Biology of mRNA: Structure and Function

The Biology of mRNA: Structure and Function

Author: Marlene Oeffinger

Publisher: Springer Nature

Published: 2019-12-06

Total Pages: 318

ISBN-13: 3030314340

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The book provides an overview on the different aspects of gene regulation from an mRNA centric viewpoint, including how mRNA is assembled and self-assembles in a complex consisting of RNA and proteins, and how its ability to be translated at the right time and space depends on many processes acting on the mRNAs, leading to a properly folded complex. This book shows how new technologies have led to a better understanding of these processes and their connected diseases.The book is written for scientists in fundamental and applied biomedical research working on different aspects of gene regulation. It is also targeted to an audience that is not implicated in these fields directly, but wants to gain a better understanding of mRNA biology.


Genetically Engineered Crops

Genetically Engineered Crops

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2017-01-28

Total Pages: 607

ISBN-13: 0309437385

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Genetically engineered (GE) crops were first introduced commercially in the 1990s. After two decades of production, some groups and individuals remain critical of the technology based on their concerns about possible adverse effects on human health, the environment, and ethical considerations. At the same time, others are concerned that the technology is not reaching its potential to improve human health and the environment because of stringent regulations and reduced public funding to develop products offering more benefits to society. While the debate about these and other questions related to the genetic engineering techniques of the first 20 years goes on, emerging genetic-engineering technologies are adding new complexities to the conversation. Genetically Engineered Crops builds on previous related Academies reports published between 1987 and 2010 by undertaking a retrospective examination of the purported positive and adverse effects of GE crops and to anticipate what emerging genetic-engineering technologies hold for the future. This report indicates where there are uncertainties about the economic, agronomic, health, safety, or other impacts of GE crops and food, and makes recommendations to fill gaps in safety assessments, increase regulatory clarity, and improve innovations in and access to GE technology.


Intracellular Delivery

Intracellular Delivery

Author: Aleš Prokop

Publisher: Springer Science & Business Media

Published: 2011-05-26

Total Pages: 871

ISBN-13: 9400712480

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This book features a special subsection of Nanomedicine, an application of nanotechnology to achieve breakthroughs in healthcare. It exploits the improved and often novel physical, chemical and biological properties of materials only existent at the nanometer scale. As a consequence of small scale, nanosystems in most cases are efficiently uptaken by cells and appear to act at the intracellular level. Nanotechnology has the potential to improve diagnosis, treatment and follow-up of diseases, and includes targeted drug delivery and regenerative medicine; it creates new tools and methods that impact significantly upon existing conservative practices. This volume is a collection of authoritative reviews. In the introductory section we define the field (intracellular delivery). Then, the fundamental routes of nanodelivery devices, cellular uptake, types of delivery devices, particularly in terms of localized cellular delivery, both for small drug molecules, macromolecular drugs and genes; at the academic and applied levels, are covered. The following section is dedicated to enhancing delivery via special targeting motifs followed by the introduction of different types of intracellular nanodelivery devices (e.g. a brief description of their chemistry) and ways of producing these different devices. Finally, we put special emphasis on particular disease states and on other biomedical applications, whilst diagnostic and sensing issues are also included. Intracellular delivery / therapy is a highly topical which will stir great interest. Intracellular delivery enables much more efficient drug delivery since the impact (on different organelles and sites) is intracellular as the drug is not supplied externally within the blood stream. There is great potential for targeted delivery with improved localized delivery and efficacy.


Goodman's Basic Medical Endocrinology

Goodman's Basic Medical Endocrinology

Author: Elizabeth H. Holt

Publisher: Academic Press

Published: 2021-02-21

Total Pages: 554

ISBN-13: 012815845X

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Goodman's Basic Medical Endocrinology, Fifth Edition, has been student tested and approved for decades. This essential textbook provides up-to-date coverage of rapidly unfolding advances in the understanding of hormones involved in regulating most aspects of bodily functions. It is richly illustrated in full color with both descriptive schematic diagrams and laboratory findings obtained in clinical studies. This is a classic reference for moving forward into advanced study. - Clinical case studies in every chapter - E-book version available with every copy for obtaining images and tables for lectures or notes - Clinicians added as co-authors to enhance usefulness by physicians and medical students and residents - Detailed molecular biology of hormones and hormone action for graduate and advanced undergraduate students - Expanded and updated color images emphasizing hormone action at the molecular level - In-depth molecular biology and clinical sections boxed for ease of access