Download or read book Engineered Synthetic Translational Control for Next Generation MRNA Gene Therapies written by Jacob Robert Becraft and published by . This book was released on 2019 with total page 148 pages. Available in PDF, EPUB and Kindle. Book excerpt: Synthetic mRNA is an emerging therapeutic modality for gene and cell therapy. Unlike their synthetic DNA counterparts, synthetic mRNA has an increased safety profile due to its transient gene expression and ability to express outside of the nucleus. Furthermore, it can be more easily delivered to cells via entry only into the cytoplasm. While synthetic biology as a field has existed for over two decades, the main area of research and development has focused on DNA interfaces, building on the mechanisms of transcription factors with small molecule interfaces to create multi-input/multi-output genetic circuitry. Until recently, the field had not developed sufficient synthetic circuit control devices at the translational level due to 1) lack of perceived need and 2) deficiency of available natural systems for adaptation. In this thesis, I present the construction of a diverse synthetic biology toolbox for RNA-only synthetic biology. The creation of new synthetic biology frameworks can be broken down into three modules: Build, Control, and Apply. In the Build phase, I demonstrate how the current toolbox of mRNA binding and recognition proteins can be utilized to form diverse and orthogonal gene regulatory networks. In Control, I construct regulatory networks capable of responding to exogenous signals and utilize advanced circuit design to motivate dynamic control for novel behaviors. When I transition to Apply, I illustrate that these next-generation circuits can be layered into biologically active modalities that are therapeutically relevant. Taken as a whole, the work presented here represents a merging of the fields of synthetic biology and mRNA therapeutics, and serves as a foundational proof-of-principle for future efforts to expand synthetic biology across novel modalities.
Download or read book The Science and Applications of Synthetic and Systems Biology written by Institute of Medicine and published by National Academies Press. This book was released on 2011-12-30 with total page 570 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many potential applications of synthetic and systems biology are relevant to the challenges associated with the detection, surveillance, and responses to emerging and re-emerging infectious diseases. On March 14 and 15, 2011, the Institute of Medicine's (IOM's) Forum on Microbial Threats convened a public workshop in Washington, DC, to explore the current state of the science of synthetic biology, including its dependency on systems biology; discussed the different approaches that scientists are taking to engineer, or reengineer, biological systems; and discussed how the tools and approaches of synthetic and systems biology were being applied to mitigate the risks associated with emerging infectious diseases. The Science and Applications of Synthetic and Systems Biology is organized into sections as a topic-by-topic distillation of the presentations and discussions that took place at the workshop. Its purpose is to present information from relevant experience, to delineate a range of pivotal issues and their respective challenges, and to offer differing perspectives on the topic as discussed and described by the workshop participants. This report also includes a collection of individually authored papers and commentary.
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 Synthetic Gene Networks written by Wilfried Weber and published by Humana Press. This book was released on 2011-11-17 with total page 393 pages. Available in PDF, EPUB and Kindle. Book excerpt: The rapid expansion of synthetic biology is due to the design and construction of synthetic gene networks that have opened many new avenues in fundamental and applied research. Synthetic Gene Networks: Methods and Protocols provides the necessary information to design and construct synthetic gene networks in different host backgrounds. Divided into four convenient sections, this volume focuses on design concepts to devise synthetic gene networks and how mathematical models can be applied to the predictable engineering of desired network features. The volume continues by highlighting the construction and validation of biologic tools, describing strategies to optimize and streamline the host cell for optimized network performance, and covering how optimally designed gene networks can be implemented in a large variety of host cells ranging from bacteria over yeast and insect cells to plant and mammalian cell culture. Written in the successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and easily accessible, Synthetic Gene Networks: Methods and Protocols serves as an invaluable resource for established biologists, engineers, and computer scientists or novices just entering into the rapidly growing field of synthetic biology
Download or read book Biodefense in the Age of Synthetic Biology written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-01-05 with total page 189 pages. Available in PDF, EPUB and Kindle. Book excerpt: Scientific advances over the past several decades have accelerated the ability to engineer existing organisms and to potentially create novel ones not found in nature. Synthetic biology, which collectively refers to concepts, approaches, and tools that enable the modification or creation of biological organisms, is being pursued overwhelmingly for beneficial purposes ranging from reducing the burden of disease to improving agricultural yields to remediating pollution. Although the contributions synthetic biology can make in these and other areas hold great promise, it is also possible to imagine malicious uses that could threaten U.S. citizens and military personnel. Making informed decisions about how to address such concerns requires a realistic assessment of the capabilities that could be misused. Biodefense in the Age of Synthetic Biology explores and envisions potential misuses of synthetic biology. This report develops a framework to guide an assessment of the security concerns related to advances in synthetic biology, assesses the levels of concern warranted for such advances, and identifies options that could help mitigate those concerns.
Download or read book Genetically Engineered Crops written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-01-28 with total page 607 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Cell Culture Engineering written by Wei-Shu Hu and published by Springer. This book was released on 2006-08-16 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the introduction of recombinant human growth hormone and insulin a quarter century ago, protein therapeutics has greatly broadened the ho- zon of health care. Many patients suffering with life-threatening diseases or chronic dysfunctions, which were medically untreatable not long ago, can attest to the wonder these drugs have achieved. Although the ?rst generation of p- tein therapeutics was produced in recombinant Escherichia coli, most recent products use mammalian cells as production hosts. Not long after the ?rst p- duction of recombinant proteins in E. coli, it was realized that the complex tasks of most post-translational modi?cations on proteins could only be ef?ciently carried out in mammalian cells. In the 1990s, we witnessed a rapid expansion of mammalian-cell-derived protein therapeutics, chie?y antibodies. In fact, it has been nearly a decade since the market value of mammalian-cell-derived protein therapeutics surpassed that of those produced from E. coli. A common characteristic of recent antibody products is the relatively large dose required for effective therapy, demanding larger quantities for the treatment of a given disease. This, coupled with the broadening repertoire of protein drugs, has rapidly expanded the quantity needed for clinical applications. The increasing demand for protein therapeutics has not been met exclusively by construction of new manufacturing plants and increasing total volume capacity. More - portantly the productivity of cell culture processes has been driven upward by an order of magnitude in the past decade.
Download or read book Cell Free Synthetic Biology written by Jian Li and published by Frontiers Media SA. This book was released on 2022-01-13 with total page 205 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Genetic Cardiomyopathies written by Gianfranco Sinagra and published by Springer Science & Business Media. This book was released on 2012-12-09 with total page 170 pages. Available in PDF, EPUB and Kindle. Book excerpt: In the last decade, genetics has been emerging as a primary issue in the diagnosis and management of cardiomyopathies. This book is intended to be a state-of-the-art monograph on these diseases, describing their genetic causes, defining the molecular basis and presenting extensive descriptions of genotype–phenotype correlations. Other chapters are focused on the role of clinical observation, on ECG and echocardiography. With its highlight on the most recent discoveries in the field of molecular genetics as well as on the correct clinical approach to patients with heart muscle disease, the book is aimed at physicians and clinical cardiologists with a particular interest in myocardial diseases and in their genetic causes.
Download or read book Modified Nucleic Acids written by Kazuhiko Nakatani and published by Springer. This book was released on 2016-04-04 with total page 280 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book spans diverse aspects of modified nucleic acids, from chemical synthesis and spectroscopy to in vivo applications, and highlights studies on chemical modifications of the backbone and nucleobases. Topics discussed include fluorescent pyrimidine and purine analogs, enzymatic approaches to the preparation of modified nucleic acids, emission and electron paramagnetic resonance (EPR) spectroscopy for studying nucleic acid structure and dynamics, non-covalent binding of low- and high-MW ligands to nucleic acids and the design of unnatural base pairs. This unique book addresses new developments and is designed for graduate level and professional research purposes.
Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Download or read book Molecular Biology of The Cell written by Bruce Alberts and published by . This book was released on 2002 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gene Therapy of the Central Nervous System From Bench to Bedside written by Michael G. Kaplitt and published by Elsevier. This book was released on 2005-11-11 with total page 372 pages. Available in PDF, EPUB and Kindle. Book excerpt: Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far 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 of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and 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 of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.
Download or read book Advanced Healthcare Materials written by Ashutosh Tiwari and published by John Wiley & Sons. This book was released on 2014-05-09 with total page 421 pages. Available in PDF, EPUB and Kindle. Book excerpt: Offers a comprehensive and interdisciplinary view of cutting-edge research on advanced materials for healthcare technology and applications Advanced healthcare materials are attracting strong interest in fundamental as well as applied medical science and technology. This book summarizes the current state of knowledge in the field of advanced materials for functional therapeutics, point-of-care diagnostics, translational materials, and up-and-coming bioengineering devices. Advanced Healthcare Materials highlights the key features that enable the design of stimuli-responsive smart nanoparticles, novel biomaterials, and nano/micro devices for either diagnosis or therapy, or both, called theranostics. It also presents the latest advancements in healthcare materials and medical technology. The senior researchers from global knowledge centers have written topics including: State-of-the-art of biomaterials for human health Micro- and nanoparticles and their application in biosensors The role of immunoassays Stimuli-responsive smart nanoparticles Diagnosis and treatment of cancer Advanced materials for biomedical application and drug delivery Nanoparticles for diagnosis and/or treatment of Alzheimers disease Hierarchical modelling of elastic behavior of human dental tissue Biodegradable porous hydrogels Hydrogels in tissue engineering, drug delivery, and wound care Modified natural zeolites Supramolecular hydrogels based on cyclodextrin poly(pseudo)rotaxane Polyhydroxyalkanoate-based biomaterials Biomimetic molecularly imprinted polymers
Download or read book Structural Aspects Of Protein Synthesis 2nd Edition written by Anders Liljas and published by World Scientific. This book was released on 2013-06-06 with total page 494 pages. Available in PDF, EPUB and Kindle. Book excerpt: This highly illustrated book provides an up-to-date description of the structure and function of the translation system including ribosomes, tRNAs, translation factors, antibiotics and aminoacyl-tRNA synthetases.Research on translation is undergoing rapid changes and is receiving significant attention as evidenced by the Nobel Prize in Chemistry 2009. The structural research by crystallography and cryo-EM forms part of an interactive framework that involves biochemistry and molecular computation. The book provides a comprehensive overview of translation in light of the structural results. It is a valuable resource for scientists in this and related fields, as well as for students taking courses with a focus on translation.There is no other book in this field currently except the previous edition of this book. The authors have for a long time worked in the field of structure and function of the translation system.
Download or read book Mammalian Synthetic Biology written by Jamie Davies and published by . This book was released on 2019-12-12 with total page 137 pages. Available in PDF, EPUB and Kindle. Book excerpt: Written primarily for mid-to-upper level undergraduates, this primer will introduce students to topics at the forefront of the subject that are being applied to probe biological problems, or to address the most pressing issues facing society. These topics will include those that form thecornerstone of contemporary research, helping students to make the transition to active researcher.This primer introduces the challenges and opportunities of applying synthetic biological techniques to mammalian cells, tissues, and organisms. It covers the special features that make engineering mammalian systems different from engineering bacteria, fungi, and plants, and provides an overview ofcurrent techniques. A variety of cutting-edge examples illustrate the different purposes of mammalian synthetic biology, including pure biomedical research, drug production, tissue engineering, and regenerative medicine.
