Rna Diseases In Humans From Fundamental Research To Therapeutic Applications

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RNA Diseases in Humans – From Fundamental Research to Therapeutic Applications

Author : Naoyuki Kataoka
Publisher : Frontiers Media SA
ISBN 13 : 2889630978
Total Pages : 123 pages
Book Rating : 4.8/5 (896 download)

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Book Synopsis RNA Diseases in Humans – From Fundamental Research to Therapeutic Applications by : Naoyuki Kataoka

Download or read book RNA Diseases in Humans – From Fundamental Research to Therapeutic Applications written by Naoyuki Kataoka and published by Frontiers Media SA. This book was released on 2019-10-15 with total page 123 pages. Available in PDF, EPUB and Kindle. Book excerpt: This Research Topic addresses the human diseases caused by a malfunction of the RNA metabolism. We aim at strengthening the link between fundamental research and therapeutic applications. In eukaryotes, RNA is transcribed from genomic DNA. RNA molecules undergo multiple post-transcriptional processes such as splicing, editing, modification, translation, and degradation. A defect, mis-regulation, or malfunction of these processes often results in diseases in humans, referred to as 'RNA diseases'. There is an increasing number of studies focused on RNA diseases, which are aimed at uncovering the fundamental molecular mechanisms at play in order to develop therapeutic approaches.

RNA Diseases in Humans - From Fundamental Research to Therapeutic Applications

Author : Naoyuki Kataoka
Publisher :
ISBN 13 :
Total Pages : 0 pages
Book Rating : 4.:/5 (136 download)

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Book Synopsis RNA Diseases in Humans - From Fundamental Research to Therapeutic Applications by : Naoyuki Kataoka

Download or read book RNA Diseases in Humans - From Fundamental Research to Therapeutic Applications written by Naoyuki Kataoka and published by . This book was released on 2019 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact.

RNA Regulation in Human Development and Disease

Author : Tariq M. Rana
Publisher : Academic Press
ISBN 13 : 9780123983169
Total Pages : 0 pages
Book Rating : 4.9/5 (831 download)

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Book Synopsis RNA Regulation in Human Development and Disease by : Tariq M. Rana

Download or read book RNA Regulation in Human Development and Disease written by Tariq M. Rana and published by Academic Press. This book was released on 2014-02-12 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA research is interdisciplinary, using biochemical, molecular and cellular biology approaches to screen and identify RNAi in humans and animals. Current medical and disease models being researched include cancer, AIDS, regenerative medicine, and Hepatitis C, Alzheimers, Parkinson’s, and other neurological diseases. RNA Regulation in Human Development and Disease is the first summative publication to offer a review of the research, timely for the multi-disciplinary researchers and graduate students studying RNA. Using clear structure and different angles from which the subject is approached, this publication includes a broad range of topics on regulation and disease, as well as therapeutic development and current techniques are discussed. An aide in establishing collaborations between basic scientists and clinicians, RNA Regulation in Human Development and Disease is extremely valuable for researchers across the fundamental and biomedical sciences. The first single resource for RNA review for researchers across disciplines Assists in collaboration from basic scientists to clinicians by discussing therapeutic applications of RNA research Offers innovative tools, a collection of current techniques

Neurodegeneration

Author : Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
ISBN 13 : 9780309285674
Total Pages : 0 pages
Book Rating : 4.2/5 (856 download)

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Book Synopsis Neurodegeneration by : Forum on Neuroscience and Nervous System Disorders

Download or read book Neurodegeneration written by Forum on Neuroscience and Nervous System Disorders and published by National Academies Press. This book was released on 2013-12-26 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurodegeneration: Exploring Commonalities Across Diseases is the summary of a workshop hosted by the Institute of Medicine\'s (IOM\'s) Forum on Neuroscience and Nervous System Disorders in Spring 2012 to explore commonalities across neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD). Participants from academia; pharmaceutical and biotechnology industries; government agencies such as the National Institutes of Health and the U.S. Department of Veterans Affairs (VA); patient advocacy groups; and private foundations presented and identified potential opportunities for collaboration across the respective research and development communities. This report identifies and discusses commonalities related to genetic and cellular mechanisms, identifies areas of fundamental science needed to facilitate therapeutics development, and explores areas of potential collaboration among the respective research communities. Neurodegenerative diseases, such as Alzheimer\'s disease, Parkinson\'s disease, ALS, and FTD, are becoming increasingly prevalent in the United States due to an aging population. Implications are grave for quality of life and health care costs. Research on neurodegenerative diseases has expanded greatly over the past four decades. Nevertheless, fundamental questions remain about the biology of these diseases, and further insights into the mechanisms of these diseases would help to inform the development of effective means to prevent and to efficiently treat them. Recent findings have revealed certain commonalities in genetic and cellular mechanisms across neurodegenerative diseases. These findings suggest that it might be valuable - at least in some cases - to change the traditional way of studying these diseases by no longer seeing each as an independent entity, but rather as clinical variants of common cellular and molecular biological defects. This approach could help enhance basic scientific understanding of neurodegenerative disease, and could help with the development of biomarkers and new therapeutics.

Innovative Medicine

Author : Kazuwa Nakao
Publisher : Springer
ISBN 13 : 4431556516
Total Pages : 330 pages
Book Rating : 4.4/5 (315 download)

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Book Synopsis Innovative Medicine by : Kazuwa Nakao

Download or read book Innovative Medicine written by Kazuwa Nakao and published by Springer. This book was released on 2015-10-13 with total page 330 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is devoted to innovative medicine, comprising the proceedings of the Uehara Memorial Foundation Symposium 2014. It remains extremely rare for the findings of basic research to be developed into clinical applications, and it takes a long time for the process to be achieved. The task of advancing the development of basic research into clinical reality lies with translational science, yet the field seems to struggle to find a way to move forward. To create innovative medical technology, many steps need to be taken: development and analysis of optimal animal models of human diseases, elucidation of genomic and epidemiological data, and establishment of “proof of concept”. There is also considerable demand for progress in drug research, new surgical procedures, and new clinical devices and equipment. While the original research target may be rare diseases, it is also important to apply those findings more broadly to common diseases. The book covers a wide range of topics and is organized into three complementary parts. The first part is basic research for innovative medicine, the second is translational research for innovative medicine, and the third is new technology for innovative medicine. This book helps to understand innovative medicine and to make progress in its realization.

Human Genome Editing

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309452880
Total Pages : 329 pages
Book Rating : 4.3/5 (94 download)

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Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine

Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.

Gene Therapy for Viral Infections

Author : Patrick Arbuthnot
Publisher : Academic Press
ISBN 13 : 0124114520
Total Pages : 392 pages
Book Rating : 4.1/5 (241 download)

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Book Synopsis Gene Therapy for Viral Infections by : Patrick Arbuthnot

Download or read book Gene Therapy for Viral Infections written by Patrick Arbuthnot and published by Academic Press. This book was released on 2015-06-01 with total page 392 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Therapy for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options. Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors. Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology. Provides coverage of gene therapy for a variety of infections, including HBV, HCV, HIV, hemorrhagic fever viruses, and respiratory and other viral infections Bridges the gap between the basic science and the important medical applications of this technology Features a broad approach to the topic, including an essential overview and the applications of gene therapy, synthetic RNA, and other antiviral strategies that involve nucleic acid engineering Presents perspectives on the future use of nucleic acids as a novel class of antiviral drugs Arms the reader with the cutting-edge information needed to stay abreast of this developing field

Adenoviral Vectors for Gene Therapy

Author : David T. Curiel
Publisher : Academic Press
ISBN 13 : 0128005106
Total Pages : 868 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Adenoviral Vectors for Gene Therapy by : David T. Curiel

Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 868 pages. Available in PDF, EPUB and Kindle. Book excerpt: Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Challenges in Delivery of Therapeutic Genomics and Proteomics

Author : Ambikanandan Misra
Publisher : Elsevier
ISBN 13 : 0123849659
Total Pages : 686 pages
Book Rating : 4.1/5 (238 download)

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Book Synopsis Challenges in Delivery of Therapeutic Genomics and Proteomics by : Ambikanandan Misra

Download or read book Challenges in Delivery of Therapeutic Genomics and Proteomics written by Ambikanandan Misra and published by Elsevier. This book was released on 2010-09-09 with total page 686 pages. Available in PDF, EPUB and Kindle. Book excerpt: Delivery of therapeutic proteomics and genomics represent an important area of drug delivery research. Genomics and proteomics approaches could be used to direct drug development processes by unearthing pathways involved in disease pathogenesis where intervention may be most successful. This book describes the basics of genomics and proteomics and highlights the various chemical, physical and biological approaches to protein and gene delivery. Covers a diverse array of topics from basic sciences to therapeutic applications of proteomics and genomics delivery Of interest to researchers in both academia and industry Highlights what’s currently known and where further research is needed

Network Medicine

Author : Joseph Loscalzo
Publisher : Harvard University Press
ISBN 13 : 0674436539
Total Pages : 449 pages
Book Rating : 4.6/5 (744 download)

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Book Synopsis Network Medicine by : Joseph Loscalzo

Download or read book Network Medicine written by Joseph Loscalzo and published by Harvard University Press. This book was released on 2017-02-01 with total page 449 pages. Available in PDF, EPUB and Kindle. Book excerpt: Big data, genomics, and quantitative approaches to network-based analysis are combining to advance the frontiers of medicine as never before. With contributions from leading experts, Network Medicine introduces this rapidly evolving field of research, which promises to revolutionize the diagnosis and treatment of human diseases.

A Guide to Human Gene Therapy

Author : Roland W. Herzog
Publisher : World Scientific
ISBN 13 : 9814280917
Total Pages : 415 pages
Book Rating : 4.8/5 (142 download)

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Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Evolution of Translational Omics

Author : Institute of Medicine
Publisher : National Academies Press
ISBN 13 : 0309224187
Total Pages : 354 pages
Book Rating : 4.3/5 (92 download)

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Book Synopsis Evolution of Translational Omics by : Institute of Medicine

Download or read book Evolution of Translational Omics written by Institute of Medicine and published by National Academies Press. This book was released on 2012-09-13 with total page 354 pages. Available in PDF, EPUB and Kindle. Book excerpt: Technologies collectively called omics enable simultaneous measurement of an enormous number of biomolecules; for example, genomics investigates thousands of DNA sequences, and proteomics examines large numbers of proteins. Scientists are using these technologies to develop innovative tests to detect disease and to predict a patient's likelihood of responding to specific drugs. Following a recent case involving premature use of omics-based tests in cancer clinical trials at Duke University, the NCI requested that the IOM establish a committee to recommend ways to strengthen omics-based test development and evaluation. This report identifies best practices to enhance development, evaluation, and translation of omics-based tests while simultaneously reinforcing steps to ensure that these tests are appropriately assessed for scientific validity before they are used to guide patient treatment in clinical trials.

Molecular Pathology

Author : William B. Coleman
Publisher : Academic Press
ISBN 13 : 0128027878
Total Pages : 802 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Molecular Pathology by : William B. Coleman

Download or read book Molecular Pathology written by William B. Coleman and published by Academic Press. This book was released on 2017-11-09 with total page 802 pages. Available in PDF, EPUB and Kindle. Book excerpt: As the molecular basis of human disease becomes better characterized, and the implications for understanding the molecular basis of disease becomes realized through improved diagnostics and treatment, Molecular Pathology, Second Edition stands out as the most comprehensive textbook where molecular mechanisms represent the focus. It is uniquely concerned with the molecular basis of major human diseases and disease processes, presented in the context of traditional pathology, with implications for translational molecular medicine. The Second Edition of Molecular Pathology has been thoroughly updated to reflect seven years of exponential changes in the fields of genetics, molecular, and cell biology which molecular pathology translates in the practice of molecular medicine. The textbook is intended to serve as a multi-use textbook that would be appropriate as a classroom teaching tool for biomedical graduate students, medical students, allied health students, and others (such as advanced undergraduates). Further, this textbook will be valuable for pathology residents and other postdoctoral fellows that desire to advance their understanding of molecular mechanisms of disease beyond what they learned in medical/graduate school. In addition, this textbook is useful as a reference book for practicing basic scientists and physician scientists that perform disease-related basic science and translational research, who require a ready information resource on the molecular basis of various human diseases and disease states. Explores the principles and practice of molecular pathology: molecular pathogenesis, molecular mechanisms of disease, and how the molecular pathogenesis of disease parallels the evolution of the disease Explains the practice of “molecular medicine and the translational aspects of molecular pathology Teaches from the perspective of “integrative systems biology Enhanced digital version included with purchase

Heritable Human Genome Editing

Author : The Royal Society
Publisher : National Academies Press
ISBN 13 : 0309671132
Total Pages : 239 pages
Book Rating : 4.3/5 (96 download)

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Book Synopsis Heritable Human Genome Editing by : The Royal Society

Download or read book Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book excerpt: Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.

RNA Delivery Function for Anticancer Therapeutics

Author : Loutfy H. Madkour
Publisher : CRC Press
ISBN 13 : 1000532240
Total Pages : 860 pages
Book Rating : 4.0/5 (5 download)

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Book Synopsis RNA Delivery Function for Anticancer Therapeutics by : Loutfy H. Madkour

Download or read book RNA Delivery Function for Anticancer Therapeutics written by Loutfy H. Madkour and published by CRC Press. This book was released on 2022-01-26 with total page 860 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents an overview of the current status of translating the RNAi cancer therapeutics in the clinic, a brief description of the biological barriers in drug delivery, and the roles of imaging in aspects of administration route, systemic circulation, and cellular barriers for the clinical translation of RNAi cancer therapeutics, and with partial content for discussing the safety concerns. It then focuses on imaging-guided delivery of RNAi therapeutics in preclinical development, including the basic principles of different imaging modalities, and their advantages and limitations for biological imaging. With growing number of RNAi therapeutics entering the clinic, various imaging methods will play an important role in facilitating the translation of RNAi cancer therapeutics from bench to bedside. RNAi technique has become a powerful tool for basic research to selectively knock down gene expression in vitro and in vivo. Our scientific and industrial communities have started to develop RNAi therapeutics as the next class of drugs for treating a variety of genetic disorders, such as cancer and other diseases that are particularly hard to address with current treatment strategies. Key Features Provides insight into the current advances and hurdles of RNAi therapeutics. Accelerates RNAi, miRNAs, and siRNA drug development for cancer therapy from bench to bedside. Addresses various modifications and novel delivery strategies for miRNAs, piRNAs and siRNA delivery in anticancer therapeutics. Explores the need for the interaction of hematologists,cell biologists, immunologists, and material scientists in the development of novel cancer therapies. Describes the current status of clinical trials related to miRNA and siRNA-based cancer therapy Presents remaining issues that need to be overcome to establish successful therapies.

Advanced Methodologies and Technologies in Medicine and Healthcare

Author : Mehdi Khosrow-Pour
Publisher : Medical Information Science Reference
ISBN 13 : 9781522574897
Total Pages : 0 pages
Book Rating : 4.5/5 (748 download)

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Book Synopsis Advanced Methodologies and Technologies in Medicine and Healthcare by : Mehdi Khosrow-Pour

Download or read book Advanced Methodologies and Technologies in Medicine and Healthcare written by Mehdi Khosrow-Pour and published by Medical Information Science Reference. This book was released on 2018-08-03 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: "Premier reference source"-- book cover.

RNA Therapeutics

Author : Mouldy Sioud
Publisher : Humana Press
ISBN 13 : 9781607616641
Total Pages : 527 pages
Book Rating : 4.6/5 (166 download)

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Book Synopsis RNA Therapeutics by : Mouldy Sioud

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.