rna therapeutics

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RNA Therapeutics
Author : Amanda L. Garner
Publisher : Springer
Release Date : 2017-09-26
ISBN 10 : 3319680919
Pages : 259 pages

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.

Entering the RNA Wonderland: Opportunities and Challenges for RNA Therapeutics in the Cardiovascular System
Author : Anna Zampetaki,Lars Maegdefessel
Publisher : Frontiers Media SA
Release Date : 2020-07-22
ISBN 10 : 2889638820
Pages : 329 pages

RNA Therapeutics
Author : Mouldy Sioud
Publisher : Humana Press
Release Date : 2011-03-04
ISBN 10 : 9781607616641
Pages : 527 pages

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.

RNA Nanotechnology and Therapeutics
Author : Peixuan Guo,Farzin Haque
Publisher : CRC Press
Release Date : 2013-07-09
ISBN 10 : 1466505834
Pages : 611 pages

Interest in RNA nanotechnology has increased in recent years as recognition of its potential for applications in nanomedicine has grown. Edited by the world's foremost experts in nanomedicine, this comprehensive, state-of-the-art reference details the latest research developments and challenges in the biophysical and single molecule approaches in R

Tumor-penetrating Delivery of Small Interfering RNA Therapeutics
Author : Yin Ren (Ph. D.),Harvard--MIT Program in Health Sciences and Technology
Publisher : N.A
Release Date : 2012
ISBN 10 :
Pages : 254 pages

Efforts to sequence cancer genomes have begun to uncover comprehensive lists of genes altered in cancer. Unfortunately, the number and complexity of identified alterations has made dissecting the underlying biology of cancer difficult, as many genes are not amenable to manipulation by small molecules or antibodies. RNA interference (RNAi) provides a direct way to assess and act on putative cancer targets. However, the translation of RNAi into the clinic has been thwarted by the "delivery" challenge, as small interfering RNA (siRNA) therapeutics must overcome clearance mechanisms and penetrate into tumor tissues to access cancer cells. This thesis sought to develop nanotechnology-based platforms to rapidly discover and validate cancer targets in vivo. First, we developed versatile surface chemistries for nanoparticle tumor targeting. Leveraging new discoveries in amplified transvascular transport, we designed a siRNA delivery system that integrates the tumor specificity and tissue-penetrating ability of tumor-penetrating peptides with membrane penetration properties of protein transduction domains to direct siRNA to tumors in vivo. Second, we utilized this delivery system to bridge the gap between cancer genomic discovery and in vivo target validation. Comprehensive analysis of ovarian cancer genomes identified candidate targets that are undruggable by traditional approaches. Tumor-penetrating delivery of siRNA against these genes potently impeded the growth of ovarian tumors in mice and improved survival, thereby credentialing their roles in tumor initiation and maintenance. Lastly, we described efforts extending this platform for clinical translation. Mechanistic studies identified functional properties that favored receptor-specific siRNA delivery. We also explored a strategy to improve the microdistribution of successively dosed siRNA therapeutics through modulating the tumor microenvironment. Finally, we investigated the utility of the system in primary human tumors derived from patients with ovarian cancer. Together, these findings illustrate that the combination of cancer genomics with the engineering of siRNA delivery nanomaterials establishes a platform for discovering genes amenable to RNAi therapies. As efforts in genome sequencing accelerate, this platform illustrates a path to clinical translation in humans.

RNA Interference from Biology to Therapeutics
Author : Kenneth A. Howard
Publisher : Springer Science & Business Media
Release Date : 2012-10-19
ISBN 10 : 1461447445
Pages : 340 pages

The enormous potential of siRNA as a therapeutic has led to an explosion of interest from the scientific community. There has been intense interest from Big Pharma to capitalise on this new technology but the fact remains that delivery is a key determinant in realizing the full clinical potential of RNA interference. There is an urgent need for better delivery methods to take this technology forward. This book addresses the role of different RNAi molecules in cellular processes as rational for diagnostic and therapeutic approaches. This book will cover RNAi therapeutic design to optimize siRNA potency and reduce off-target effects and current delivery technologies to overcome both intracellular and extracellular barriers. The reader will gain an insight into RNA interference from the cellular mechanisms to screening to siRNA design right through to diagnostic and therapeutic applications.

RNA Therapeutics
Author : Paloma H. Giangrande,Vittorio Defranciscis,John J. Rossi
Publisher : Academic Press
Release Date : 2021-06-01
ISBN 10 : 0128217391
Pages : 512 pages

RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with 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 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

Dendrimers in Biomedical Applications
Author : Barbara Klajnert,Ling Peng,Valentin Cena
Publisher : Royal Society of Chemistry
Release Date : 2013
ISBN 10 : 1849736111
Pages : 204 pages

Dendrimers are important molecules that are currently undergoing investigation for use in a variety of different biomedical applications. This book explores the use of dendrimers for a variety of potential functions, including antiamyloidogenic agents, drug delivery systems, nucleic acid and RNA delivery vectors and to produce hybrid fibre platforms for nantechnology. Following the work of COST action TD0802, the main objective of which is to improve existing therapies and find new drugs based on dendrimers, the book will provide comprehensive coverage of dendrimer applications. Coverage includes modelling and molecular dynamic studies of dendrimers and dendrons, anionic dendrimer polymers, cationic carbosilane dendrimers and self-assembled multivalent dendrimers. Providing clear indications for future research and applications, this text will appeal to chemists, biologists and materials scientists, working in both academia and industry.

Advanced Delivery and Therapeutic Applications of RNAi
Author : Kun Cheng,Ram I. Mahato
Publisher : John Wiley & Sons
Release Date : 2013-04-19
ISBN 10 : 1118610733
Pages : 536 pages

Commonly used by researchers to develop technologies for modifying and studying genetic process, RNA interference (RNAi) has many potential uses in medicine, biotechnology, and functional genomics. This book covers all essential aspects involved in the development of RNAi therapeutics, providing detailed guidance on the challenges and opportunities of bringing RNAi technologies from bench to clinic. It explores the design and mechanism of RNAi molecules, delivery strategies, and therapeutic applications in various diseases. Preclinical, regulatory, market, and intellectual aspects of RNAi technologies are also covered.

Pharmaceutical Perspectives of Cancer Therapeutics
Author : Yi Lu,Ram I. Mahato
Publisher : Springer Science & Business Media
Release Date : 2009-06-23
ISBN 10 : 9781441901316
Pages : 694 pages

Pharmaceutical Perspectives of Cancer Therapeutics covers a wide variety of therapeutic approaches including gene therapy, immunological therapy; cancer vaccines; strategy for solid tumors as well as for hematological cancers; methods to suppress tumor angiogenesis and metastasis; development and utilization of relevant animal models; introduction of new concepts such as cancer stem cells and new technologies, such as DNA and tissue microarrays; and RNA interference. In addition, clinical application, the development of DNA diagnosis biomarkers and cancer prevention, as well as the utilization of imaging in cancer therapy are also discussed. The use of synthetic carriers, such as lipids, polymers, and peptides for delivery and targeting of small molecules, proteins, and nucleic acids to cancer cells in vivo are discussed. Pharmaceutical Perspectives of Cancer Therapeutics also includes cancer therapy modality in surgery, chemotherapy, and radiotherapy, as well as in combination or multi-modality, giving our book a more focused view of cancer therapy.

Handbook of Applied Therapeutics
Author : Mary Anne Koda-Kimble
Publisher : Lippincott Williams & Wilkins
Release Date : 2007
ISBN 10 : 9780781790260
Pages : 1104 pages

This portable pocket version of Applied Therapeutics: The Clinical Use of Drugs, Eighth Edition offers quick access to the most pertinent drug-related information from the text. It follows the same chapter structure and approach as the textbook. This completely updated Eighth Edition provides in-depth information on the latest drug treatments for over 85 diseases and conditions, including heart failure, acne, schizophrenia, viral hepatitis, and diabetes mellitus. Detailed algorithms and charts guide students and practitioners through complex therapeutic decisions. More than 700 tables provide rapid access to comparative drug information, pharmacokinetic properties, treatment options, dosing guidelines, risk factors, and disease information.

Drug Delivery Systems
Author : Kewal K. Jain
Publisher : Springer Science & Business Media
Release Date : 2008-03-07
ISBN 10 : 1588298914
Pages : 251 pages

In this concise and systematic book, a team of experts select the most important, cutting-edge technologies used in drug delivery systems. They take into account significant drugs, new technologies such as nanoparticles, and therapeutic applications. The chapters present step-by-step laboratory protocols following the highly successful Methods in Molecular BiologyTM series format, offering readily reproducible results vital for pharmaceutical physicians and scientists.

Small Animal Clinical Pharmacology and Therapeutics - E-Book
Author : Dawn Merton Boothe
Publisher : Elsevier Health Sciences
Release Date : 2011-07-25
ISBN 10 : 1437723578
Pages : 1348 pages

Confidently utilize the rapidly growing selection of pharmaceuticals used to treat small animals. Small Animal Pharmacology and Therapeutics, 2nd Edition helps you understand both the therapeutic uses of common pharmaceuticals and the pharmacology behind them, giving you all of the information you need to design and modify dosing regimens, identify factors that cause drugs to fail, and anticipate adverse drug reactions. Comprehensive approach emphasizes the use of drugs for prevention as well as treatment. Clear, consistent organization makes it easy to find the information you need when you need it. Dosage tables help you find essential pharmaceutical information at a glance. Pharmacogenetics chapter helps you understand how to use this emerging science to find the right dose for each patient, optimizing efficiency and minimizing toxicity. Routes of administration and sample pharmaceutical calculations provide fast, efficient access to comprehensive drug administration all in one inclusive resource. Multiple chapters on Antimicrobial Drugs and Antimicrobial Therapy highlight the impact of antimicrobial resistance on current practice.

Therapeutics and Human Physiology
Author : Elsie Gaskell,Chris Rostron
Publisher : Oxford University Press
Release Date : 2013-02-14
ISBN 10 : 0199655294
Pages : 213 pages

The Integrated Foundations of Pharmacy series is for those at the start of their journey to become a pharmacist. It helps students understand how a drug molecule is made and then turned into a medicine; the role they will have when dispensing; and how the medicine works in the body. Most importantly, it shows how all of these aspects come together.

Kumar & Clark's Medical Management and Therapeutics - E-Book
Author : Michael L Clark,Parveen Kumar
Publisher : Elsevier Health Sciences
Release Date : 2011-07-06
ISBN 10 : 0702049123
Pages : 798 pages

Readers of Kumar & Clark's Clinical Medicine have consistently requested a handbook with the essentials for medical management and therapeutics, and this new handbook concentrates on the therapeutic side of what procedures to perform, how and - critically - why. Written by young doctors at the forefront of healthcare delivery, Kumar & Clark's Medical Management and Therapeutics is portable, easy to access when you need it most, and full of cutting edge information and insights. "Snappy and concise, just what the junior doctor ordered!" - Dr Lyn D Ferguson, FY1 "Prescriptive and to the point" - Catherine Drouot, 3rd year medical student, Barts "An excellent text for both medical students and junior doctors alike." - Dr Lucy Blair, FY1 Pocket-sized ward reference Concentrates on the management and treatment of medical disorders For junior doctors and senior medical students Perfect complement to Kumar & Clark's Clinical Medicine and Ballinger's Essentials of Kumar & Clark's Clinical Medicine

Anti-Aging Therapeutics Volume XIV
Author : A4m American Academy
Publisher : eBookIt.com
Release Date : 2012-10-15
ISBN 10 : 1934715107
Pages : 194 pages

Proceedings of the American Academy of Anti-Aging Medicine's (A4M) Nineteenth World Congress on Anti-Aging Medicine & Regenerative Biomedical Technologies, Spring and Winter Sessions (2011 conference year). Also includes Anti-Aging Clinical Protocols, 2012-2013,

Drug Discovery and Development - E-Book
Author : Raymond G Hill
Publisher : Elsevier Health Sciences
Release Date : 2012-07-20
ISBN 10 : 0702053163
Pages : 368 pages

The modern pharmacopeia has enormous power to alleviate disease, and owes its existence almost entirely to the work of the pharmaceutical industry. This book provides an introduction to the way the industry goes about the discovery and development of new drugs. The first part gives a brief historical account from its origins in the mediaeval apothecaries’ trade, and discusses the changing understanding of what we mean by disease, and what therapy aims to achieve, as well as summarising case histories of the discovery and development of some important drugs. The second part focuses on the science and technology involved in the discovery process: the stages by which a promising new chemical entity is identified, from the starting point of a medical need and an idea for addressing it. A chapter on biopharmaceuticals, whose discovery and development tend to follow routes somewhat different from synthetic compounds, is included here, as well as accounts of patent issues that arise in the discovery phase, and a chapter on research management in this environment. The third section of the book deals with drug development: the work that has to be undertaken to turn the drug candidate that emerges from the discovery process into a product on the market. The definitive introduction to how a pharmaceutical company goes about its business of discovering and developing drugs. The second edition has a new editor: Professor Raymond Hill ● non-executive director of Addex Pharmaceuticals, Covagen and of Orexo AB ● Visiting Industrial Professor of Pharmacology in the University of Bristol ● Visiting Professor in the School of Medical and Health Sciences at the University of Surrey ● Visiting Professor in Physiology and Pharmacology at the University of Strathclyde ● President and Chair of the Council of the British Pharmacological Society ● member of the Nuffield Council on Bioethics and the Advisory Council on Misuse of Drugs. New to this edition: Completely rewritten chapter on The Role of Medicinal Chemistry in the Drug Discovery Process. New topic - DMPK Optimization Strategy in drug discovery. New chapter on Scaffolds: Small globular proteins as antibody substitutes. Totally updated chapters on Intellectual Property and Marketing 50 new illustrations in full colour Features Accessible, general guide to pharmaceutical research and development. Examines the interfaces between cost and social benefit, quality control and mass production, regulatory bodies, patent management, and all interdisciplinary intersections essential to effective drug development. Written by a strong team of scientists with long experience in the pharmaceutical industry. Solid overview of all the steps from lab bench to market in an easy-to-understand way which will be accessible to non-specialists. From customer reviews of the previous edition: ‘... it will have everything you need to know on this module. Deeply referenced and, thus, deeply reliable. Highly Commended in the medicine category of the BMA 2006 medical book competition Winner of the Royal Society of Medicine Library Prize for Medical Book of the Year

Biotechnology and Biopharmaceuticals
Author : N.A
Publisher : John Wiley & Sons
Release Date : 2013-09-19
ISBN 10 : 1118659988
Pages : 744 pages

Biotechnology and Biopharmaceuticals: Transforming Proteins and Genes into Drugs, Second Edition addresses the pivotal issues relating to translational science, including preclinical and clinical drug development, regulatory science, pharmaco-economics and cost-effectiveness considerations. The new edition also provides an update on new proteins and genetic medicines, the translational and integrated sciences that continue to fuel the innovations in medicine, as well as the new areas of therapeutic development including cancer vaccines, stem cell therapeutics, and cell-based therapies.

Biological Drug Products
Author : Wei Wang,Manmohan Singh
Publisher : John Wiley & Sons
Release Date : 2013-08-29
ISBN 10 : 1118695224
Pages : 744 pages

Tested and proven solutions to the challenges of biologicaldrug product development Biological drug products play a central role in combating humandiseases; however, developing new successful biological drugspresents many challenges, including labor intensive productionprocesses, tighter regulatory controls, and increased marketcompetition. This book reviews the current state of the science,offering readers a single resource that sets forth the fundamentalsas well as tested and proven development strategies for biologicaldrugs. Moreover, the book prepares readers for the challenges thattypically arise during drug development, offering straightforwardsolutions to improve their ability to pass through all theregulatory hurdles and deliver new drug products to the market. Biological Drug Products begins with generalconsiderations for the development of any biological drug productand then explores the strategies and challenges involved in thedevelopment of specific types of biologics. Divided into fiveparts, the book examines: Part 1: General Aspects Part 2: Proteins and Peptides Part 3: Vaccines Part 4: Novel Biologics Part 5: Product Administration/Delivery Each chapter has been prepared by one or more leading experts inbiological drug development. Contributions are based on acomprehensive review and analysis of the current literature as wellas the authors' first-hand experience developing and testing newdrugs. References at the end of each chapter serve as a gateway tooriginal research papers and reviews in the field. By incorporating lessons learned and future directions forresearch, Biological Drug Products enables pharmaceuticalscientists and students to improve their success rate in developingnew biologics to treat a broad range of human diseases.

RNA Interference
Author : David R. Engelke,John J. Rossi
Publisher : Elsevier
Release Date : 2005-02-28
ISBN 10 : 0080455395
Pages : 496 pages

The critically acclaimed laboratory standard, Methods in Enzymology, is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. The series contains much material still relevant today - truly an essential publication for researchers in all fields of life sciences. RNA Interference will cover RNAi in non-vertebrates (plants, C. elegans, drosophila, and S. pombe), and Mammalian systems (human and non-human cells). This volume discusses extensive methodology related to delivery methods high throughput strategies and prospects as a human therapy agent. * One of the most highly respected publications in the field of biochemistry since 1955 * Frequently consulted, and praised by researchers and reviewers alike * Truly an essential publication for anyone in any field of the life sciences