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Author : Olga N. Ilinskaya,Hector A. Cabrera-Fuentes,Derek John Hausenloy,Marina A. Zenkova
Publisher : Frontiers Media SA
Page : 251 pages
File Size : 43,6 Mb
Release : 2020-06-04
Category : RNA targeting
ISBN : 9782889637751
Author : Stanley T. Crooke
Publisher : CRC Press
Page : 848 pages
File Size : 41,9 Mb
Release : 2007-07-25
Category : Medical
ISBN : 9781439832509
Extensively revised and updated, Antisense Drug Technology: Principles, Strategies, and Applications, Second Edition reflects the logarithmic progress made in the past four years of oligonucleotide-based therapies, and, in particular, antisense therapeutics and research. Interpreting lessons learned from the clinical trials of first generati
Author : Mouldy Sioud
Publisher : Humana Press
Page : 527 pages
File Size : 43,9 Mb
Release : 2011-03-04
Category : Science
ISBN : 1607616645
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.
Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Page : 93 pages
File Size : 52,6 Mb
Release : 2019-12-01
Category : Medical
ISBN : 9780309495844
On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.
Author : R. K. Gaur,Yedidya Gafni,P. Sharma,V. K. Gupta
Publisher : CRC Press
Page : 380 pages
File Size : 52,9 Mb
Release : 2016-04-19
Category : Science
ISBN : 9781439873250
RNAi technology is used for large-scale screens that systematically shut down each gene in the cell, which can help identify the components necessary for a particular cellular process or an event such as cell division. Exploitation of the pathway is also a promising tool in biotechnology and medicine. Introducing new technology in the study of RNA
Author : Michael J Gait,Sudhir Agrawal
Publisher : Royal Society of Chemistry
Page : 566 pages
File Size : 49,9 Mb
Release : 2019-02-11
Category : Science
ISBN : 9781788012096
The sequencing of the human genome and subsequent elucidation of the molecular pathways that are important in the pathology of disease have provided unprecedented opportunities for the development of new therapeutics. Nucleic acid-based drugs have emerged in recent years to yield extremely promising candidates for drug therapy to a wide range of diseases. Advances in Nucleic Acid Therapeutics is a comprehensive review of the latest advances in the field, covering the background of the development of nucleic acids for therapeutic purposes to the array of drug development approaches currently being pursued using antisense, RNAi, aptamer, immune modulatory and other synthetic oligonucleotides. Nucleic acid therapeutics is a field that has been continually innovating to meet the challenges of drug discovery and development; bringing contributions together from leaders at the forefront of progress, this book depicts the many approaches currently being pursued in both academia and industry. A go-to volume for medicinal chemists, Advances in Nucleic Acid Therapeutics provides a broad overview of techniques of contemporary interest in drug discovery.
Author : Danzhou Yang,Clement Lin
Publisher : Humana
Page : 437 pages
File Size : 45,9 Mb
Release : 2020-09-07
Category : Medical
ISBN : 1493996681
This volume covers the structures, properties, and functions of G-quadruplexes in a wide range of biological disciplines, including therapeutic intervention and biomaterial application. The chapters in this book explore a wide range of vital and new experimental techniques used in the study of G-quadruplexes. 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, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Practical and cutting-edge, G-Quadruplex DNA: Methods and Protocols is a valuable resource for both novice and experienced researchers who work in biophysics, structural biology, computational biology, biochemistry, and molecular and cell biology, and who want to learn more about the potential roles and effects of G-quadruplex in these fields.
Author : Kazuwa Nakao,Nagahiro Minato,Shinji Uemoto
Publisher : Springer
Page : 330 pages
File Size : 44,6 Mb
Release : 2015-10-13
Category : Science
ISBN : 9784431556510
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.
Author : John Schneekloth,Martin Pettersson
Publisher : Wiley-VCH
Page : 0 pages
File Size : 52,6 Mb
Release : 2024-10-21
Category : Medical
ISBN : 3527351000
Addressing a long-standing challenge for medicinal chemists, this practical reference provides the concepts and tools for developing drug-like small molecules that selectively modulate non-coding RNAs. The introductory parts describes the wide range of druggable RNAs, available techniques for their characterization, binding assay development as well privileged structures in small molecule RNA binders. The main part systematically covers different RNA targets and how to approach them from a drug discovery angle, from splice modulation to riboswitches to targeted degradation, translation blockers and RNA modification. Throughout, the first successful examples of RNA-targeting drug candidates from the development pipeline of small and large pharmaceutical companies are discussed. This seminal reference on a new paradigm in drug discovery is required reading for every medicinal chemists who needs to stay abreast of current developments.
Author : National Academies of Sciences, Engineering, and Medicine,Division on Earth and Life Studies,Board on Life Sciences,Board on Chemical Sciences and Technology,Committee on Strategies for Identifying and Addressing Potential Biodefense Vulnerabilities Posed by Synthetic Biology
Publisher : National Academies Press
Page : 189 pages
File Size : 52,9 Mb
Release : 2019-01-05
Category : Technology & Engineering
ISBN : 9780309465182
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.
Author : Renato Baserga,David T. Denhardt
Publisher : Unknown
Page : 372 pages
File Size : 43,9 Mb
Release : 1992
Category : Science
ISBN : UVA:X002192859
The antisense strategy has been used to study cellular proliferation and differentiation as well as to target chemotherapy against viral products such as HIV. It shows promise in the isolation of bone marrow stem cells and the purging of bone marrow. This volume, covering cellular proliferation, inhibition of viral replication, ribozymes-transcriptural regulation, cellular differentiation, and transgenic animals should be important to both basic and clinical scientists.
Author : Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Publisher : Springer Science & Business Media
Page : 469 pages
File Size : 42,6 Mb
Release : 2007-10-26
Category : Medical
ISBN : 9781597452229
The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.
Author : Aamir Ahmad
Publisher : Springer Nature
Page : 427 pages
File Size : 41,9 Mb
Release : 2019-08-27
Category : Medical
ISBN : 9783030203016
Resistance to therapies, both targeted and systemic, and metastases to distant organs are the underlying causes of breast cancer-associated mortality. The second edition of Breast Cancer Metastasis and Drug Resistance brings together some of the leading experts to comprehensively understand breast cancer: the factors that make it lethal, and current research and clinical progress. This volume covers the following core topics: basic understanding of breast cancer (statistics, epidemiology, racial disparity and heterogeneity), metastasis and drug resistance (bone metastasis, trastuzumab resistance, tamoxifen resistance and novel therapeutic targets, including non-coding RNAs, inflammatory cytokines, cancer stem cells, ubiquitin ligases, tumor microenvironment and signaling pathways such as TRAIL, JAK-STAT and mTOR) and recent developments in the field (epigenetic regulation, microRNAs-mediated regulation, novel therapies and the clinically relevant 3D models). Experts also discuss the advances in laboratory research along with their translational and clinical implications with an overarching goal to improve the diagnosis and prognosis, particularly that of breast cancer patients with advanced disease.
Author : Bruce Alberts
Publisher : Unknown
Page : 0 pages
File Size : 48,8 Mb
Release : 2002
Category : Cytology
ISBN : 0815332181
Author : Dagmar Klostermeier,Christian Hammann
Publisher : Walter de Gruyter
Page : 432 pages
File Size : 50,9 Mb
Release : 2013-10-14
Category : Science
ISBN : 9783110284959
While structure-function relationships of proteins have been studied for a long time, structural studies of RNA face additional challenges. Nevertheless, with the continuous discovery of novel RNA molecules with key cellular functions and of novel pathways and interaction networks, the need for structural information of RNA is still increasing. This volume provides an introduction into techniques to assess structure and folding of RNA. Each chapter explains the theoretical background of one technique, and illustrates possibilities and limitations in selected application examples.
