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RNA Therapeutics

RNA Therapeutics
Function, Design, and Delivery

by Mouldy Sioud

  • Publisher : Humana
  • Release : 2016-08-23
  • Pages : 526
  • ISBN : 9781493961511
  • Language : En, Es, Fr & De
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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 Therapeutics

RNA Therapeutics
A Book

by Amanda L. Garner

  • Publisher : Springer
  • Release : 2018-08-15
  • Pages : 259
  • ISBN : 9783319885469
  • Language : En, Es, Fr & De
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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

Entering the RNA Wonderland: Opportunities and Challenges for RNA Therapeutics in the Cardiovascular System
A Book

by Anna Zampetaki,Lars Maegdefessel

  • Publisher : Frontiers Media SA
  • Release : 2020-07-22
  • Pages : 129
  • ISBN : 2889638820
  • Language : En, Es, Fr & De
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RNA Therapeutics

RNA Therapeutics
The Evolving Landscape of RNA Therapeutics

by Paloma H. Giangrande,Vittorio Defranciscis,John J. Rossi

  • Publisher : Academic Press
  • Release : 2022-02-01
  • Pages : 512
  • ISBN : 0128217391
  • Language : En, Es, Fr & De
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RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an 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 a 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

RNA Interference from Biology to Therapeutics

RNA Interference from Biology to Therapeutics
A Book

by Kenneth A. Howard

  • Publisher : Springer Science & Business Media
  • Release : 2012-10-19
  • Pages : 340
  • ISBN : 1461447445
  • Language : En, Es, Fr & De
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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 Technologies and Their Applications

RNA Technologies and Their Applications
A Book

by Volker A. Erdmann,Jan Barciszewski

  • Publisher : Springer Science & Business Media
  • Release : 2010-09-02
  • Pages : 452
  • ISBN : 9783642121685
  • Language : En, Es, Fr & De
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RNA technologies are the driving forces of modern medicine and biotechnology. They combine the fields of biochemistry, chemistry, molecular biology, cell biology, physics, nanotechnology and bioinformatics. The combination of these topics is set to revolutionize the medicine of tomorrow. After more than 15 years of extensive research in the field of RNA technologies, the first therapeutics are ready to reach the first patients. Thus we are witnessing the birth of a very exciting time in the development of molecular medicine, which will be based on the methods of RNA technologies. This volume is the first of a series. It covers various aspects of RNA interference and microRNAs, although antisense RNA applications, hammerhead ribozyme structure and function as well as non-coding RNAs are also discussed. The authors are internationally highly respected experts in the field of RNA technologies.

RNA-Based Regulation in Human Health and Disease

RNA-Based Regulation in Human Health and Disease
A Book

by Anonim

  • Publisher : Academic Press
  • Release : 2020-08-15
  • Pages : 444
  • ISBN : 0128171944
  • Language : En, Es, Fr & De
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RNA-based Regulation in Human Health and Disease offers an in-depth exploration of RNA mediated genome regulation at different hierarchies. Beginning with multitude of canonical and non-canonical RNA populations, especially noncoding RNA in human physiology and evolution, further sections examine the various classes of RNAs (from small to large noncoding and extracellular RNAs), functional categories of RNA regulation (RNA-binding proteins, alternative splicing, RNA editing, antisense transcripts and RNA G-quadruplexes), dynamic aspects of RNA regulation modulating physiological homeostasis (aging), role of RNA beyond humans, tools and technologies for RNA research (wet lab and computational) and future prospects for RNA-based diagnostics and therapeutics. One of the core strengths of the book includes spectrum of disease-specific chapters from experts in the field highlighting RNA-based regulation in metabolic & neurodegenerative disorders, cancer, inflammatory disease, viral and bacterial infections. We hope the book helps researchers, students and clinicians appreciate the role of RNA-based regulation in genome regulation, aiding the development of useful biomarkers for prognosis, diagnosis, and novel RNA-based therapeutics. Comprehensive information of non-canonical RNA-based genome regulation modulating human health and disease Defines RNA classes with special emphasis on unexplored world of noncoding RNA at different hierarchies Disease specific role of RNA - causal, prognostic, diagnostic and therapeutic Features contributions from leading experts in the field

Long Non-Coding RNA

Long Non-Coding RNA
From Disease Biomarkers to Targeted Therapeutics

by Syed Mudasir Ahmad,Nazir Ahmad Ganai

  • Publisher : Cambridge Scholars Publishing
  • Release : 2020-09-03
  • Pages : 201
  • ISBN : 1527559106
  • Language : En, Es, Fr & De
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Long non-coding RNAs (LncRNAs) were originally regarded as “noise” in the genome due to their lack of protein-encoding capacity. However, their roles are now understood to cover various biological functions like gene regulation, cell proliferation, survival, differentiation, and chromatin remodeling. As an emerging group of non-coding RNA molecules, LncRNAs are involved in diverse diseases like cancer, cardiovascular, autoimmune, hormonal disorders, neurodegeneration, and infection, among others. However, knowledge of the mechanisms by which they act is still ambiguous. As such, this book meets the essential need for a textbook dedicated to this topic and provides a comprehensive and captivating account of lncRNA research.

Design and Application of RNA Therapeutics for Splice Site Mutations

Design and Application of RNA Therapeutics for Splice Site Mutations
A Book

by Anonim

  • Publisher : Unknown Publisher
  • Release : 2015
  • Pages : 67
  • ISBN : 9789175499611
  • Language : En, Es, Fr & De
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RNA Nanotechnology and Therapeutics

RNA Nanotechnology and Therapeutics
A Book

by Peixuan Guo,Farzin Haque

  • Publisher : CRC Press
  • Release : 2013-07-09
  • Pages : 611
  • ISBN : 1466505664
  • Language : En, Es, Fr & De
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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 RNA nanotechnology. In addition, the text also provides in-depth discussions of RNA structure for nanoparticle construction, RNA computation and modeling, single molecule imaging of RNA, RNA nanoparticle assembly, RNA nanoparticles in therapeutics, RNA chemistry for nanoparticle synthesis, and conjugation and labeling.

AGO-Driven Non-Coding RNAs

AGO-Driven Non-Coding RNAs
Codes to Decode the Therapeutics of Diseases

by Bibekanand Mallick

  • Publisher : Academic Press
  • Release : 2019-01-20
  • Pages : 387
  • ISBN : 0128156708
  • Language : En, Es, Fr & De
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The ‘RNA world’ hypothesis that proposed RNA molecules as the first form of genetic material was put forwarded in the late 1980s but got impetus only recently when high-throughput sequencing technologies began unearthing new types of non-coding RNA (ncRNA) transcripts in higher eukaryotes. Till then, research on ncRNAs were primarily confined to transfer RNAs and, ribosomal RNAs, which act as the messengers of the protein synthesis and allow translation of genetic information encoded by DNA into proteins. In recent years, the integration of high-throughput genomic technologies with molecular biology and omics sciences have revolutionized the fields of ncRNA research by identifying the hidden treasures of several new types of ncRNAs encoded in the genomes of several organisms and decrypting their versatile roles in gene expression and epigenetics. Among these, two small endogenous ncRNAs, namely microRNAs (miRNAs) and piwi-interacting RNAs (piRNAs) that drive argonaute (AGO) family of proteins namely Ago and Piwi respectively and silence the expression of genes have geared up molecular and disease biology research in recent years. Both miRNAs and piRNAs are expressed in higher eukaryotes, including human and act as cellular rheostats by regulating the expression of significant fraction of genes encoded in the genomes. The aberrant expressions of these small ncRNAs within the cells cause various abnormalities and diseases including cancer. Manipulating their aberrant expression or function can serve as potential novel diagnostic/prognostic biomarkers and bring in new therapeutic strategies for multiple human diseases. This can be further translated from bench-side to clinic for improving human health. This book captures the essence of the pioneering work of some of the world’s leading researchers showcasing the scientific excitements surrounding the evolving regulatory roles of miRNAs and piRNAs highlighting their potential towards the diagnosis and therapeutics of various diseases. The book is geared towards scientists, students, and will particularly appeal to active investigators in RNA biology, molecular biology, cancer research as well as clinicians and will provide them a comprehensive view of recent discoveries and research progresses to utilize miRNAs, piRNAs and their interacting proteins, Ago and Piwi for diagnosis, prognosis and therapeutics of diseases. Provides a unified cutting-edge resource for both miRNAs and piRNAs, two promising AGO-clade small ncRNAs, their functions and potential applications Showcase high-throughput technologies and other approaches for discovery of these small ncRNAs and their targets Unveils the diverse molecular mechanisms by which miRNAs and piRNAs regulate gene expression in animal cells Showcase recent discoveries on involvement of Argonaute and Piwi proteins in different biological processes and diseases as well as their possible use in diagnosis Report breakthroughs in the use of small ncRNAs for diagnosis and personalized therapy

RNA Nanotechnology and Therapeutics

RNA Nanotechnology and Therapeutics
A Book

by Peixuan Guo,Farzin Haque

  • Publisher : CRC Press
  • Release : 2013-07-09
  • Pages : 611
  • ISBN : 1466505834
  • Language : En, Es, Fr & De
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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

Therapeutic RNA Nanotechnology

Therapeutic RNA Nanotechnology
Immunomodulation and Dynamicity

by Kirill A. Afonin,Morgan Chandler

  • Publisher : CRC Press
  • Release : 2021-07-22
  • Pages : 1228
  • ISBN : 1000263479
  • Language : En, Es, Fr & De
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This collection of research articles and reviews covers the latest work in the design, delivery, dynamic abilities, and immune stimulation of RNA nanoparticles which have driven the utilization of their immunomodulatory properties. The unknown immune properties of nucleic acid nanoparticles have been a major hurdle in their adaptation until the works herein began assessing their structure-activity relationships. This collection chronologically follows the path of investigating the recognition of design components to implementing them into nucleic acid nanostructures. RNA nanotechnology is an emerging platform for therapeutics with increasing clinical relevance as this approach becomes more widely used and approved for the treatment of various diseases. The latest research aims to take advantage of RNA’s modular nature for the design of nanostructures which can interact with their environments to communicate programmed messages with intracellular pathways. In doing so, nanoparticles can be used to elicit or elude responses by the immune system as desired in conjunction with their therapeutic applications. This collection of research articles and reviews covers the latest work in the design, delivery, dynamic abilities, and immune stimulation of RNA nanoparticles which have driven the utilization of their immunomodulatory properties.

From Nucleic Acids Sequences to Molecular Medicine

From Nucleic Acids Sequences to Molecular Medicine
A Book

by Volker A. Erdmann,Jan Barciszewski

  • Publisher : Springer Science & Business Media
  • Release : 2012-06-13
  • Pages : 654
  • ISBN : 3642274269
  • Language : En, Es, Fr & De
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Despite a half century of structural, biophysical and biochemical investigations of ribonucleic acids, they are still mysterious. RNAs stand at fertile crossroads of disciplines, integrating concepts from genomics, proteomics, dynamics as well as biochemistry and molecular biology. From 20 years it is clear, that genetic regulation of eukaryotic organisms has been misunderstood for the last years that the expression of genetic information is effected only by proteins. Basic understanding of nucleic acids has enhanced our foundation to probe novel biological functions. This is especially evident for RNA molecules whose functionality, maturation, and regulation require formation of correct secondary structure through encoded base-pairing interactions.

Nucleic Acid Therapeutics in Cancer

Nucleic Acid Therapeutics in Cancer
A Book

by Alan M. Gewirtz

  • Publisher : Springer Science & Business Media
  • Release : 2004-03-25
  • Pages : 221
  • ISBN : 1592597777
  • Language : En, Es, Fr & De
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A cutting-edge review of the important issues underlying the therapeutic use of nucleic acid-mediated gene silencing. Topics range from basic methodology and delivery to targeting and clinical targets. The authors thoroughly explain the latest developments in RNA biology, as well as the underpinnings of RNA interference, oligodeoxynucleotide delivery into cells, and strategies for targeting these molecules to accessible regions within the mRNA. They also provide some examples of how these new therapeutic compounds are being used clinically.

The Use of CRISPR/cas9, ZFNs, TALENs in Generating Site-Specific Genome Alterations

The Use of CRISPR/cas9, ZFNs, TALENs in Generating Site-Specific Genome Alterations
A Book

by Anonim

  • Publisher : Academic Press
  • Release : 2014-11-04
  • Pages : 570
  • ISBN : 0128013346
  • Language : En, Es, Fr & De
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This new volume of Methods in Enzymology continues the legacy of this premier serial with quality chapters authored by leaders in the field. This volume covers recent research and methods development for changing the DNA sequence within the genomes of cells and organisms. Focusing on enzymes that generate double-strand breaks in DNA, the chapters describe use of molecular tools to introduce or delete genetic information at specific sites in the genomes of animal, plant and bacterial cells. Continues the legacy of this premier serial with quality chapters authored by leaders in the field Covers research methods in biomineralization science Contains sections on such topics as genome editing, genome engineering, CRISPR, Cas9, TALEN and zinc finger nuclease

Tumor-penetrating Delivery of Small Interfering RNA Therapeutics

Tumor-penetrating Delivery of Small Interfering RNA Therapeutics
A Book

by Yin Ren (Ph. D.),Harvard--MIT Program in Health Sciences and Technology

  • Publisher : Unknown Publisher
  • Release : 2012
  • Pages : 254
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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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.

Therapeutic RNA Nanotechnology

Therapeutic RNA Nanotechnology
Immunomodulation and Dynamicity

by Kirill A. Afonin,Morgan Chandler

  • Publisher : CRC Press
  • Release : 2021-07-22
  • Pages : 1228
  • ISBN : 1000263177
  • Language : En, Es, Fr & De
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This collection of research articles and reviews covers the latest work in the design, delivery, dynamic abilities, and immune stimulation of RNA nanoparticles which have driven the utilization of their immunomodulatory properties. The unknown immune properties of nucleic acid nanoparticles have been a major hurdle in their adaptation until the works herein began assessing their structure-activity relationships. This collection chronologically follows the path of investigating the recognition of design components to implementing them into nucleic acid nanostructures. RNA nanotechnology is an emerging platform for therapeutics with increasing clinical relevance as this approach becomes more widely used and approved for the treatment of various diseases. The latest research aims to take advantage of RNA’s modular nature for the design of nanostructures which can interact with their environments to communicate programmed messages with intracellular pathways. In doing so, nanoparticles can be used to elicit or elude responses by the immune system as desired in conjunction with their therapeutic applications.

RNA Interference

RNA Interference
Application to Drug Discovery and Challenges to Pharmaceutical Development

by Paul H. Johnson

  • Publisher : John Wiley & Sons
  • Release : 2011-03-01
  • Pages : 328
  • ISBN : 9780470934678
  • Language : En, Es, Fr & De
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RNA Interference: Application to Drug Discovery and Challenges to Pharmaceutical Development provides a general overview of this rapidly emerging field, with a strong emphasis on issues and aspects that are important to a drug development team. The first part covers more general background of RNA interference and its application in drug discovery. In the second part, the book addresses siRNA (small interfering RNA), a pharmaceutically potent form, and its use and delivery in therapeutics along with manufacturing and delivery aspects.

Gold Nanoparticle Enhancements in Electroporation Mediated DNA and RNA Therapeutics

Gold Nanoparticle Enhancements in Electroporation Mediated DNA and RNA Therapeutics
A Book

by Shuyan Huang

  • Publisher : Unknown Publisher
  • Release : 2015
  • Pages : 224
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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