Advanced Delivery and Therapeutic Applications of RNAi PDF Download

Author: Kun Cheng
Publisher: John Wiley & Sons
ISBN: 1118610733
Category : Science
Languages : en
Pages : 660

View

Book Description
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.

Author: Kenneth A. Howard
Publisher: Springer Science & Business Media
ISBN: 1461447437
Category : Medical
Languages : en
Pages : 348

View

Book Description
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.

Author: John F. Reidhaar-Olson
Publisher: Humana Press
ISBN: 9781617794940
Category : Medical
Languages : en
Pages : 0

View

Book Description
The phenomenon of RNA interference has rapidly moved from groundbreaking scientific discovery to promising therapeutic approach. However, even as RNAi-based drugs enter the clinic, significant challenges remain, particulary in the area of delivery. Therapeutic Applications of RNAi provides detailed protocols in key areas of current focus, including testing of delivery vehicles, identification of appropriate model systems, and evaluation of the effects of RNAi in vivo. Produced by a team of internationally renowned authors, the volume describes the therapeutic applications of RNAi and potential pitfalls in oncology, viral infections and CNS disease, using a variety of delivery methods, including liposomes, peptide-based nanoparticles, polycationic polymers, and viral vehicles. Written in the highly successful Methods in Molecular BiologyTM series format, the chapters include brief introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Therapeutic Applications of RNAi is an ideal guide for scientists attempting to solve the numerous challenges in this field and revolutionize the treatment of disease.

Author: Gaetano Lamberti
Publisher:
ISBN: 9783039362004
Category :
Languages : en
Pages : 286

View

Book Description
The new frontier of pharmaceutical sciences is gene therapy, which is the use of molecules able to interact directly with the expression of the genetic material of the patient as well as of the disease-causing guest (bacteria, virus, parasites, and tumor cells). Among the molecules of interest for gene therapy, a relevant role is played by small interfering RNA (siRNA) molecules able to interfere with the expression of genes of interest for some diseases. However, siRNA molecules, even if they are powerful as drugs, are difficult to deliver since they are sensitive to enzymes present in plasma and they are large and negatively charged, so are difficult to administer into the cell nuclei, since the cell walls are scarcely permeable to large molecules and are also negatively charged. Therefore, the focus of research on siRNA-based therapies is their delivery, which can be performed by chemical modification, association with aptamers or polycations, or embedding them into properly designed liposomes. This book is centered on the more recent development in siRNA delivery techniques toward the clinical applications of this potent class of drugs.

Author: Patrick Arbuthnot
Publisher: Caister Academic Press Limited
ISBN: 9781908230430
Category : Science
Languages : en
Pages : 0

View

Book Description
Since the discovery of RNA interference (RNAi) in 1998, research on the topic has advanced at an impressive pace. Small RNAs and, in particular, micro RNAs (miRNAs) play a fundamental role in gene regulation through the activation of RNAi. Detailed insights into the mechanisms of RNAi have led to an improved understanding of gene regulation in normal and disease states, and thereby enabled the exploitation of RNAi for a variety of applications. In this book, an international panel of RNAi experts critically reviews the most interesting advances in basic applied RNAi research, highlighting the applications in RNAi-based therapies and discussing the technical hurdles that remain. Topics covering the fundamental biological aspects of applied RNAi research include: the role of miRNAs in trinucleotide repeat disorders * miRNAs and HIV pathogenesis * miRNAs for epigenetic gene silencing * the role of miRNAs in virus-related cancers * non-canonical miRNA biogenesis. In the area of RNAi-based therapy, topics include: harnessing RNAi for the treatment of viral infections * optimizing the design of exogenous RNAi activators * blocking miRNA function with synthetic agents * somatic cell reprogramming * high-content miRNA-based screening tools * the use of miRNA target sites for control of transgene expression. The book will be essential reading for everyone involved in RNAi research, drug discovery and delivery, biomedical engineering, and biomaterials. Subject: Molecular Biology, Genomics, Life Science]

Author: Loutfy H. Madkour
Publisher: CRC Press
ISBN: 1000532240
Category : Science
Languages : en
Pages : 860

View

Book Description
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.

Author: Volker A. Erdmann
Publisher: Springer Science & Business Media
ISBN: 3642121683
Category : Medical
Languages : en
Pages : 460

View

Book Description
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.

Author: R. K. Gaur
Publisher: CRC Press
ISBN: 1439873259
Category : Science
Languages : en
Pages : 380

View

Book Description
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: Muhammad Sohail
Publisher: CRC Press
ISBN: 020348925X
Category : Medical
Languages : en
Pages : 398

View

Book Description
Maximizing the potential of RNA interference in functional genomics - as well as in the development of therapeutics - continues to be at the forefront of biomedical research. Unlike journal articles, Gene Silencing by RNA Interference: Technology and Application combines essential background to the RNAi field with practical techniques designed by r

Author: Stanley T. Crooke
Publisher: CRC Press
ISBN: 1439832501
Category : Medical
Languages : en
Pages : 848

View

Book Description
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