Drug Delivery of SiRNA Therapeutics

Drug Delivery of SiRNA Therapeutics PDF Author: Gaetano Lamberti
Publisher:
ISBN: 9783039362004
Category :
Languages : en
Pages : 286

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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.

Drug Delivery of SiRNA Therapeutics

Drug Delivery of SiRNA Therapeutics PDF Author: Gaetano Lamberti
Publisher:
ISBN: 9783039362004
Category :
Languages : en
Pages : 286

Get Book Here

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.

siRNA and miRNA Gene Silencing

siRNA and miRNA Gene Silencing PDF Author: Mouldy Sioud
Publisher: Humana Press
ISBN: 9781603275460
Category : Science
Languages : en
Pages : 472

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Book Description
RNA interference has become a key method in the suppression of gene expression and the development of therapeutic agents, yet there is still the problem of delivery, stability, and the danger of off-target effects such as the silencing of unwanted genes and activation of innate immunity. In siRNA and miRNA Gene Silencing: From Bench to Bedside, expert researchers explore the most recent advances in siRNA design, expression, delivery, in vivo imaging, and methods to minimize siRNA’s unwanted effects and promote successful use in patients. As part of the highly successful Methods in Molecular BiologyTM series, the chapters focus on their respective subjects with easy-to-use, up-to-date information, including several step-by-step laboratory protocols on topics such as new delivery formulations and strategies with promising applications in vitro and in vivo, validated therapeutic target genes, and components of miRNA function, biogenesis, and interference with virus infection. Comprehensive and cutting-edge, siRNA and miRNA Gene Silencing: From Bench to Bedside offers an excellent collection of chapters to aid all those with an interest in RNAi, gene regulation, and new therapies.

Therapeutic Oligonucleotides

Therapeutic Oligonucleotides PDF Author: Jens Kurreck
Publisher: Royal Society of Chemistry
ISBN: 0854041168
Category : Medical
Languages : en
Pages : 362

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Book Description
This book provides a compelling overall update on current status of RNA interference

RNA Delivery Function for Anticancer Therapeutics

RNA Delivery Function for Anticancer Therapeutics PDF Author: Loutfy H. Madkour
Publisher: CRC Press
ISBN: 1000532240
Category : Science
Languages : en
Pages : 860

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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.

Therapeutic Applications of RNAi

Therapeutic Applications of RNAi PDF Author: John F. Reidhaar-Olson
Publisher: Humana Press
ISBN: 9781603272940
Category : Medical
Languages : en
Pages : 0

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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.

Design and Delivery of SiRNA Therapeutics

Design and Delivery of SiRNA Therapeutics PDF Author: Henrik J. Ditzel
Publisher:
ISBN: 9781071612989
Category : Drug development
Languages : en
Pages : 469

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Book Description
This volume details protocols on rationale design of therapeutic siRNA molecules and its encapsulation with smart vehicles to overcome the barriers to an effective administration in vivo. 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. Authoritative and cutting-edge, Design and Delivery of SiRNA Therapeutics aims to ensure successful results in the further study of this vital field. This volume details protocols on rationale design of therapeutic siRNA molecules and its encapsulation with smart vehicles to overcome the barriers to an effective administration in vivo. 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. Authoritative and cutting-edge, Design and Delivery of SiRNA Therapeutics aims to ensure successful results in the further study of this vital field.

RNA Nanotechnology

RNA Nanotechnology PDF Author: Bin Wang
Publisher: CRC Press
ISBN: 9814411647
Category : Medical
Languages : en
Pages : 468

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Book Description
In the past few decades there has been incredible growth in "bionano"-related research, which has been accompanied by numerous publications in this field. Although various compilations address topics related to deoxyribonucleic acid (DNA) and protein, there are few books that focus on determining the structure of ribonucleic acid (RNA) and using RNA as building blocks to construct nanoarchitectures for biomedical and healthcare applications. RNA Nanotechnology is a comprehensive volume that details both the traditional approaches and the latest developments in the field of RNA-related technology. This book targets a wide audience: a broad introduction provides a solid academic background for students, researchers, and scientists who are unfamiliar with the subject, while the in-depth descriptions and discussions are useful for advanced professionals. The book opens with reviews on the basic aspects of RNA biology, computational approaches for predicting RNA structures, and traditional and emerging experimental approaches for probing RNA structures. This section is followed by explorations of the latest research and discoveries in RNA nanotechnology, including the design and construction of RNA-based nanostructures. The final segment of the book includes descriptions and discussions of the potential biological and therapeutic applications of small RNA molecules, such as small/short interfering RNAs (siRNAs), microRNAs (miRNAs), RNA aptamers, and ribozymes.

Nanotechnology for the Delivery of Therapeutic Nucleic Acids

Nanotechnology for the Delivery of Therapeutic Nucleic Acids PDF Author: Dan Peer
Publisher: CRC Press
ISBN: 9814411043
Category : Medical
Languages : en
Pages : 242

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Book Description
Nucleic acid (NA) therapeutics has been extensively studied both in the academia and in the pharmaceutical industry and is still considered the promise for new therapeutic modalities, especially in personalized medicine. The only hurdle that limits the translation of NA therapeutics from an academic idea to the new therapeutic modality is the lack of efficient and safe delivery strategies. Nanotechnology for the Delivery of Therapeutic Nucleic Acids, written by world experts in the field of nanotechnology for NA delivery, the contributing authors bring together the state of the art in delivery strategies with strong emphasis on aspects that are of essence to the pharmaceutical industry, such as stability, general toxicity, immune-toxicity, pharmacokinetics, efficacy, and validation of new drug targets using unique approaches based on exquisite nanotechnology strategies.

Nanopharmaceutical Advanced Delivery Systems

Nanopharmaceutical Advanced Delivery Systems PDF Author: Vivek Dave
Publisher: John Wiley & Sons
ISBN: 1119711681
Category : Computers
Languages : en
Pages : 467

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Book Description
The book provides a single volume covering detailed descriptions about various delivery systems, their principles and how these are put in use for the treatment of multiple diseases. It is divided into four sections where the first section deals with the introduction and importance of novel drug delivery system. The second section deals with the most advanced drug delivery systems like microbubbles, dendrimers, lipid-based nanoparticles, nanofibers, microemulsions etc., describing the major principles and techniques of the preparations of the drug delivery systems. The third section elaborates on the treatments of diverse diseases like cancer, topical diseases, tuberculosis etc. The fourth and final section provides a brief informative description about the regulatory aspects of novel drug delivery system that is followed in various countries.

Nucleic Acids

Nucleic Acids PDF Author: Marcelo Larramendy
Publisher: BoD – Books on Demand
ISBN: 9535122649
Category : Science
Languages : en
Pages : 222

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Book Description
This edited book, "Nucleic Acids - From Basic Aspects to Laboratory Tools", contains a series of chapters that highlight the development and status of the various aspects of the nucleic acids related to DNA chemistry and biology and the molecular application of these small DNA molecules and related synthetic analogues within biological systems. Furthermore, it is hoped that the information in the present book will be of value to those directly engaged in the handling and use of nucleic acids, and that this book will continue to meet the expectations and needs of all who are interested in the different fascinating aspects of molecular biology.