Download or read book Drug Delivery of SiRNA Therapeutics written by Gaetano Lamberti and published by . This book was released on 2020-06-15 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Design and Delivery of SiRNA Therapeutics written by Henrik J. Ditzel and published by . This book was released on 2021 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book siRNA and miRNA Gene Silencing written by Mouldy Sioud and published by Humana Press. This book was released on 2008-11-26 with total page 472 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Therapeutic Oligonucleotides written by Jens Kurreck and published by Royal Society of Chemistry. This book was released on 2008 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a compelling overall update on current status of RNA interference
Download or read book RNA Nanotechnology written by Bin Wang and published by CRC Press. This book was released on 2014-04-02 with total page 468 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Nucleic Acids as Gene Anticancer Drug Delivery Therapy written by Loutfy H. Madkour and published by Academic Press. This book was released on 2019-08-27 with total page 650 pages. Available in PDF, EPUB and Kindle. Book excerpt: Nucleic Acids as Gene Anticancer Drug Delivery Therapy highlights the most recent developments in cancer treatment using nucleic acids, nanoparticles and polymer nanoparticles for genomic nanocarriers as drug delivery, including promising opportunities for targeted and combination therapy. The development of a wide spectrum of nanoscale technologies is beginning to change the scientific landscape in terms of disease diagnosis, treatment, and prevention. This book presents the use of nanotechnology for medical applications, focusing on its use for anticancer drug delivery. Various intelligent drug delivery systems such as inorganic nanoparticles and polymer-based drug delivery are discussed. The use of smart drug delivery systems seems to be a promising approach for developing intelligent therapeutic systems for cancer immunotherapies and is discussed in detail along with nucleic acid-targeted drug delivery combination therapy for cancer. Nucleic Acids as Gene Anticancer Drug Delivery Therapy will be a useful reference for pharmaceutical scientists, pharmacologiests, and those involved in nanotechnology and cancer research. Discusses intelligent drug delivery systems such as inorganic nanoparticles and polymer-based drug delivery Contains a comprehensive comparison of various delivery systems, listing their advantages and limitations Presents combination therapy as a new hope for enhancing current gene-based treatment efficacy
Download or read book SiRNA Delivery Methods written by Kato Shum and published by Humana. This book was released on 2015-10-16 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume provides a collection of cutting-edge strategies in siRNA delivery that were developed and refined over the years with tried-and-true methods. Written by a team of internationally renowned authors, this book describes, in detail, a variety of successful siRNA delivery methods, including peptide-based nanoparticles, liposomes, exosomes, polymers, aptamers, and viral vehicles. Written in the highly successful Methods in Molecular Biology series format, each proven protocol includes brief 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. Comprehensive and authoritative, SiRNA Delivery Methods: Methods and Protocols, will provide researchers, educators, clinicians, and biotechnology specialists with a broad understanding of the issues in siRNA delivery and how they can be overcome strategically.
Download or read book Drug Delivery Systems written by Kewal K. Jain and published by Springer Science & Business Media. This book was released on 2008-03-07 with total page 255 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book RNA Delivery Function for Anticancer Therapeutics written by Loutfy Hamid Madkour and published by CRC Press. This book was released on 2022 with total page 403 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This book describes the status of clinical trials related to siRNA-based cancer therapy, as well as the remaining issues that need to be overcome to establish a successful therapy. It, then introduces various promising design strategies of delivery vehicles for stable and targeted siRNA delivery, including the prospects for future design"--
Download or read book Nanotechnology for the Delivery of Therapeutic Nucleic Acids written by Dan Peer and published by CRC Press. This book was released on 2013-03-27 with total page 242 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Lipid Modification of Polymeric Nanocarriers for Drug and SiRNA Delivery written by Arash Falamarzian and published by . This book was released on 2013 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: The use of nanotechnology in pharmaceutical development has progressed significantly in recent decades. This rapid advancement is driven by crucial need for improving the performance of present diagnostic and therapeutic modalities, as well as development of a new class of delivery systems for complex entities such as genes and proteins. Nanocarriers currently in use for the delivery of drugs and genetic materials can be generally divided to two categories: those made from lipids and those made from synthetic or natural polymers. Although lipid-based carriers are generally regarded to be safe and efficient, they do not possess sufficient chemical flexibility to fit individual requirements in delivery. In this thesis, we have explored lipid-substitution of three different polymer-based nanocarriers as means to develop optimum structures for drug as well as siRNA delivery. For the delivery of a potent amphiphilic antifungal drug, amphotericin B (AmB), lipid modification (particularly cholesteryl modification) of the core structure in poly(ethylene oxide)-poly(caprolactone) micelles was proved to be efficient in enhancing the solubility while reducing the hemolytic activity of encapsulated AmB. On the other hand, lipid modification of low molecular weight (2 kDa) polyethyleneimine (PEI2) has enhanced the properties of the nanocarriers in the delivery of STAT3-siRNA in wild type and resistant breast cancer cell models leading to an improved anti-cancer efficacy in combination with chemotherapeutic drugs, i.e. DOX and PTX. Finally, cholesteryl modification of poly(ethylene oxide)-poly(caprolactone-g-spermine) enhanced the properties of these nanocarriers for in vivo delivery of siRNA. This modification enhanced the stability, safety and cellular uptake of complexed siRNA leading to better silencing activity at the mRNA level. Overall, our results pointed to the positive impact of lipid modification in enhancing the properties of polymeric nanocarriers leading to viable formulations for effective delivery of AmB and siRNA therapeutics.
Download or read book Therapeutic Applications of RNAi written by John F. Reidhaar-Olson and published by Humana Press. This book was released on 2009-07-09 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book RNA Therapeutics written by Paloma H. Giangrande and published by Academic Press. This book was released on 2022-04-10 with total page 474 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Antisense Drug Technology written by Stanley T. Crooke and published by CRC Press. This book was released on 2007-07-25 with total page 848 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Silk based Drug Delivery Systems written by Elia Bari and published by Royal Society of Chemistry. This book was released on 2020-10-07 with total page 243 pages. Available in PDF, EPUB and Kindle. Book excerpt: Covering spider silk and silk worm cocoons, the editors elucidate the extraction, structure and properties of silk sericin and silk fibroin.
Download or read book Engineering Delivery Vehicles for Sirna Therapeutics written by Daniel Burton Vocelle and published by . This book was released on 2020 with total page 125 pages. Available in PDF, EPUB and Kindle. Book excerpt: Small molecule and protein-based drugs, while critically important therapies, cannot treat all diseases. As such, alternative treatment modalities must be developed to complement existing strategies. One potential alternative is small interfering RNA (siRNA) therapeutics, which are capable of specific inhibition of a wide range of intracellular, membrane, and extracellular proteins. siRNAs are hydrophilic due to their anionic backbone and do not readily diffuse across cellular membranes. During systemic delivery, naked siRNAs are rapidly filtered by the kidneys or degraded by serum nucleases and can often initiate an immune response. Thus, for siRNAs to be useful as therapeutics, they must be complexed with delivery vehicles for protection during extracellular transport and cellular internalization. Once delivered to the cytoplasm, siRNAs act through RNA interference (RNAi) to degrade messenger RNAs (mRNAs) in a sequence-specific manner, thereby reducing target protein expression. Despite the recent clinical success, development of siRNA therapeutics is limited due to the inefficiency, toxicity, and immunogenicity of current delivery vehicles. To overcome these hurdles, this research aimed to understand the role of delivery vehicle characteristics in influencing the cellular uptake and processing of siRNA-containing complexes. While many types of delivery vehicles have been developed for siRNAs, the characteristics that are essential for success are still not well understood. To address this issue, we synthesized a variety of silica nanoparticles (sNPs), and assessed their ability to effectively deliver siRNAs to human lung carcinoma cells (H1299). By varying the concentration of amines and dextran during sNP synthesis, we defined chemical/physical characteristics important for active siRNA delivery. Another roadblock in the development of siRNA therapeutics is a limited understanding of the intracellular processing of siRNA-containing complexes leading to initiation of RNAi. With recent evidence showing that the intracellular fate of endocytosed material was influenced by the endocytic pathway used for internalization, we developed a novel assay capable of differentiating uptake among the different endocytic pathways and assessing their functionality in initiating RNAi. Our results showed that Lipofectamine 2000 (LF2K) was internalized by Graf1-, Arf6-, or flotillin-mediated endocytosis for the initiation of RNAi, depending on cell type. Additionally, our study identified functional differences among endocytic pathways in a cell, indicating that uptake alone was not sufficient to initiate silencing. In a mixed cell population, we found that targeted inhibition of the non-functional pathways in some cells enhanced silencing in the uninhibited cells. These findings suggest that designing delivery vehicles for specific endocytic pathways may enhance the activity of the delivered siRNAs by directing them preferentially to the intended target cells.Finally, due to the limitations of current techniques, the intracellular pathways used in processing siRNA-containing complexes are not well defined. As a result, it is unclear how delivery vehicle characteristics affect the intracellular trafficking of siRNAs. To address this issue, we developed a novel microscopy-based assay that uses automated multi-well live-cell imaging to track the intracellular location of siRNAs over time. Through this assay we determined the intracellular pathways utilized in sNP-mediated siRNA delivery and identified how dextran functionalization of sNPs altered the intracellular trafficking of siRNAs. This assay provides a new analytical technique to assess intracellular pathways and could aid in the development of more efficient siRNA delivery vehicles.
Download or read book Molecular Regulation of Endocytosis written by Brian Ceresa and published by BoD – Books on Demand. This book was released on 2012-07-06 with total page 470 pages. Available in PDF, EPUB and Kindle. Book excerpt: Molecular Regulation of Endocytosis is a compilation of scientific "short stories" about the entry of external substances into cells. As one can see from the chapters, endocytosis regulates diverse processes such as homeostasis of the cell, signal transduction, entry of pathogens and viruses. In addition to the experimental techniques embedded in each chapter, entire chapters are dedicated to experimental approaches that will be useful to all scientists and their model systems. For those more clinically oriented, the final chapters look to the future and ways of utilizing endocytic pathways for therapeutic purposes.
