Pharmaceutical Perspectives Of Nucleic Acid Based Therapy
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Pharmaceutical Perspectives of Nucleic Acid-Based Therapy by Ram I. Mahato,Sung Wan Kim Pdf
Emphasizing its uses in cancer and cardiovascular and autoimmune diseases, Pharmaceutical Perspectives of Nucleic Acid-Based Therapy presents a comprehensive account of gene therapy, from development in the laboratory to clinical applications. Internationally acclaimed scientists discuss the potential use of lipids, peptides and polymers for the in
Oligonucleotide-Based Drugs and Therapeutics by Nicolay Ferrari,Rosanne Seguin Pdf
A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.
Pharmaceutical Perspectives of Cancer Therapeutics by Yi Lu,Ram I. Mahato Pdf
Pharmaceutical Perspectives of Cancer Therapeutics covers a wide variety of therapeutic approaches including gene therapy, immunological therapy; cancer vaccines; strategy for solid tumors as well as for hematological cancers; methods to suppress tumor angiogenesis and metastasis; development and utilization of relevant animal models; introduction of new concepts such as cancer stem cells and new technologies, such as DNA and tissue microarrays; and RNA interference. In addition, clinical application, the development of DNA diagnosis biomarkers and cancer prevention, as well as the utilization of imaging in cancer therapy are also discussed. The use of synthetic carriers, such as lipids, polymers, and peptides for delivery and targeting of small molecules, proteins, and nucleic acids to cancer cells in vivo are discussed. Pharmaceutical Perspectives of Cancer Therapeutics also includes cancer therapy modality in surgery, chemotherapy, and radiotherapy, as well as in combination or multi-modality, giving our book a more focused view of cancer therapy.
In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders by Houria Bachtarzi Pdf
Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis.
Nucleic Acids as Gene Anticancer Drug Delivery Therapy by Loutfy H. Madkour Pdf
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
Modern Avenues in Metal-Nucleic Acid Chemistry by Jens Müller,Bernhard Lippert Pdf
Volume 25, entitled Modern Avenues in Metal-Nucleic Acid Chemistry, of the series Metal Ions in Life Sciences (MILS), provides a timely overview of selected relevant developments in a field which previously had a pronounced focus on fundamental chemistry aspects such as coordination principles, structural chemistry, and consequences for biological processes and disease-related medicinal issues. More recently, also as a consequence of the advent of supramolecular chemistry, advancements in nanochemistry, and new bio-analytical methods, novel aspects of this field have emerged. MILS-25 gives a state-of-the-art account on the present state of research in this field, addressing, among others, porous materials, non-covalent interactions, stimuli-responsive bioconjugates, DNA-templated metal arrays and nanoclusters, genotyping, guanine quadruplexes, and riboswitches. It consists of 15 comprehensive chapters with over 1600 citations, 160 figures (90 of them in color), with key contributions from 40 international experts from Europe, Asia, and America. It is an essential resource for scientists from numerous disciplines, ranging from materials science to biochemistry, by way of classical chemistry and physics. • It offers an update on nucleic acid – metal complex bioconjugates and their applications • It provides an insight into coordination polymers involving nucleic acid components • It covers a wide range of nucleic acids, from DNA duplexes, triplexes and quadruplexes to RNA
New Antisense Strategies: Chemical Synthesis of RNA Oligomers, by Junichi Yano und Gerald E. Smyth Development and Modification of Decoy Oligodeoxynucleotides for Clinical Application, by Mariana Kiomy Osako, Hironori Nakagami und Ryuichi Morishita Modulation of Endosomal Toll-Like Receptor-Mediated Immune Responses by Synthetic Oligonucleotides, by Ekambar R. Kandimalla und Sudhir Agrawal Delivery of Nucleic Acid Drugs, by Yan Lee und Kazunori Kataoka Aptamer: Biology to Applications, by Yoshikazu Nakamura Development and Clinical Applications of Nucleic Acid Therapeutics, by Veenu Aishwarya, Anna Kalota und Alan M. Gewirtz
Pharmaceutical Dosage Forms and Drug Delivery, Second Edition by Ram I. Mahato,Ajit S. Narang Pdf
In the second edition of Pharmaceutical Dosage Forms and Drug Delivery the authors integrate aspects of physical pharmacy, biopharmaceuticals, drug delivery, and biotechnology, emphasizing the increased attention that the recent spectacular advances in dosage form design and drug delivery, gene therapy, and nanotechnology have brought to the field. Highlights of the Second Edition: Additional author Ajit S. Narang brings an industrial practitioner perspective with increased focus on pharmacy math and statistics, and powders and granules Reorganized into three parts: Introduction, Physicochemical Principles, and Dosage Forms Chapters on pharmaceutical calculations, compounding principles, and powders and granules provide a complete spectrum of application of pharmaceutical principles Expansion of review questions and answers clarifies concepts for students and adds to their grasp of key concepts covered in the chapter Coverage of complexation and protein binding aspects of physical pharmacy includes the basic concepts as well as recent progress in the field Although there are numerous books on the science of pharmaceutics and dosage form design, most cover different areas of the discipline and do not provide an integrated approach to the topics. This book not only provides a singular perspective of the overall field, but it supplies a unified source of information for students, instructors, and professionals.
Nucleic Acid Transfection by Wolfgang Bielke,Christoph Erbacher Pdf
Gene Delivery into Mammalian Cells: An Overview on Existing Approaches Employed In Vitro and In Vivo, by Peter Hahn and Elizabeth Scanlan * Strategies for the Preparation of Synthetic Transfection Vectors, by Asier Unciti-Broceta, Matthew N. Bacon, and Mark Bradley * Cationic Lipids: Molecular Structure/Transfection Activity Relationships and Interactions with Biomembranes, by Rumiana Koynova and Boris Tenchov * Hyperbranched Polyamines for Transfection, by Wiebke Fischer, Marcelo Calderon, and Rainer Haag * Carbohydrate Polymers for Nonviral Nucleic Acid Delivery, by Antons Sizovs, Patrick M. McLendon, Sathya Srinivasachari, and Theresa M. Reineke * Cationic Liposome–Nucleic Acid Complexes for Gene Delivery and Silencing: Pathways and Mechanisms for Plasmid DNA and siRNA, by Kai K. Ewert, Alexandra Zidovska, Ayesha Ahmad, Nathan F. Bouxsein, Heather M. Evans, Christopher S. McAllister, Charles E. Samuel, and Cyrus R. Safinya * Chemically Programmed Polymers for Targeted DNA and siRNA Transfection, by Eveline Edith Salcher and Ernst Wagner * Photochemical Internalization: A New Tool for Gene and Oligonucleotide Delivery, by Kristian Berg, Maria Berstad, Lina Prasmickaite, Anette Weyergang, Pål K. Selbo, Ida Hedfors, and Anders Høgset * Visualizing Uptake and Intracellular Trafficking of Gene Carriers by Single-Particle Tracking, by N. Ruthardt and C. Bräuchle
DNA Damage and Double Strand Breaks Part B by Anonim Pdf
Intersection of Iron and Lipid Metabolism, Volume 52 in the Enzymes series, highlights new advances in the field, with this new volume presenting interesting chapters on a variety of timely topics relating to enzymes research. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in The Enzymes series
Pharmaceutical Nanobiotechnology for Targeted Therapy by Hamed Barabadi,Ebrahim Mostafavi,Muthupandian Saravanan Pdf
The field of nanotechnology for targeted therapy initiated more than decade ago has grown fast and interest is increasing. Given the importance of the field for targeted drug and gene delivery systems, there are a large number of laboratory investigations today researching nanobiomaterials for diagnostic and therapeutic applications. Because of the ability of scientists to load nanoparticles with any agent, interest continues to grow and technology in this arena is rapidly evolving. These emerging nanobiomaterials-based medicines can overcome the disadvantages of traditional medicines by target-oriented and site-specific delivery of precise medicines (immunotherapeutic agents, chemotherapeutic agents, diagnostic agents, and so on). Pharmaceutical Nanobiotechnology for Targeted Therapy presents an updated overview of recent advancements in the field of pharmaceutical nanobiotechnology and nano-based drug and gene delivery systems. This comprehensive knowledge will allow researchers to discover innovative nanobiomaterials for targeted therapeutics. The chapters deal with various emerging nanobiomaterials for targeted therapeutic delivery systems and the writing is in a style that is easily disseminated and in a manner that can be readily adopted as sources for new and further studies. This book should be useful for researchers and professionals from academia and industry working in the field of nanotechnology, nanobiotechnology, as well as in the field of pharmaceutical nanotechnology. It should also be useful to those interested in a range of disciplines from material science, chemistry, molecular biology, polymer chemistry, and many more interdisciplinary areas.
Nuclear, Chromosomal, and Genomic Architecture in Biology and Medicine by Malgorzata Kloc,Jacek Z. Kubiak Pdf
This volume reviews the latest research on the functional implications of nuclear, chromosomal and genomic organization and architecture on cell and organismal biology, and development and progression of diseases. The architecture of the cell nucleus and non-random arrangement of chromosomes, genes, and the non-membranous nuclear bodies in the three-dimensional (3D) space alters in response to the environmental, mechanical, chemical, and temporal cues. The changes in the nuclear, chromosomal, or genomic compaction and configuration modify the gene expression program and induce or inhibit epigenetic modifications. The intrinsically programmed rearrangements of the nuclear architecture are necessary for cell differentiation, the establishment of cell fate during development and maturation of tissues and organs including the immune, muscle, and nervous systems. The non-programmed changes in the nuclear architecture can lead to fragmentation of the nucleus and instability of the genome and thus cause cancer. Microbial and viral infections can lead to a clustering of centromeres, telomeres and ribosomal DNA and alter the properties of the nuclear membrane, allowing large immobile macromolecules to enter the nucleus. Recent advances in next-generation sequencing technologies combined with nucleus/chromosome conformation capture, super-resolution imaging, chromosomal contact maps methods, integrative modeling, and genetic approaches, are uncovering novel features and importance of nuclear, chromosomal and genomic architecture. This book is an interesting read for cell biologists, researchers studying the structure and function of chromosomes, and anyone else who wants to get an overview of the field of nuclear, chromosomal and genomic architecture.
Minicircle and Miniplasmid DNA Vectors by Martin Schleef Pdf
This first title on the topic provides complete coverage, including the molecular basis, production and possible biomedical applications. Written by the most prominent academic researchers in the field as well as by researchers at one of the world's leading companies in industrial production of minicircle DNA, this practical book is aimed at everyone who is directly or indirectly involved in the development of gene therapies.
DNA Interactions with Polymers and Surfactants by Rita Dias,Bjorn Lindman Pdf
A broad overview of the interaction of DNA with surfactants and polymers Due to the potential benefits of biotechnology, interest in the interaction between DNA and surfactants and polymers has become increasingly significant. Now, DNA Interactions with Polymers and Surfactants provides an extensive, up-to-date overview of the subject, giving readers a basis for understanding the factors leading to complexation between DNA and different cosolutes, including metal ions, polyelectrolytes, spermine, spermidine, surfactants and lipids, and proteins. Topical coverage includes: Polyelectrolytes, physico-chemical aspects and biological significance Solution behavior of nucleic acids Single DNA molecules: compaction and decompaction Interaction of DNA with surfactants and cationic polymers Interactions of histones with DNA DNA-DNA interactions The hydration of DNA-amphiphile complexes DNA-surfactant/lipid complexes at liquid interfaces DNA and DNA-surfactant complexes at solid surfaces The role of correlation forces for DNA-cosolute interactions Simulations of polyions Cross-linked DNA gels and gel particles DNA as an amphiphilic polymer Lipid-DNA interactions Covering both theoretical and practical aspects of the subject, DNA Interactions with Polymers and Surfactants is an ideal resource for chemists and biochemists working in gene and DNA delivery research in industry and academia, as well as for cell biologists, chemical engineers, molecular biologists, and development biologists in the pharmaceutical industry.