Commercialization of Oligonucleotides as Therapeutic Agents

Commercialization of Oligonucleotides as Therapeutic Agents PDF Author: Brian Minter
Publisher:
ISBN:
Category : Antisense nucleic acids
Languages : en
Pages : 94

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Book Description


Oligonucleotides as Therapeutic Agents

Oligonucleotides as Therapeutic Agents PDF Author: Derek J. Chadwick
Publisher: John Wiley & Sons
ISBN: 0470515406
Category : Science
Languages : en
Pages : 260

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Book Description
The use of oligonucleotides as therapeutic agents rests upon their ability to interfere, in a sequence-specific manner, with the fundamental machinery of protein synthesis either by binding to the mRNAs transcribed from a gene or by binding directly to a target gene. This approach can be used not only for inhibition of the synthesis of host proteins but also of those required by invading pathogens. Potential therapeutic applications are enormous, ranging over hypertension, cardiovascular disease, autoimmune disease, vital and other parasitic infections (especially HIV), and cancer. This book discusses the chemistry and pharmacokinetics of oligonucleotides and their analogues, and surveys the results of structure-activity studies and current clinical trials. It also critically reviews the problems with antisense therapy, such as the enzymatic destruction of oligonucleotides, the doses required for a therapeutic response, the difficulty in directing oligonucleotides to particular target tissues and cells, the need for parenteral administration, and doubts concerning the mechanism of action (especially problems associated with non-specific binding to proteins) and long-term effects.

Oligonucleotide-Based Drugs and Therapeutics

Oligonucleotide-Based Drugs and Therapeutics PDF Author: Nicolay Ferrari
Publisher: John Wiley & Sons
ISBN: 1119070295
Category : Medical
Languages : en
Pages : 576

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Book Description
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.

Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors

Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors PDF Author: Eric Wickstrom
Publisher: CRC Press
ISBN: 1000145956
Category : Medical
Languages : en
Pages : 452

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Book Description
An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors furnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.

Antisense Drug Technology

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

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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

Antisense Therapeutics

Antisense Therapeutics PDF Author: Sudhir Agrawal
Publisher:
ISBN: 9781592595853
Category : Antisense nucleic acids
Languages : en
Pages : 276

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Book Description
Annotation Dr. Agrawal's newest book is of significant importance since oligonucleotide-based drugs have the advantage of retaining their recognition for the target RNA, and provide the tools needed to modulate the expression of specific genes, making Antisense Therapeutics an essential resource for all antisense researchers.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products PDF Author: Maria Cristina Galli
Publisher: Springer
ISBN: 3319186183
Category : Medical
Languages : en
Pages : 235

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Book Description
This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

High Density Lipoproteins

High Density Lipoproteins PDF Author: Arnold von Eckardstein
Publisher: Springer
ISBN: 9783319096643
Category : Medical
Languages : en
Pages : 0

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Book Description
In this Handbook of Experimental Pharmacology on “High Density Lipoproteins – from biological understanding to clinical exploitation” contributing authors (members of COST Action BM0904/HDLnet) summarize in more than 20 chapters our current knowledge on the structure, function, metabolism and regulation of HDL in health and several diseases as well as the status of past and ongoing attempts of therapeutic exploitation. The book is of interest to researchers in academia and industry focusing on lipoprotein metabolism, cardiovascular diseases and immunology as well as clinical pharmacologists, cardiologists, diabetologists, nephrologists and other clinicians interested in metabolic or inflammatory diseases.

Commercialization of Nanotechnologies–A Case Study Approach

Commercialization of Nanotechnologies–A Case Study Approach PDF Author: Dermot Brabazon
Publisher: Springer
ISBN: 3319569791
Category : Technology & Engineering
Languages : en
Pages : 317

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Book Description
This book covers diverse areas in which nanoscience and nanotechnology have led to significant technological advances and practical applications, with special emphasis on novel types of nanomaterials and their applicability into a new generation of nano- and micro-devices. Different nanomaterials are reviewed with a focus on several practical application areas and their commercial utilization. Production technologies of nanomaterials are presented as one of the challenges today. Sectors where nanotechnology has already significantly contributed are presented, along with specific nanotechnology solutions: energy related sectors, NEMS/MEMS, micro power generators, spintronics and healthcare. The basic properties and applications of nanostructured thermoelectric materials, ferroelectric and piezoelectric nanomaterials are reviewed. Examples of several developed thin-film thermogenerators are shown. A review of existing solutions and developing challenges are given regarding sustainable energy production, photovoltaics, solar cells, hydrogen economy and improved classes of batteries as contributions to green products and circular economy. Novel, highly promising areas in nanotechnology, are shown, such as voltage-driven nano-spintronics. Recent advances in friction characterisation at the nano level are described. Several proven nanomaterials have been reviewed pertaining to biomedicine. The use of nanomaterials in ophthalmology and cosmetic industry are reviewed, and the potential for silver nanoparticles and iron-based nanomaterials in biomedicine, also with recognised challenges and possible threats of non-controlled use of nanomaterials. This work is the result of joint efforts of different companies, academic, and research institutions participating in WIMB Tempus project, 543898-TEMPUS-1-2013-1-ES-TEMPUS-JPHES, "Development of Sustainable Interrelations between Education, Research and Innovation at WBC Universities in Nanotechnologies and Advanced Materials where Innovation Means Business", co-funded by the Tempus Programme of the European Union.

Nuclear Medicine and Immunology

Nuclear Medicine and Immunology PDF Author: Sara Harsini
Publisher: Springer Nature
ISBN: 3030812618
Category : Medical
Languages : en
Pages : 532

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Book Description
This book explores the close connection between immunology and nuclear medicine, which has led to radioimmunoimaging and radioimmunotherapy (RIT). Molecular imaging with positron emission tomography (PET) and single-photon emission computed tomography (SPECT) is increasingly being used to diagnose, characterize, and monitor disease activity in the context of inflammatory disorders of known and unknown etiology, such as sarcoidosis, atherosclerosis, vasculitis, inflammatory bowel disease, rheumatoid arthritis, and degenerative joint disease. The first chapters discuss the various radiopharmaceutical agents and radiolabeled preparations that have been employed in inflammation imaging. Of these, FDG-PET imaging has been shown to have the great value in the detection of inflammation and has become the centerpiece of several initiatives over the last several years. This very powerful technique will play an increasingly important role in the management of patients with inflammatory conditions in the future. The book also explores the growing role of nuclear medicine and molecular imaging in the diagnosis and treatment of cancer. The rapid pace of change has been fueled by advances in our understanding of tumor biology, on the one hand, and the development of specifically targeted medical therapies, diagnostic agents, and radiotherapies, on the other. Written by leading international experts in the field, this book is an invaluable tool for nuclear medicine physicians, radiologists, oncologists, and immunologists.