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Author: Yashwant Pathak
Publisher: CRC Press
ISBN: 1000580296
Category : Medical
Languages : en
Pages : 317
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Book Description
This unique volume in our Drugs and Pharmaceutical Sciences series covers the development of gene therapy today, the technology involved, clinical applications of siRNA, non-viral vector-based mRNA delivery using nanotechnology, and RNA based vaccines for treating the infectious diseases. It also presents the current application of the CRISPR/Cas9 gene-editing technique which has revolutionized genome editing and which was awarded the 2020 Nobel Prize in Chemistry. Several new drug delivery systems are explored for the applications of gene therapy. These are found to be useful in treating chronic illnesses, including cancer and infectious diseases. Key Features: Overview of the development of gene therapy Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing Presents CRISPR gene therapy recent trends and applications Discusses siRNA, mRNA, and DNA plasmids
Author: Yashwant Pathak
Publisher: CRC Press
ISBN: 1000580296
Category : Medical
Languages : en
Pages : 317
Get Book Here
Book Description
This unique volume in our Drugs and Pharmaceutical Sciences series covers the development of gene therapy today, the technology involved, clinical applications of siRNA, non-viral vector-based mRNA delivery using nanotechnology, and RNA based vaccines for treating the infectious diseases. It also presents the current application of the CRISPR/Cas9 gene-editing technique which has revolutionized genome editing and which was awarded the 2020 Nobel Prize in Chemistry. Several new drug delivery systems are explored for the applications of gene therapy. These are found to be useful in treating chronic illnesses, including cancer and infectious diseases. Key Features: Overview of the development of gene therapy Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing Presents CRISPR gene therapy recent trends and applications Discusses siRNA, mRNA, and DNA plasmids
Author: Ravin Narain
Publisher: Woodhead Publishing
ISBN: 0081005210
Category : Science
Languages : en
Pages : 303
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Book Description
Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. - Explores current challenges in the research of genetic diseases - Discusses polymers for gene therapy and their function in designing advanced materials - Provides examples of organic and inorganic nanomaterials for gene therapy - Includes labeling, targeting, and assays - Looks at characterization, physico-(bio)chemical properties, and applications
Author: David T. Curiel
Publisher: Academic Press
ISBN: 0128005106
Category : Science
Languages : en
Pages : 870
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Book Description
Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. - Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors - Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement - Demonstrates noninvasive imaging of adenovirus-mediated gene transfer - Discusses utility of adenoviral vectors in animal disease models - Considers Federal Drug Administration regulations for human clinical trials
Author: Ali Demir Sezer
Publisher: BoD – Books on Demand
ISBN: 9535108107
Category : Medical
Languages : en
Pages : 516
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Book Description
This contribution book collects reviews and original articles from eminent experts working in the interdisciplinary arena of novel drug delivery systems and their uses. From their direct and recent experience, the readers can achieve a wide vision on the new and ongoing potentialities of different drug delivery systems. Since the advent of analytical techniques and capabilities to measure particle sizes in nanometer ranges, there has been tremendous interest in the use of nanoparticles for more efficient methods of drug delivery. On the other hand, this reference discusses advances in the design, optimization, and adaptation of gene delivery systems for the treatment of cancer, cardiovascular, pulmonary, genetic, and infectious diseases, and considers assessment and review procedures involved in the development of gene-based pharmaceuticals.
Author: Inamuddin
Publisher: John Wiley & Sons
ISBN: 1119654866
Category : Technology & Engineering
Languages : en
Pages : 544
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Book Description
The aim of this book is to explore the history, fundamentals, manufacturing processes, optimization parameters, and applications of electrospun materials. The book includes various types of electrospun materials such as antimicrobial, smart, bioinspired systems. It focuses on the many application areas for electrospun materials such as energy storage and harvesting, catalysis, biomedical including gene delivery and tissue engineering, separation, adsorption and water treatment technologies, packaging. The book emphasizes the enhanced sustainable properties of electrospun materials, with the challenges and future developments being discussed in detail. The chapters are written by top-class researchers and experts from throughout the world.
Author: Roland W. Herzog
Publisher: World Scientific
ISBN: 9814280917
Category : Medical
Languages : en
Pages : 415
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Book Description
1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309495849
Category : Medical
Languages : en
Pages : 93
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Book Description
On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.
Author: Vanessa Zimmer
Publisher:
ISBN: 9781536162684
Category : Gene therapy
Languages : en
Pages : 0
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Book Description
Gene Delivery: Methods and Applications provides a comprehensive overview on viral and non-viral methods used to genetically engineer human mesenchymal stromal cells. In addition, an update on ongoing and completed clinical studies with engineered mesenchymal stromal cells will be provided, as well as a snapshot of the advances and technical challenges yet to be addressed.Next, a variety of gene delivery systems including physical transfection techniques, virus-based delivery vectors, chemically engineered delivery systems and bio-inspired vehicles are reviewed and their strengths, shortcomings and biomedical applications are discussed.Selfish DNA called transposons capable of cutting out and pasting into the host genome are active throughout the phylogenetic kingdoms. Researchers have repurposed natural transposons for use in delivering a gene-of-interest, enabling for the study of a large and growing list of preclinical gene therapy applications. As such, the authors discuss the past achievements and future challenges of this early-stage technology.The closing chapter introduces cell-penetrating peptides as an efficient tool for DNA transfection. HR9, a designed cell-penetrating peptides, containing nona-arginine flanked by cysteine and penta-histidine displayed a high penetrating ability in mammalian cells.
Author: Rajesh K. Kesharwani
Publisher: CRC Press
ISBN: 1000290557
Category : Medical
Languages : en
Pages : 261
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Book Description
Biotechnology in the Modern Medicinal System: Advances in Gene Therapy, Immunotherapy, and Targeted Drug Delivery presents an informative picture of the state-of-the-art research and development of actionable knowledge in medical biotechnology, specifically involving gene therapy, immunotherapy, and targeted drug delivery systems. The book includes novel approaches for therapy of various ailments and the real-world challenges and complexities of the current drug delivery methodologies and techniques. The volume helps to bridge the gap between academic research and real-time clinical applications and the needs of medical biotechnology methods. This edited book also provides a detailed application of medical biotechnology in drug discovery and the treatment of various deadly diseases. Chapters discuss targeted drug delivery to specific sites to avoid possible entry to non-targeted sites, minimizing adverse effects. The volume provides information about the roles of alternative routes of drug targeting, like intranasal and transdermal, resulting in improving patient compliance. Targeted drug delivery is explored for several health issues, such as neurodegenerative disorders, cancer, malaria, and hemoglobin disorders. Also considered is the role of genes in various genetic diseases and gene therapy, and immunogene therapy as alternative approaches to conventional cancer therapy. Finally, the book investigates the important role of computers in biotechnology to accelerate research and development in the modern medicinal field for better and optimum results. Studies show that significant improvement has been observed in the development of a faster and less invasive diagnostic system for the treatment of diseases by utilizing both artificial intelligence (AI) and biotechnology. This valuable volume provides a wealth of information that will be valuable to scientists and researchers, faculty, and students.
Author: Manzoor M. Khan
Publisher: Springer Science & Business Media
ISBN: 0387779760
Category : Medical
Languages : en
Pages : 275
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Book Description
During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.
