Download or read book Viral Vectors for Gene Therapy written by Fredric P. Manfredsson and published by . This book was released on 2019 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. 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. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.
Download or read book Viral Vectors for Gene Therapy written by Otto-Wilhelm Merten and published by Humana. This book was released on 2011-05-19 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.
Download or read book Gene Therapy Methods written by M. Ian Phillips and published by Academic Press. This book was released on 2002-02-22 with total page 770 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease
Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 868 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Viral Vectors for Gene Therapy written by Curtis A. Machida and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 591 pages. Available in PDF, EPUB and Kindle. Book excerpt: Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors.
Download or read book Gene Therapy Protocols written by Paul D. Robbins and published by Humana PressInc. This book was released on 1997 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: Thirty protocols by leading researchers describe in detail all the essential molecular methods for working with gene transfer systems, along with the methods for gene transfer to specific tissue types either in vivo or ex vivo. The easily reproducible methods range from those for specific viral and nonviral for both genetic and acquired diseases, to those concerned with gene delivery to particular tissues. Methods for applying specific therapeutic systems, such as ribozymes and tumor supressor genes for the treatment of AIDS and cancer, are also included in this authoritative collection.
Download or read book Translating Gene Therapy to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2014-11-14 with total page 347 pages. Available in PDF, EPUB and Kindle. Book excerpt: Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials
Download or read book Gene Therapy for Cancer written by Kelly K. Hunt and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.
Download or read book Somatic Gene Therapy written by P.L. Chang and published by CRC Press. This book was released on 2018-01-17 with total page 313 pages. Available in PDF, EPUB and Kindle. Book excerpt: As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.
Download or read book Human Gene Therapy written by Eve K. Nichols and published by Harvard University Press. This book was released on 1988 with total page 268 pages. Available in PDF, EPUB and Kindle. Book excerpt: Nichols explores the potential for gene therapy and identifies those who are candidates for it. Having provided a biomedical background for understanding somatic cell gene therapy, she takes a thoughtful look at complex and sensitive issues surrounding ethical, economic, and policy aspects of manipulating human genes.
Download or read book Gene Therapy for Neurological Disorders written by Fredric P. Manfredsson and published by Humana. This book was released on 2015-11-26 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? 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 thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.
Download or read book Introduction to Basics of Pharmacology and Toxicology written by Gerard Marshall Raj and published by Springer Nature. This book was released on 2019-11-16 with total page 410 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.
Download or read book A Handbook of Gene and Cell Therapy written by Clévio Nóbrega and published by Springer Nature. This book was released on 2020-06-27 with total page 195 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field.
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Download or read book Gene Therapeutics written by Jon A. Wolff and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 431 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the first half century of genetics, coinciding with the first half of this cen tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex tremely efficient in transferring their contents to the chromosomes of cultured cells.
