Gene Therapeutics

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Gene Therapy of Cancer

Stanton L. Gerson,Edmund C. Lattime
Gene Therapy of Cancer Book PDF

Author : Stanton L. Gerson,Edmund C. Lattime
Publisher : Elsevier
Page : 534 pages
File Size : 46,5 Mb
Release : 2002-04-04
Category : Science
ISBN : 0080491367

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Gene Therapy of Cancer by Stanton L. Gerson,Edmund C. Lattime Pdf

The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

Translating Gene Therapy to the Clinic

Jeffrey Laurence,Michael Franklin
Translating Gene Therapy to the Clinic Book PDF

Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Page : 346 pages
File Size : 41,7 Mb
Release : 2014-11-14
Category : Science
ISBN : 9780128005644

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Translating Gene Therapy to the Clinic by Jeffrey Laurence,Michael Franklin Pdf

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

Fast Facts: Gene Therapy

R. Herzog,L. Popplewell
Fast Facts Gene Therapy Book PDF

Author : R. Herzog,L. Popplewell
Publisher : Karger Medical and Scientific Publishers
Page : 102 pages
File Size : 50,8 Mb
Release : 2020-04-24
Category : Medical
ISBN : 9783318066678

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Fast Facts: Gene Therapy by R. Herzog,L. Popplewell Pdf

Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Gene Therapy

Nancy Smyth Templeton,D. D. Lasic
Gene Therapy Book PDF

Author : Nancy Smyth Templeton,D. D. Lasic
Publisher : CRC Press
Page : 616 pages
File Size : 42,6 Mb
Release : 2000-06-15
Category : Medical
ISBN : UOM:39015050321036

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Gene Therapy by Nancy Smyth Templeton,D. D. Lasic Pdf

This text covers all aspects of gene therapy, including basic principles, viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents.

Gene and Cell Therapy: Biology and Applications

Giridhara R. Jayandharan
Gene and Cell Therapy Biology and Applications Book PDF

Author : Giridhara R. Jayandharan
Publisher : Springer
Page : 305 pages
File Size : 51,5 Mb
Release : 2018-09-12
Category : Medical
ISBN : 9789811304811

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Gene and Cell Therapy: Biology and Applications by Giridhara R. Jayandharan Pdf

Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics.

Gene Therapeutics

Jon A. Wolff
Gene Therapeutics Book PDF

Author : Jon A. Wolff
Publisher : Springer Science & Business Media
Page : 431 pages
File Size : 45,5 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781468468229

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Gene Therapeutics by Jon A. Wolff Pdf

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.

Stem Cell and Gene Therapy for Cardiovascular Disease

Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Stem Cell and Gene Therapy for Cardiovascular Disease Book PDF

Author : Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Publisher : Academic Press
Page : 538 pages
File Size : 49,7 Mb
Release : 2015-08-21
Category : Science
ISBN : 9780128018637

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Stem Cell and Gene Therapy for Cardiovascular Disease by Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson Pdf

Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI Covers the prevailing opinions on how to harness the body’s natural repair mechanisms Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Maria Cristina Galli
Regulatory Aspects of Gene Therapy and Cell Therapy Products Book PDF

Author : Maria Cristina Galli
Publisher : Springer Nature
Page : 243 pages
File Size : 52,9 Mb
Release : 2023-08-01
Category : Science
ISBN : 9783031345678

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Regulatory Aspects of Gene Therapy and Cell Therapy Products by Maria Cristina Galli Pdf

This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. 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 and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, 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 & 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.

Adenoviral Vectors for Gene Therapy

David T. Curiel
Adenoviral Vectors for Gene Therapy Book PDF

Author : David T. Curiel
Publisher : Academic Press
Page : 868 pages
File Size : 44,7 Mb
Release : 2016-03-10
Category : Science
ISBN : 9780128005101

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Adenoviral Vectors for Gene Therapy by David T. Curiel Pdf

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

Manufacturing of Gene Therapeutics

G. Subramanian
Manufacturing of Gene Therapeutics Book PDF

Author : G. Subramanian
Publisher : Springer Science & Business Media
Page : 355 pages
File Size : 51,7 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781461513537

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Manufacturing of Gene Therapeutics by G. Subramanian Pdf

Advances in molecular biology and recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches and methods are readily available for gene cloning and therapeutic vector construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus gene therapy has already shown that it holds great promise for the treatment of many diseases and disorders. In general it involves the delivery of recombinant genes or transgenes into somatic cells to replace proteins with a genetic defect or to transfer with the pathological process of an illness. The viral and non-viral delivery systems may hold the potential for future non-invasive, cost-effective oral therapy of genetically-based disorders. Recent years have seen considerable progress in the discovery and early clinical development of a variety of gene therapeutic products. The availability, validation, and implementation of gene therapeutic products has also enabled success in testing and evaluation. New challenges will need to be overcome to ensure that products will also be successful in later clinical development and ultimately for marketing authorisation. These new challenges will include improvements in delivery systems, better control of in-vivo targeting, increased level transduction and duration of expression of the gene, and manufacturing process efficiencies that enable reduction in production costs. Perhaps profound understanding of regulated gene design may result in innovative bioproducts exhibiting safety and efficacy profiles that are significantly superior to those achieved by the use of naturally occurring genes. This procedure may contribute considerably to fulfilling standards set by regulatory authorities. This book provides an overview of the current advances in the field of gene therapy and the methods that are being successfully applied in the manufacture of gene therapeutic products, and hopefully will stimulate further progress and advancement in this field to meet the ever-increasing demands.

Gene Therapy

Joseph Panno
Gene Therapy Book PDF

Author : Joseph Panno
Publisher : Infobase Publishing
Page : 193 pages
File Size : 42,5 Mb
Release : 2005
Category : Biology
ISBN : 9780816067343

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Gene Therapy by Joseph Panno Pdf

Many diseases are caused by a simple point mutation.

Gene Therapy for Cancer

Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Gene Therapy for Cancer Book PDF

Author : Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Publisher : Springer Science & Business Media
Page : 469 pages
File Size : 40,7 Mb
Release : 2007-10-26
Category : Medical
ISBN : 9781597452229

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Gene Therapy for Cancer by Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher Pdf

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.

Gene and Cell Therapy

Nancy Smyth Templeton
Gene and Cell Therapy Book PDF

Author : Nancy Smyth Templeton
Publisher : CRC Press
Page : 894 pages
File Size : 45,6 Mb
Release : 2003-12-17
Category : Science
ISBN : 9780824758608

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Gene and Cell Therapy by Nancy Smyth Templeton Pdf

This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field.

Somatic Gene Therapy

P.L. Chang
Somatic Gene Therapy Book PDF

Author : P.L. Chang
Publisher : CRC Press
Page : 313 pages
File Size : 50,6 Mb
Release : 2018-01-17
Category : Science
ISBN : 9781351085212

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Somatic Gene Therapy by P.L. Chang Pdf

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.

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Nicola Brunetti-Pierri
Safety and Efficacy of Gene Based Therapeutics for Inherited Disorders Book PDF

Author : Nicola Brunetti-Pierri
Publisher : Springer
Page : 220 pages
File Size : 53,7 Mb
Release : 2017-05-22
Category : Medical
ISBN : 9783319534572

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Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders by Nicola Brunetti-Pierri Pdf

In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.