Gene Therapeutics
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Gene Therapeutics
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Author : Jon A. Wolff
language : en
Publisher: Springer Science & Business Media
Release Date : 2012-12-06
Gene Therapeutics written by Jon A. Wolff and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-12-06 with Medical categories.
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.
Somatic Gene Therapy
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Author : P.L. Chang
language : en
Publisher: CRC Press
Release Date : 2018-01-17
Somatic Gene Therapy written by P.L. Chang and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-01-17 with Science categories.
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.
Manufacturing Of Gene Therapeutics
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Author : G. Subramanian
language : en
Publisher: Springer Science & Business Media
Release Date : 2012-12-06
Manufacturing Of Gene Therapeutics written by G. Subramanian and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-12-06 with Medical categories.
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
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Author : Joseph Panno
language : en
Publisher: Infobase Publishing
Release Date : 2005
Gene Therapy written by Joseph Panno and has been published by Infobase Publishing this book supported file pdf, txt, epub, kindle and other format this book has been release on 2005 with Biology categories.
Many diseases are caused by a simple point mutation.
Gene Therapy Of Cancer
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Author : Stanton L. Gerson
language : en
Publisher: Elsevier
Release Date : 2002-04-04
Gene Therapy Of Cancer written by Stanton L. Gerson and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2002-04-04 with Science categories.
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. 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
An Introduction To Molecular Medicine And Gene Therapy
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Author : Thomas F. Kresina
language : en
Publisher: John Wiley & Sons
Release Date : 2004-03-24
An Introduction To Molecular Medicine And Gene Therapy written by Thomas F. Kresina and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2004-03-24 with Science categories.
An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections - an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy: * Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations * Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases * Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter * Contains appendices on commercial implications and a review of the history of gene therapy This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine.
Gene And Cell Therapies
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Author : Eve Hanna
language : en
Publisher: CRC Press
Release Date : 2020-05-19
Gene And Cell Therapies written by Eve Hanna and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-05-19 with Medical categories.
The major advances in the field of biotechnology and molecular biology in the twenty-first century have led to a better understanding of the pathophysiology of diseases. A new generation of biopharmaceuticals has emerged, including a wide and heterogeneous range of innovative cell and gene therapies. These therapies aim to prevent or treat chronic and serious life-threatening diseases, previously considered incurable. This book describes the evolution and adaptation of the regulatory environment to assess these therapies in contrast with the resistance of health technology assessment (HTA) agencies and payers to acknowledge the specificity of cell and gene therapies and the need to adapt existing decision-making frameworks. This book provides insights on the learnings from the experience of current cell and gene therapies (regulatory approval, HTA, and market access), in addition to future trends to enhance patient access to these therapies. Key Features: Describes the potential change of treatment paradigm and the specificity of cell and gene therapies, including the gradual move from repeated treatment administration to one-time single administration with the potential to be definite cure Highlights the challenges at the HTA level Discusses the affordability of future cell and gene therapies and the possible challenges for health insurance systems Provides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of healthcare systems
Gene Therapeutics
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Author : Jon Asher Wolff
language : en
Publisher:
Release Date : 1994
Gene Therapeutics written by Jon Asher Wolff and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1994 with Gene therapy categories.
Development Of Gene Therapies
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Author : Avery McIntosh
language : en
Publisher: Chapman & Hall/CRC Biostatistics Series
Release Date : 2024-03-13
Development Of Gene Therapies written by Avery McIntosh and has been published by Chapman & Hall/CRC Biostatistics Series this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-03-13 with categories.
Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.
Gene Based Therapies For Cancer
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Author : Jack A. Roth
language : en
Publisher: Springer Science & Business Media
Release Date : 2010-07-28
Gene Based Therapies For Cancer written by Jack A. Roth and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010-07-28 with Medical categories.
Cancer gene therapy is a novel therapy that targets the underlying genetic defects in the cancer cell. Progress in this field has been rapid and gene therapy promises to further extend personalized cancer treatment. In this volume leading experts have contributed their experience in developing gene therapies for a variety of cancers. Translational gene therapy approaches are emphasized. Chapters include discussions of specific gene delivery technologies as well as their application to various cancers with extensive discussions of ongoing clinical trials. This information should be useful to both students, fellows, and experienced scientists with an interest in this rapidly developing area.