Development Of Gene Therapies

Download Development Of Gene Therapies full books in PDF, epub, and Kindle. Read online Development Of Gene Therapies ebook anywhere anytime directly on your device. Fast Download speed and no annoying ads. We cannot guarantee that every ebooks is available!

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309672988
Total Pages : 127 pages
Book Rating : 4.3/5 (96 download)

DOWNLOAD NOW!

Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine

Download or read book Exploring Novel Clinical Trial Designs for Gene-Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Development of Gene Therapies

Author : Avery McIntosh
Publisher : Chapman & Hall/CRC Biostatistics Series
ISBN 13 : 9781032136554
Total Pages : 0 pages
Book Rating : 4.1/5 (365 download)

DOWNLOAD NOW!

Book Synopsis Development of Gene Therapies by : Avery McIntosh

Download or read book Development of Gene Therapies written by Avery McIntosh and published by Chapman & Hall/CRC Biostatistics Series. This book was released on 2024-03-13 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 Therapy of Cancer

Author : Stanton L. Gerson
Publisher : Elsevier
ISBN 13 : 0080491367
Total Pages : 555 pages
Book Rating : 4.0/5 (84 download)

DOWNLOAD NOW!

Book Synopsis Gene Therapy of Cancer by : Stanton L. Gerson

Download or read book Gene Therapy of Cancer written by Stanton L. Gerson and published by Elsevier. This book was released on 2002-04-04 with total page 555 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Gene Therapy for Cancer

Author : Kelly K. Hunt
Publisher : Springer Science & Business Media
ISBN 13 : 159745222X
Total Pages : 469 pages
Book Rating : 4.5/5 (974 download)

DOWNLOAD NOW!

Book Synopsis Gene Therapy for Cancer by : Kelly K. Hunt

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.

The Development of Human Gene Therapy

Author : Theodore Friedmann
Publisher : CSHL Press
ISBN 13 : 9780879695286
Total Pages : 748 pages
Book Rating : 4.6/5 (952 download)

DOWNLOAD NOW!

Book Synopsis The Development of Human Gene Therapy by : Theodore Friedmann

Download or read book The Development of Human Gene Therapy written by Theodore Friedmann and published by CSHL Press. This book was released on 1999 with total page 748 pages. Available in PDF, EPUB and Kindle. Book excerpt: The idea of human gene therapy was accepted by the medical community and society at large long before believable clinical benefits began to emerge. In this book, some of the fields most distinguished contributors chronicle the evolution of this momentous direction for medicine, illustrating how imaginative concepts shaped the development of technologies and brought the daring new idea to its current position of imminent practical success. This is a book designed to endure as clinical advances accumulate, a clear-eyed work of reference that will anchor the further development of this revolution in therapy. It is an essential addition to libraries of clinical medicine, biotechnology, and public policy, and a resource that no laboratory investigator with an interest in the biology of gene transfer should be without.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Author : Maria Cristina Galli
Publisher : Springer
ISBN 13 : 3319186183
Total Pages : 235 pages
Book Rating : 4.3/5 (191 download)

DOWNLOAD NOW!

Book Synopsis Regulatory Aspects of Gene Therapy and Cell Therapy Products by : Maria Cristina Galli

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Gene Therapy in Neurological Disorders

Author : Mingjie Li
Publisher : Academic Press
ISBN 13 : 012809821X
Total Pages : 450 pages
Book Rating : 4.1/5 (28 download)

DOWNLOAD NOW!

Book Synopsis Gene Therapy in Neurological Disorders by : Mingjie Li

Download or read book Gene Therapy in Neurological Disorders written by Mingjie Li and published by Academic Press. This book was released on 2018-05-25 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders. Each chapter gives an in-depth introduction to the relevant field before diving into the specific tool or application. The book aims to help investigators, students and research staff better understand the principles of gene therapy and its application in the nervous system. Provides background information and experimental details of gene therapy tools applied for neuroscience research and neurological disorders Covers a broad range of gene delivery and regulation tools, therapeutic agents, and target cells, including emerging new technologies such as CRISPR/Cas9 genome editing Discusses applications of gene therapy tools to neurological disorders including neurodegeneration, muscular dystrophy, trauma and chronic pain, and neoplastic diseases

Somatic Gene Therapy

Author : P.L. Chang
Publisher : CRC Press
ISBN 13 : 1351093665
Total Pages : 242 pages
Book Rating : 4.3/5 (51 download)

DOWNLOAD NOW!

Book Synopsis Somatic Gene Therapy by : P.L. Chang

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 242 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.

Gene Therapy of the Central Nervous System: From Bench to Bedside

Author : Michael G. Kaplitt
Publisher : Elsevier
ISBN 13 : 0080454372
Total Pages : 372 pages
Book Rating : 4.0/5 (84 download)

DOWNLOAD NOW!

Book Synopsis Gene Therapy of the Central Nervous System: From Bench to Bedside by : Michael G. Kaplitt

Download or read book Gene Therapy of the Central Nervous System: From Bench to Bedside written by Michael G. Kaplitt and published by Elsevier. This book was released on 2005-11-11 with total page 372 pages. Available in PDF, EPUB and Kindle. Book excerpt: Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

An Introduction to Molecular Medicine and Gene Therapy

Author : Thomas F. Kresina
Publisher : John Wiley & Sons
ISBN 13 : 0471461040
Total Pages : 408 pages
Book Rating : 4.4/5 (714 download)

DOWNLOAD NOW!

Book Synopsis An Introduction to Molecular Medicine and Gene Therapy by : Thomas F. Kresina

Download or read book An Introduction to Molecular Medicine and Gene Therapy written by Thomas F. Kresina and published by John Wiley & Sons. This book was released on 2004-03-24 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Oversight and Review of Clinical Gene Transfer Protocols

Author : Institute of Medicine
Publisher : National Academies Press
ISBN 13 : 030929665X
Total Pages : 78 pages
Book Rating : 4.3/5 (92 download)

DOWNLOAD NOW!

Book Synopsis Oversight and Review of Clinical Gene Transfer Protocols by : Institute of Medicine

Download or read book Oversight and Review of Clinical Gene Transfer Protocols written by Institute of Medicine and published by National Academies Press. This book was released on 2014-03-27 with total page 78 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

Pharmaceutical Biotechnology

Author : Daan J. A. Crommelin
Publisher : CRC Press
ISBN 13 : 9780415285018
Total Pages : 456 pages
Book Rating : 4.2/5 (85 download)

DOWNLOAD NOW!

Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin

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.

Cell and Gene Therapies

Author : Miguel-Angel Perales
Publisher : Springer
ISBN 13 : 3319543687
Total Pages : 288 pages
Book Rating : 4.3/5 (195 download)

DOWNLOAD NOW!

Book Synopsis Cell and Gene Therapies by : Miguel-Angel Perales

Download or read book Cell and Gene Therapies written by Miguel-Angel Perales and published by Springer. This book was released on 2018-11-27 with total page 288 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Cellular Therapy Of Cancer: Development Of Gene Therapy Based Approaches

Author : Robert E Hawkins
Publisher : World Scientific
ISBN 13 : 9814618535
Total Pages : 210 pages
Book Rating : 4.8/5 (146 download)

DOWNLOAD NOW!

Book Synopsis Cellular Therapy Of Cancer: Development Of Gene Therapy Based Approaches by : Robert E Hawkins

Download or read book Cellular Therapy Of Cancer: Development Of Gene Therapy Based Approaches written by Robert E Hawkins and published by World Scientific. This book was released on 2014-05-05 with total page 210 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cancer research has progressed enormously in recent years. This review volume will address recent findings in the area of T-cell therapy for cancer, including use of tumour infiltrating lymphocytes (TILs) as a therapy for melanoma, choice of target antigens, advances in engineered receptors, methods of gene transfer to T cells, review of cell processing methods and clinical trial design. Written by leadings scientists in the field, this up-to-date review on cancer research will be an important reference source to the researchers and healthcare professionals in the field.

Polymers and Nanomaterials for Gene Therapy

Author : Ravin Narain
Publisher : Woodhead Publishing
ISBN 13 : 0081005210
Total Pages : 303 pages
Book Rating : 4.0/5 (81 download)

DOWNLOAD NOW!

Book Synopsis Polymers and Nanomaterials for Gene Therapy by : Ravin Narain

Download or read book Polymers and Nanomaterials for Gene Therapy written by Ravin Narain and published by Woodhead Publishing. This book was released on 2016-01-09 with total page 303 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Gene Therapy

Author : Mauro Giacca
Publisher : Springer Science & Business Media
ISBN 13 : 8847016436
Total Pages : 318 pages
Book Rating : 4.8/5 (47 download)

DOWNLOAD NOW!

Book Synopsis Gene Therapy by : Mauro Giacca

Download or read book Gene Therapy written by Mauro Giacca and published by Springer Science & Business Media. This book was released on 2010-11-01 with total page 318 pages. Available in PDF, EPUB and Kindle. Book excerpt: I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided.

Drug Development for Gene Therapy

Author : Yanmei Lu
Publisher : John Wiley & Sons
ISBN 13 : 1119852781
Total Pages : 500 pages
Book Rating : 4.1/5 (198 download)

DOWNLOAD NOW!

Book Synopsis Drug Development for Gene Therapy by : Yanmei Lu

Download or read book Drug Development for Gene Therapy written by Yanmei Lu and published by John Wiley & Sons. This book was released on 2024-02-28 with total page 500 pages. Available in PDF, EPUB and Kindle. Book excerpt: Drug Development for Gene Therapy Industry-centric perspective on translational and bioanalytical challenges and best practices for gene therapies Drug Development for Gene Therapy focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities, presenting a significant body of data, including information related to safety and efficacy, necessary to advance through the development pipeline into clinical use. The text covers bioanalytical methods and platforms including patient screening assays, different PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy, along with companion diagnostics regulations from US and EU perspectives. The chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industries. To aid in reader comprehension, the text includes clinical examples of relevant therapies in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Written by two highly qualified authors with significant experience in the field, Drug Development for Gene Therapy includes information on: Bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids Detection of cellular immunity and humoral response to viral capsids and transgene proteins, and immunogenicity of gene therapy products Nonclinical and clinical study considerations and methods for biodistribution and shedding Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy Detection and quantification of rAAV integration and off-target editing Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics With comprehensive coverage of the subject, Drug Development for Gene Therapy is a must-have resource for researchers and developers in the areas of pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), along with professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences.