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Gene Therapy

Gene Therapy
  • Author : Evelyn B. Kelly
  • Publisher :Unknown
  • Release Date :2007
  • Total pages :198
  • ISBN : 0313337608
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Summary : Presents an introduction to gene therapy, discussing its scientific background, the types of disorders which it can potentially treat, the ethical, social, and religious issues surrounding its use, and the role of government regulation.

Gene Therapy of Cancer

Gene Therapy of Cancer
  • Author : Edmund C. Lattime,Stanton L. Gerson
  • Publisher :Unknown
  • Release Date :2013-08-28
  • Total pages :554
  • ISBN : 9780123946324
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Summary : Gene therapy as a treatment for cancer is at a critical point in its evolution. Exciting new developments in gene targeting and vector technology, coupled with results from the first generation of preclinical and clinical studies have led to the design and testing of new therapeutic approaches. The Third Edition of Gene Therapy of Cancer provides crucial updates on the basic and applied sciences of gene therapy. It offers a comprehensive assessment of the field including the areas of suicide gene therapy, oncogene and suppressor gene targeting, immunotherapy, drug resistance gene therapy, and the genetic modification of stem cells. Researchers at all levels of development, from basic laboratory investigators to clinical practitioners, will find this book to be instructive. Cancer gene therapy, like cancer therapy in general, is evolving rapidly, testing new concepts, targets and pathways, evoking new technologies, and passing new regulatory hurdles. Its essence, however, has not changed: the hope and challenges of returning altered genes to normal, using targeted gene expression to alter the function of both tumor and microenvironment, and in some cases normal cells, and delivering functionally important genes to specific cell types to increase sensitivity to killing or to protect normal cells from cancer therapies. In some instances, gene therapy for cancer forms a continuum from gene repair through the use of molecularly modified cells; the use of viral and non-viral vector based gene delivery to both tumor and tumor microenvironment; the use of viral and gene based vaccines; and development of new gene-based therapeutics. The unique mechanistically chosen vector platforms are at the heart of this technology because they allow for direct and selective cell death and transient to sustained delivery of vaccine molecules or molecules that affect the microenvironment, vasculature, or the immune response. Explains the underlying cancer biology necessary for understanding proposed therapeutic approaches Presents in-depth description of targeting systems and treatment strategies Covers the breadth of gene therapy approaches including immunotherapeutic, drug resistance,oncolytic viruses, as well as regulatory perspectives from both the NCI and FDA

Gene Therapy Methods

Gene Therapy Methods
  • Author : M. Ian Phillips
  • Publisher :Unknown
  • Release Date :2002
  • Total pages :728
  • ISBN : 9780121822477
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Summary : 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. 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.

A Guide to Human Gene Therapy

A Guide to Human Gene Therapy
  • Author : Roland W. Herzog,Sergei Zolotukhin
  • Publisher :Unknown
  • Release Date :2010
  • Total pages :388
  • ISBN : 9789814280914
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Summary : Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy - though still in its infancy - remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ( vector) to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession. This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system.Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Gene Therapy of Cancer

Gene Therapy of Cancer
  • Author : Wolfgang Walther,Ulrike Stein
  • Publisher :Unknown
  • Release Date :2000
  • Total pages :645
  • ISBN : 9781592590865
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Summary : In Gene Therapy of Cancer: Methods and Protocols, Wolfgang Walther and Ulrike Stein survey the rapidly evolving field cancer gene therapy and provide a broad array of leading-edge protocols for the delivery of therapeutic genes into tumors. Described in step-by-step fashion and enriched with each author's own practical tips, these readily reproducible methods are currently being widely applied in cancer gene therapy investigations, including immunotherapy and tumor vaccination, suicide gene therapy, antioncogene therapy, and antisense and ribozyme gene therapy. Representative strategies are provided for gene targeting and for viral or nonviral gene delivery in cancer therapy, as well as a significant number of clinical protocols for the development of novel cancer gene therapies. Gene Therapy of Cancer: Methods and Protocols offers basic and clinical researchers a broad ranging overview and collection of the most recent advances in gene transfer techniques. Written by leading international authorities, its readily reproducible, cutting-edge methods constitute today's most valuable tools for the study of cancer gene therapy in both the laboratory and clinical trials.

Gene Therapy of Cancer

Gene Therapy of Cancer
  • Author : Wolfgang Walther,Ulrike Stein
  • Publisher :Unknown
  • Release Date :2000-02-17
  • Total pages :645
  • ISBN : 0896038432
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Summary : Since the discovery of the molecular structure of genes and the unveiling of the molecular basis of numerous human diseases, scientists have been fas- nated with the possibility of treating certain diseases by transducing foreign DNA into the affected cells. Initially, it was proposed that the foreign DNA could either replace defective nonfunctional genes, or code for therapeutic proteins. This concept has evolved into the rapidly growing field of gene therapy. Even though surgery, radiotherapy, and chemotherapy are widely ava- able and routinely used for cancer treatment, these therapies fail to cure approximately 50 percent of cancer patients. Therefore, since it is a disease characterized by aberrant gene expression, cancer has been a target of gene therapy research since the inception of this treatment modality. Numerous cancer gene therapy strategies are currently being investigated, including gene replacement therapy, the regulation of gene expression to modulate immu- logical responses to tumors, the direct killing of tumor cells, and direct int- ference with tumor growth. In this context, gene transfer systems, tumor-specific expression vectors, and novel therapeutic genes have been extensively st- ied. All these strategies aim for the selective destruction of human malignant disease while circumventing the destruction of nonmalignant cells and tissues thereby minimizing toxicity to the patient.

Gene Therapy for Viral Infections

Gene Therapy for Viral Infections
  • Author : Patrick Arbuthnot
  • Publisher :Unknown
  • Release Date :2015-06-01
  • Total pages :392
  • ISBN : 9780124114524
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Summary : Gene Therapy for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options. Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors. Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology. Provides coverage of gene therapy for a variety of infections, including HBV, HCV, HIV, hemorrhagic fever viruses, and respiratory and other viral infections Bridges the gap between the basic science and the important medical applications of this technology Features a broad approach to the topic, including an essential overview and the applications of gene therapy, synthetic RNA, and other antiviral strategies that involve nucleic acid engineering Presents perspectives on the future use of nucleic acids as a novel class of antiviral drugs Arms the reader with the cutting-edge information needed to stay abreast of this developing field

Gene Therapy

Gene Therapy
  • Author : Blankenstein
  • Publisher :Unknown
  • Release Date :2012-12-06
  • Total pages :380
  • ISBN : 9783034870115
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Summary : K. Sikora Gene therapy is one of the fastest developing areas in modern medical research. Transcending the classical preclinical and clinical disciplines, it is likely to have far reaching consequences in the practice of medicine, as we enter the next millennium. Currently, there are over 200 seperate active clinical trials with over 2,500 patients entered. These studies involve over 20 countries and include patients with a wide range of diseases, including cancer, HIV infection; cystic fibrosis (CF), haemophilia, diabetes, immune deficiencies, metabolic disorders, ischaemic heart disease and arthritis. Gene therapy can be defined as the deliberate transfer of DNA for therapeutic purposes. There is a further implication that only specific sequences containing rel evant genetic information are used; otherwise, transplantation procedures involving bone marrow, kidney or liver could be considered a form of gene therapy. The con cept of transfer of genetic information as a practical clinical tool arose from the gene-cloning technology, developed during the 1970s. Without the ability to isolate and replicate defined genetic sequences, it would be impossible to produce purified material for clinical use. The drive for the practical application of this technology came from the biotechnology industry with its quest for complex human biomole cules produced by recombinant techniques in bacteria. Within a decade, pharma ceutical-grade insulin, interferon, interleukin 2 and tumour necrosis factor were all involved in clinical trials. The next step was to obtain gene expression in vivo.

Fast Facts: Gene Therapy

Fast Facts: Gene Therapy
  • Author : R. Herzog,L. Popplewell
  • Publisher :Unknown
  • Release Date :2020-04-24
  • Total pages :104
  • ISBN : 9783318066678
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Summary : 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 Protocols

Gene Therapy Protocols
  • Author : Joseph LeDoux
  • Publisher :Unknown
  • Release Date :2014-11-04
  • Total pages :313
  • ISBN : 1627039643
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Summary : In Gene Therapy Protocols, Volumes 1 and 2, internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors (Volume 1) and Design and Characterization of Gene Transfer Vectors (Volume 2). In this second volume, readers will find a comprehensive resource of current and emerging methods for the processing and characterization of viral and non-viral gene transfer vectors.

Muscle Gene Therapy

Muscle Gene Therapy
  • Author : Dongsheng Duan
  • Publisher :Unknown
  • Release Date :2009-11-26
  • Total pages :275
  • ISBN : 144191207X
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Summary : Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.

Gene Therapy for Autoimmune and Inflammatory Diseases

Gene Therapy for Autoimmune and Inflammatory Diseases
  • Author : Yuti Chernajovsky,Paul D. Robbins
  • Publisher :Unknown
  • Release Date :2011-01-28
  • Total pages :239
  • ISBN : 3034601654
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Summary : In this monograph about gene therapy of autoimmune and inflammatory d- orders we have gathered international experts and leaders from different fields to review the state of the art advances on topics ranging from disease entities to vectors and engineered cells. The different approaches described in each chapter take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long-term genetic interventions. Gene therapy also serves as a testing ground for new therapeutic entities and helps provide proof of principle for their potential therapeutic role in animal models of disease. Scaling up from mice to men still remains an important h- dle not only from the quantitative point of view, but also for currently unknown and unexpected secondary effects of the vector or the transgene. Some of these approaches have already been tested in the clinic, but much more needs to be done to understand the human conditions treated and the n- ural history of their pathology. We are indebted to the secretarial assistance of Ms. Lin Wells (Bone and Joint Research Unit, London, UK) and the help of Hans Detlef Klüber for his help in getting this book published. We hope this book will be of interest to c- nicians and scientists and inspiring to students of the subject who will use their own ingenuity and knowledge to further forward this discipline into clinical use.

Interdisciplinary Approaches to Gene Therapy

Interdisciplinary Approaches to Gene Therapy
  • Author : Stefan Müller,Jürgen W. Simon,Jan W. Vesting
  • Publisher :Unknown
  • Release Date :2012-12-06
  • Total pages :292
  • ISBN : 9783642608292
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Summary : Current therapies for most human genetic diseases are inadequate. In response to the need for effective treatments, modern molecular genetics is providing tools for an unprecedented new approach to the treatment of diseases; e.g. the direct manipulation of mutant genes or the input on new therapeutic genes. The treatment of human disease by gene transfer has now moved from the theoretical to the practical realm. With the initiation of clinical trials involving somatic gene therapy in different countries, a critical assessment of the different aspects involved with this new technique is necessary. This volume provides an overview on all these interdisciplinary aspects by some well known experts all over the world.

Gene Therapy

Gene Therapy
  • Author : Surendra Nimesh
  • Publisher :Unknown
  • Release Date :2013
  • Total pages :380
  • ISBN : 1907568409
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Summary : Gene therapy is emerging as a new class of therapeutics for the treatment of inherited and acquired diseases. However, poor cellular uptake and instability of DNA in the physiological milieu limits its therapeutic potential, hence a vector which can protect and efficiently transport DNA to the target cells must be developed. Nanotechnology-based non-viral vectors have been proposed as potential candidates. Various polymeric nanoparticles have been shown to be suitable, with high cellular uptake efficiencies and reduced cytotoxicity. These delivery vectors form condensed complexes with DNA which result in shielding against enzymatic degradation and enhanced cellular targeting. Advantages including easy manipulatibility, high stability, low cost and high payload, mean that nanoparticles from various polymers have been exploited. Gene therapy gives a systematic account of the many aspects of nanotechnology mediated gene therapy, from the preparation of nanoparticles to physicochemical characterization, and follows with applications in in vitro and in vivo models. This book emphasizes the various aspects of nanotechnology-based gene therapy, with initial chapters detailing the tools and techniques available for preparation and in vitro and in vivo characterization of nanoparticles. Later chapters provide exhaustive details on polymeric systems employed for gene therapy. Provides an overview of nanotechnology applications in gene therapy, from preparation of nanoparticles to in vitro and in vivo studies Details the tools and techniques available for preparation, characterization and in vitro and in vivo study of nanoparticles Details the limitations of nanoparticle-mediated gene therapy and proposes ways in which they may be overcome

Gene Therapy

Gene Therapy
  • Author : Mauro Giacca
  • Publisher :Unknown
  • Release Date :2010-11-01
  • Total pages :303
  • ISBN : 8847016436
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Summary : 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.

Suicide Gene Therapy

Suicide Gene Therapy
  • Author : Caroline J. Springer
  • Publisher :Unknown
  • Release Date :2004
  • Total pages :555
  • ISBN : 9781592594290
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Summary : The first comprehensive review of both the theory and practice of suicide gene therapy. The authors cover all the major aspects of suicide gene therapy, including the design and use of vectors in gene transduction, various enzyme and prodrug systems, the mechanistic analysis of the bystander effect, the design and synthesis of prodrugs, immunological implications, and its clinical impact. They also describe all the cutting-edge methods needed to explore, study, and advance understanding of the basic biology underlying gene therapy. Each fully tested method includes step-by-step instructions, a discussion of the principle behind the technique, equipment and reagent lists, tips on troubleshooting and avoiding pitfalls, and notes on the interpretation and use of results. Understand the latest developments in suicide gene therapy Use readily reproducible methods to explore, study, and advance suicide gene therapy Refer to the first book to integrate the theory and practice of suicide gene therapy.

Translating Gene Therapy to the Clinic

Translating Gene Therapy to the Clinic
  • Author : Jeffrey Laurence,Michael Franklin
  • Publisher :Unknown
  • Release Date :2014-11-14
  • Total pages :346
  • ISBN : 9780128005644
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Summary : 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

Genetic Testing and Gene Therapy

Genetic Testing and Gene Therapy
  • Author : James Wolfe
  • Publisher :Unknown
  • Release Date :2015-07-15
  • Total pages :128
  • ISBN : 9781622755738
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Summary : This book covers the foundations of genes and heredity to give readers a solid understanding of what modern genetics has been built on, before examining the ways in which genetic testing is used to assess genetic risk.

The Ethics of Human Gene Therapy

The Ethics of Human Gene Therapy
  • Author : LeRoy Walters,Joseph P Kennedy Professor of Christian Ethics at the Kennedy Institute of Ethics LeRoy Walters,Julie Gage Palmer,Julie G. Palmer
  • Publisher :Unknown
  • Release Date :1997
  • Total pages :209
  • ISBN : 0195059557
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Summary : They start with the current techniques of gene addition, using non-reproductive (somatic) cells in an effort to cure or treat disease. Next they address the technical problems and moral issues facing attempts to prevent disease through genetically modifying early human embryos or sperm and egg cells. These changes would be passed on to future generations. Chapter 4, in many ways the most original part of this volume, confronts the issue of employing genetic means to improve human abilities and appearance.

Suicide Gene Therapy

Suicide Gene Therapy
  • Author : Nejat Düzgüneş
  • Publisher :Unknown
  • Release Date :2019-02-04
  • Total pages :190
  • ISBN : 1493989219
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Summary : This detailed volume explores the methods used for most of the recent approaches to suicide gene therapy of cancer, which exploits promoters that are specific to cancer cells, thereby ensuring (or greatly increasing the likelihood) that the therapeutic gene is expressed only in cancer cells. The book also contains chapters describing methods to improve the safety of cell therapy and techniques utilizing bone marrow mesenchymal cells. Written for the highly successful Methods in Molecular Biology series, 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 practical, Suicide Gene Therapy: Methods and Protocols serves as an ideal guide for researchers expanding upon our knowledge and application of this vital form of cancer therapy.

Gene Therapy for HIV

Gene Therapy for HIV
  • Author : Gerhard Bauer,Joseph S. Anderson
  • Publisher :Unknown
  • Release Date :2014-02-07
  • Total pages :72
  • ISBN : 9781493904341
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Summary : This Brief describes the concept and realization of gene therapy for HIV from the unique historic perspective and insight of two pioneers of the clinical applications of stem cell gene therapy for HIV. Gerhard Bauer applied ribozyme-anti-HIV and other vectors to manufacture clinical grade, HIV-resistant hematopoietic stem cells for the first patients that received stem cell gene therapy for HIV, including the first child in the world and the first fully marrow-ablated HIV infected patient. Joseph Anderson developed the most recent and most potent combination anti-HIV lentiviral vectors and pluripotent stem cell applications for HIV gene therapy and tested these in the appropriate in vitro and vivo models, paving the way for novel HIV gene therapy approaches to possibly cure patients. In Gene Therapy for HIV, Bauer and Anderson discuss the unique aspects of this therapy, including its limitations and proper safety precautions and outline a path for a possible functional cure for HIV using stem cell gene therapy based on a cure already achieved with a bone marrow stem cell transplantation performed in Germany using donor stem cells with a naturally arising CCR5 mutation. In addition, the Brief provides a thorough and methodical explanation of the basics of gene therapy, gene therapy vector development, in vitro and in vivo models for HIV gene therapy and clinical applications of HIV gene therapy, including Good Manufacturing Practices.