Gene Therapy - From Laboratory To The Clinic


Book Description

With advances in our understanding of the molecular biology of human diseases and the development of efficient gene transfer techniques, the treatment of such diseases as cancer and infectious disease using gene therapy has progressed from a distant prospect to a distinct possibility in a very short time. The development of gene transfer methods which are suitable for different forms of therapy has been a major topic of research over the past several years. A common goal of this research has been to achieve the efficient delivery of genes into cells. The successful implementation of gene transfer as a cure for diseases, however, will continue to require the translation of preclinical studies in gene therapy into effective clinical protocols. This volume outlines the latest developments in cancer treatment using various gene delivery systems, which include cytokine gene transfer, the delivery of anti-ras DNA by retroviral vector and the injection of allogeneic HLA DNA via liposomes. Several of these molecular approaches have recently been approved by the US FDA as human clinical trial protocols in order to assess their therapeutic efficiency and safety for cancer treatment. Further developments in recombinant DNA technology within this field should ultimately lead to dramatic improvements in the practice of medicine.




Exploring Novel Clinical Trial Designs for Gene-Based Therapies


Book Description

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.




Gene Therapy Technologies, Applications and Regulations


Book Description

This text brings together the current data previously separated. It combines information on gene therapy technologies and the attendant regulatory requirements for gene therapy, products and procedures.




Gene Therapy in the Treatment of Cancer


Book Description

The clinical applications of human gene therapy have been particularly fruitful in oncology, and in the past two decades there has been explosive growth in understanding of the genetic lesions leading to neoplasia. This volume in the series Cancer: Clinical Science in Practice reviews progress in the basic and clinical science of gene therapy in oncology, and looks toward future developments. It considers what has worked and what has not in the fast-evolving field of gene therapy, drawing on laboratory studies and clinical trials, including the ground-breaking work of the contributors themselves. Up to date and authoritative, the volumes in this series are intended for a wide audience of clinicians and researchers with an interest in the applications of biomedical science to the understanding and management of cancer.




Gene Therapy of Cancer


Book Description

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.




Cellular Transplantation


Book Description

There have been tremendous strides in cellular transplantation in recent years, leading to accepted practice for the treatment of certain diseases, and use for many others in trial phases. The long history of cellular transplantation, or the transfer of cells from one organism or region of the body to another, has been revolutionized by advances in stem cell research, as well as developments in gene therapy. Cellular Transplants: From Lab to Clinic provides a thorough foundation of the basic science underpinning this exciting field, expert overviews of the state-of-the-art, and detailed description of clinical success stories to date, as well as insights into the road ahead. As highlighted by this timely and authoritative survey, scale-up technologies and whole organ transplantation are among the hurdles representing the next frontier. The contents are organized into four main sections, with the first covering basic biology, including transplant immunology, the use of immunosuppressive drugs, stem cell biology, and the development of donor animals for transplantation. The next part looks at peripheral and reconstructive applications, followed by a section devoted to transplantation for diseases of the central nervous system. The last part presents efforts to address the key challenges ahead, such as identifying novel transplantable cells and integrating biomaterials and nanotechnology with cell matrices. - Provides detailed description of clinical trials in cell transplantation - Review of current therapeutic approaches - Coverage of the broad range of diseases addressed by cell therapeutics - Discussion of stem cell biology and its role in transplantation




Gene Therapy


Book Description

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.




Self-Assembling Complexes for Gene Delivery


Book Description

Recent years have witnessed an explosion of activity in the field of gene therapy. Following advances in our understanding of the molecular basis of disease, hopes are high that the tremendous potential market for drugs employing antisense nucleotides and genes will one day be fulfilled. However, many obstacles remain, not least problems in the technology of gene delivery systems. Much of today's research focuses on non-viral approaches to gene delivery. Of particular importance are supramolecular complexes formed between DNA and various natural and synthetic polymers and lipids, otherwise known as 'self-assembling complexes for gene delivery'. In view of this fact, the editors of this volume have assembled an international team of contributors to present up-to-date reviews of the major chemical, biological and clinical aspects of such gene delivery vectors. Reflecting the diversity of research in this field, this book comprehensively covers: * the principles of self-assembly * natural mechanisms for gene delivery to cells * cationic lipids and liposomes * polyelectrolyte DNA complexes * systemic biodistribution of drug delivery systems * targeting of conjugates for gene delivery * new approaches to gene delivery * clinical evaluation Self-assembling Complexes for Gene Delivery is an essential reference for all professionals with an interest in gene and antisense therapy or novel drug delivery systems, including medicinal and pharmaceutical chemists, clinicians, human geneticists, molecular biologists and pharmacologists.




Gene Therapy for Cancer


Book Description

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 Therapy


Book Description

In 1982, a meeting of unusual influence was held at the Banbury Conference Center of Cold Spring Harbor Laboratory. After an early attempt at treating clinical disease with transferred genes had ignited public attention and scientific controversy, a group of distinguished biologists and physicians came together to assess practical progress towards gene therapy and what its future might be. The geneticist Ted Friedmann wrote a narrative account of the participants' contributions to the meeting, ending with a personal discussion of ethical issues raised by genetic technologies.His book, the first on gene therapy, was widely read but has long been unavailable. It has been reprinted with a new introduction entitled "Gene Therapy 1994", in which the author reviews the field's technical accomplishments and ethical dilemmas. Now that gene therapy has become part of the medical landscape, this volume is of interest as both a historical document and an assessment of the field's current challenges.