Progress in Gene Therapy

Progress in Gene Therapy
Author: Roger Bertolotti
Publisher: VSP
Total Pages: 576
Release: 2000-09-28
Genre: Science
ISBN: 9789067643283

Gene Therapy is expected to revolutionize the practice of medicine at the turn of the third Millennium. Therapeutic/prophylactic benefits should arise from both gene transfer and gene repair/inactivation protocols devised for patient's somatic cells. Gene expression cassettes, designed for the production of therapeutic proteins and non-coding RNA, are thus experimented together with emerging gene repair/inactivation techniques on a variety of inherited, acquired and infectious/parasitic diseases, including complex neuro-degenerative processes. This book presents a collection of chapters on the main aspects of Gene Therapy, some of which have already been treated in the past, and updates and further develops the current survey. It is aimed at understanding why Human Gene Therapy is likely to be a medical breakthrough, although definitive clinical success still needs time to accomplish. The contributions focus on both technical/biomedical concepts and on experimental/clinical data that appear to confer potential universality to Gene Therapy. This book does not claim to provide an exhaustive review of the pathologies, which are currently approached with Gene Therapy. The presentation of current and emerging approaches, together with problems and tentative issues aims to serve as a booster for the development of new applications in every field of medicine and will, therefore, be of value and interest to researchers in this exciting field.

Pharmaceutical Biotechnology

Pharmaceutical Biotechnology
Author: Daan J. A. Crommelin
Publisher: CRC Press
Total Pages: 456
Release: 2002-11-14
Genre: Medical
ISBN: 9780415285018

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.

Gene Therapy and Cancer Research Progress

Gene Therapy and Cancer Research Progress
Author: Jessica L. Lewis
Publisher: Nova Publishers
Total Pages: 240
Release: 2008
Genre: Health & Fitness
ISBN: 9781600218118

Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. This new book presents the latest research in the field from around the world.

Gene Therapy for Cancer

Gene Therapy for Cancer
Author: Kelly K. Hunt
Publisher: Springer Science & Business Media
Total Pages: 469
Release: 2007-10-26
Genre: Medical
ISBN: 159745222X

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

Gene and Cell Therapy
Author: Nancy Smyth Templeton
Publisher: CRC Press
Total Pages: 1154
Release: 2008-10-06
Genre: Science
ISBN: 084938799X

Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Immunopharmacology

Immunopharmacology
Author: Manzoor M. Khan
Publisher: Springer Science & Business Media
Total Pages: 275
Release: 2008-12-19
Genre: Medical
ISBN: 0387779760

During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders
Author: Mingjie Li
Publisher: Academic Press
Total Pages: 450
Release: 2018-05-25
Genre: Medical
ISBN: 012809821X

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

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 127
Release: 2020-08-27
Genre: Medical
ISBN: 0309672988

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.