Download or read book Novel Viral Vectors for Gene Therapy 2023 written by Ottmar Herchenröder and published by . This book was released on 2024-03-18 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Viral vectors are gene transfer tools assembled from the backbones of naturally occurring viruses. These replication-incompetent vehicles transfer assigned payloads into eukaryotic cells. Numerous viral vector systems that influence cells or tissues have been used to perform basic and preclinical research. Some virus-derived vectors found their way into clinical practice.
Download or read book Viral Vectors in Gene Therapy written by Einari Aavik and published by . This book was released on 2010 with total page 150 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Viral Vectors for Gene Therapy written by Curtis A. Machida and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 591 pages. Available in PDF, EPUB and Kindle. Book excerpt: Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors.
Download or read book Viral Gene Therapy written by Ke Xu and published by BoD – Books on Demand. This book was released on 2011-07-20 with total page 466 pages. Available in PDF, EPUB and Kindle. Book excerpt: The development of technologies that allow targeting of specific cells has progressed substantially in recent years for several types of vectors, particularly viral vectors, which have been used in 70% of gene therapy clinical trials. Particular viruses have been selected as gene delivery vehicles because of their capacities to carry foreign genes and their ability to efficiently deliver these genes associated with efficient gene expression. This book is designed to present the most recent advances in viral gene therapy
Download or read book Engineering of Novel Adeno Associated Virus Vectors for Gene Therapy Applications written by Jorge Luis Santiago Ortiz and published by . This book was released on 2016 with total page 102 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy – the introduction of genetic material into cells and tissues of interest for a therapeutic purpose – has emerged as a very promising treatment for many diseases. Recent advances in genomics and proteomics, coupled with the advent of genome editing technologies, have generated an immense pool of potential nucleic acid cargoes that could be delivered as therapies for a wide array of diseases, ranging from monogenic disorders to cancer. However, before such therapies can be successful, a major hurdle must be overcome: the development of gene-carrying vehicles – also referred to as vectors – that can safely, efficiently, and specifically deliver those therapeutic payloads to the desired cells. The goal of this dissertation was therefore to address a major need in the field: the development of improved gene delivery vectors. To date, more than 2,000 clinical trials employing gene transfer have taken place, establishing the safety of a number of vectors. Non-viral vectors can be easily produced at a large-scale and are amenable to the engineering of their chemical and physical properties via chemical modifications, but they suffer from a low delivery efficiency and cell toxicity. On the other hand, viral vectors harness the highly evolved mechanisms that viruses have developed to efficiently recognize and infect cells and offer several advantages that make them suitable candidates for use in gene delivery, both for therapeutic application and as tools for biological studies. In fact, gene therapy has enjoyed increasing success in clinical trials for numerous disease targets in large part due to the gene delivery capabilities viral vectors. Vectors derived from viruses have been used in the majority (over 68%) of gene therapy clinical trials to date, and the most frequently used have been based on adenovirus, retrovirus, vaccinia virus, herpesvirus, and adeno-associated virus (AAV). AAV vectors are non-pathogenic and can transduce numerous dividing and non-dividing cell types. Because of these characteristics, AAV vectors have been utilized for gene therapy in various tissues. The amino acid composition of the viral capsid affects tropism (tissue specificity), cell receptor usage, and susceptibility to anti-AAV neutralizing antibodies – properties that influence efficacy in therapeutic gene delivery. However, AAV vectors can still encounter formidable impediments to efficacious gene delivery, including poor transduction (infection and expression of delivered gene) of some cell types, off-target transduction, difficulties with biological transport barriers, and potential risks associated with the integration of their genetic load. Extensive engineering of the AAV capsid promises to overcome these delivery challenges and improve numerous clinically relevant properties. To this end, the overarching goal of my work in the Schaffer Laboratory, which is presented in this thesis dissertation, was to advance current gene delivery methods through the engineering and characterization of novel adeno-associated virus vectors for gene therapy and research applications. To access new viral capsid sequences with potentially enhanced infectious properties and to gain insights into AAV’s evolutionary history, we computationally designed and experimentally constructed an ancestral AAV capsid library. We performed selection for infectivity on the library, studied the resulting amino acid distribution, and characterized the selected variants, which yielded viral particles that were broadly infectious across multiple cell types. Ancestral variants displayed higher thermostability than modern (extant) natural AAV serotypes, a property that makes them promising templates for protein engineering applications, including directed evolution. Additionally, some variants displayed high in vivo infectivity on a mouse model, highlighting their potential for gene therapy. Motivated by the success of directed evolution in the engineering of proteins with novel or enhanced properties, I worked in the engineering of AAV vectors for gene delivery to glioblastoma multiforme (GBM), a highly aggressive type of brain cancer. For this, I conducted directed evolution to select AAV variants with selective localization to and infectivity on GBM tumor cells and tumor initiating cells (TICs). Using an accurate GBM mouse model, I performed in vitro and in vivo selection, recovering viral particles that successfully trafficked to tumor cells and TICs in the brain after systemic administration to tumor-bearing animals. Following three rounds of in vivo selection, convergence was achieved upon several variants, the most abundant of which emerged from the ancestral reconstruction library. The selected variants are currently being characterized and assessed for their ability to deliver reporter and therapeutic genes, hopefully resulting in improved suppression of tumor progression compared to delivery with existing AAV serotypes. These novel vectors could enable new, potent therapies to treat GBM tumors and pave the way for engineering AAV vectors for other cancer targets. In summary, this dissertation presents work on the development and characterization of a novel AAV capsid library, as well as on the implementation of this and of other libraries towards the engineering of novel AAV variants with selective gene delivery properties for brain tumors. The work herein presented aims to advance both the field of AAV vector engineering as a whole and the specific application of AAV vectors towards next generation cancer therapies.
Download or read book Nonviral Vectors for Gene Therapy written by Mien-Chie Hung and published by Elsevier. This book was released on 1999-07-01 with total page 471 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer within humans has been an obstacle until about 10 years ago. At that time, it was found that viral vectors were effective carriers of "healthy genes" into patients' cells. The problem, however, was that viral vectors proved unnecessarily harmful to humans: subjects experienced inflamatory activity and negative immunological responses to the genes. Viral vectors were also unable to meet the needs of the pharmaceutical community: they were not reproducible in large-scale proportions in cost-effective ways. Thus, research was undertaken to find a safer way to transfer genes to patients without jeopardizing the safety of the patient. And so non-viral vectors were discovered. This volume presents the various non-viral vectors currently under development. Although not methodologically oriented, it will provide the necessary details behind the development of the vectors. This information will prove useful to both researchers and clinicians. Key Features * Presents state-of-the art developments of nonviral vectors as tools for modern molecular medicine * Covers all types of nonviral vectors, from molecular structure to therapeutic application Provides a comprehensive review of synthetic vectors * Includes contributions from major investigators and leading experts in the field
Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Recombinant Adeno associated Virus based Vectors for Gene Therapy written by Verena Vanessa Emmerling and published by . This book was released on 2016 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Development of Novel Episomal Non viral Vectors for Stable Long term Expression for Gene Therapy written by Orestis Argyros and published by . This book was released on 2008 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Immune Responses to Viral Vectors for Gene Therapy written by and published by . This book was released on 2003 with total page 66 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Engineering of Non Viral Vectors for Gene Therapy written by Marcus Laird Forrest and published by . This book was released on 2003 with total page 14 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gene Therapy New Developments of Non viral Vectors written by Iulian Oprea and published by . This book was released on 2010 with total page 256 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Fields Virology Emerging Viruses written by Peter M. Howley and published by Lippincott Williams & Wilkins. This book was released on 2020-02-11 with total page 2597 pages. Available in PDF, EPUB and Kindle. Book excerpt: Now in four convenient volumes, Field’s Virology remains the most authoritative reference in this fast-changing field, providing definitive coverage of virology, including virus biology as well as replication and medical aspects of specific virus families. This volume of Field’s Virology: Emerging Viruses, 7th Edition covers recent changes in emerging viruses, providing new or extensively revised chapters that reflect these advances in this dynamic field.
Download or read book Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies written by Daniel Scherman and published by World Scientific. This book was released on 2013-12-10 with total page 557 pages. Available in PDF, EPUB and Kindle. Book excerpt: This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.
Download or read book Cell Culture Engineering written by Wei-Shu Hu and published by Springer. This book was released on 2006-08-16 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the introduction of recombinant human growth hormone and insulin a quarter century ago, protein therapeutics has greatly broadened the ho- zon of health care. Many patients suffering with life-threatening diseases or chronic dysfunctions, which were medically untreatable not long ago, can attest to the wonder these drugs have achieved. Although the ?rst generation of p- tein therapeutics was produced in recombinant Escherichia coli, most recent products use mammalian cells as production hosts. Not long after the ?rst p- duction of recombinant proteins in E. coli, it was realized that the complex tasks of most post-translational modi?cations on proteins could only be ef?ciently carried out in mammalian cells. In the 1990s, we witnessed a rapid expansion of mammalian-cell-derived protein therapeutics, chie?y antibodies. In fact, it has been nearly a decade since the market value of mammalian-cell-derived protein therapeutics surpassed that of those produced from E. coli. A common characteristic of recent antibody products is the relatively large dose required for effective therapy, demanding larger quantities for the treatment of a given disease. This, coupled with the broadening repertoire of protein drugs, has rapidly expanded the quantity needed for clinical applications. The increasing demand for protein therapeutics has not been met exclusively by construction of new manufacturing plants and increasing total volume capacity. More - portantly the productivity of cell culture processes has been driven upward by an order of magnitude in the past decade.
Download or read book Adeno Associated Virus AAV Vectors in Gene Therapy written by Kenneth I. Berns and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se quence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected gene must be available. For a vector to be of use several attributes are highly desirable: these include ability to carry the intact gene (although this may be either the genomic or the cDNA form) in a stable form, ability to introduce the gene into the desired cell type, ability to express the introduced gene in an appropriately regulated manner for an extended period of time, and a lack of toxicity for the recipient. Also of concern is the frequency of cell transformation and, in some cases, the ability to introduce the gene into nondividing stem cells. Sev eral animal viruses have been tested as potential vectors, but none has proven to have all the desired properties described above. For example, retroviruses are difficult to propagate in sufficient titers, do not integrate into nondividing cells, and are of concern because of their oncogenic properties in some hosts and because they integrate at many sites in the genome and, thus, are potentially insertional mutagens. Additionally, genes introduced by retroviral vectors are frequently expressed for relatively short periods of time. A second virus used as a vector in model systems has been adenovirus (Ad).
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.
