Adeno-Associated Virus (AAV) Vectors in Gene Therapy PDF Download
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Author: Kenneth I. Berns
Publisher: Springer Science & Business Media
ISBN: 3642802079
Category : Medical
Languages : en
Pages : 179
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Book Description
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).
Author: Kenneth I. Berns
Publisher: Springer Science & Business Media
ISBN: 3642802079
Category : Medical
Languages : en
Pages : 179
Get Book Here
Book Description
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).
Author: Andras Guttman
Publisher: Newnes
ISBN: 0080931359
Category : Science
Languages : en
Pages : 391
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Book Description
Capillary Gel Electrophoresis and Related Microseparation Techniques covers all theoretical and practical aspects of capillary gel electrophoresis. It also provides an excellent overview of the key application areas of nucleic acid, protein and complex carbohydrate analysis, affinity-based methodologies, micropreparative aspects and related microseparation methods. It not only gives readers a better understanding of how to utilize this technology, but also provides insights into how to determine which method will provide the best technical solutions to particular problems. This book can also serve as a textbook for undergraduate and graduate courses in analytical chemistry, analytical biochemistry, molecular biology and biotechnology courses. - Covers all theoretical and practical aspects of capillary gel electrophoresis - Excellent overview of the key applications of nucleic acid, protein and complex carbohydrate analysis, affinity-based methodologies, micropreparative aspects and related microseparation methods - Teaches readers how to use the technology and select methods that are ideal for fundamental problems - Can serve as a textbook for undergraduate and graduate courses in analytical chemistry, analytical biochemistry, molecular biology and biotechnology courses
Author: Kenneth Brigham
Publisher: CRC Press
ISBN: 9780824700607
Category : Science
Languages : en
Pages : 460
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Book Description
This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems.
Author: Peter M. Howley
Publisher: Lippincott Williams & Wilkins
ISBN: 1975112555
Category : Medical
Languages : en
Pages : 2737
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Book Description
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.
Author: Terence R. Flotte
Publisher: Elsevier
ISBN: 9780444519498
Category : Biochemistry
Languages : en
Pages : 304
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Book Description
Author: Dongsheng Duan
Publisher: Springer Science & Business Media
ISBN: 144191207X
Category : Medical
Languages : en
Pages : 281
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Book Description
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.
Author: Fredric P. Manfredsson
Publisher:
ISBN: 9781493990658
Category : Electronic books
Languages : en
Pages : 328
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Book Description
This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. Written in the highly successful Methods in Molecular Biology series format, 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. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.
Author: Nicholas Muzyczka
Publisher: Springer
ISBN: 9783540524311
Category : Medical
Languages : en
Pages : 176
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Book Description
In the past ten years there has been enormous progress in the development of eukaryotic viral vectors. In general, these vectors have been developed for one of three reasons: to achieve high levels of expression of a particular gene product (poxvirus, baculovirus, and adenovirus), to clone eukaryotic genes in combination with functional assays (Epstein-Barr virus), of for use as delivery vehicles for the stable introduction of foreign genes into mammalian cells (retroviruses, Epstein-Barr virus, and adeno-associated virus). Each vector has its strengths and weaknesses that are rooted in the sometimes bewildering stra tegies that the parent viruses use for propagation. No one of these vectors is appropriate for all of the problems that a mole cular biology laboratory is likely to encounter, and few of us are knowledgeable in the molecular virology of all of these viruses. This volume represents an attempt by the authors to assem ble a review of these vectors in one place and in a form useful to laboratories that do not necessarily have experience with eukaryotic viruses. Clearly, any virus can be modified to serve as a vector for some purposes, and it was not possible to include a description of all of these. In addition, one eukaryotic vector, SV40 (the first one developed), has been reviewed so widely that we saw no reason to include it here.
Author: Esteban Domingo
Publisher: Elsevier
ISBN: 0080564968
Category : Science
Languages : en
Pages : 573
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Book Description
New viral diseases are emerging continuously. Viruses adapt to new environments at astounding rates. Genetic variability of viruses jeopardizes vaccine efficacy. For many viruses mutants resistant to antiviral agents or host immune responses arise readily, for example, with HIV and influenza. These variations are all of utmost importance for human and animal health as they have prevented us from controlling these epidemic pathogens. This book focuses on the mechanisms that viruses use to evolve, survive and cause disease in their hosts. Covering human, animal, plant and bacterial viruses, it provides both the basic foundations for the evolutionary dynamics of viruses and specific examples of emerging diseases. - NEW - methods to establish relationships among viruses and the mechanisms that affect virus evolution - UNIQUE - combines theoretical concepts in evolution with detailed analyses of the evolution of important virus groups - SPECIFIC - Bacterial, plant, animal and human viruses are compared regarding their interation with their hosts
Author: Rishabha Malviya
Publisher: CRC Press
ISBN: 1040052118
Category : Medical
Languages : en
Pages : 235
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Book Description
Neurological illnesses pose one of the biggest hazards to the healthcare system today. This new book brings together the latest methods of gene therapy that can be used to treat both inherited and sporadic neurodegenerative diseases. It presents the most significant advances in gene transfer methods as well as the most recent understandings of the mechanisms behind specific neurodegenerative illnesses, placing these into the context of gene therapy approaches for the central nervous system. The book introduces the basics of neurodegenerative conditions and the physiological basis for their occurrence in humans. It also discusses the various proteins useful in neuro diseases and their viral applications. The book explores how stem cells can be used to learn about the future of gene therapy along with the significance of the sequences that silence genes. It discusses the functions of astrocytes in different brain regions, as well as in-vivo research in gene therapy for neurodegenerative disorders.
