Adeno-Associated Virus (AAV) Vectors in Gene Therapy

Adeno-Associated Virus (AAV) Vectors in Gene Therapy
Title Adeno-Associated Virus (AAV) Vectors in Gene Therapy PDF eBook
Author Kenneth I. Berns
Publisher Springer Science & Business Media
Pages 179
Release 2012-12-06
Genre Medical
ISBN 3642802079

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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).

Fields Virology: Emerging Viruses

Fields Virology: Emerging Viruses
Title Fields Virology: Emerging Viruses PDF eBook
Author Peter M. Howley
Publisher Lippincott Williams & Wilkins
Pages 2737
Release 2020-02-11
Genre Medical
ISBN 1975112555

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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.

Viral Vectors for Gene Therapy

Viral Vectors for Gene Therapy
Title Viral Vectors for Gene Therapy PDF eBook
Author Fredric P. Manfredsson
Publisher
Pages 328
Release 2019
Genre Electronic books
ISBN 9781493990658

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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.

Muscle Gene Therapy

Muscle Gene Therapy
Title Muscle Gene Therapy PDF eBook
Author Dongsheng Duan
Publisher Springer Science & Business Media
Pages 281
Release 2009-11-26
Genre Medical
ISBN 144191207X

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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.

Laboratory techniques in biochemistry and molecular biology

Laboratory techniques in biochemistry and molecular biology
Title Laboratory techniques in biochemistry and molecular biology PDF eBook
Author Terence R. Flotte
Publisher Elsevier
Pages 304
Release 1969
Genre Biochemistry
ISBN 9780444519498

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Immunopharmacology

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

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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.

Holland-Frei Cancer Medicine

Holland-Frei Cancer Medicine
Title Holland-Frei Cancer Medicine PDF eBook
Author Robert C. Bast, Jr.
Publisher John Wiley & Sons
Pages 2004
Release 2017-03-10
Genre Medical
ISBN 111900084X

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Holland-Frei Cancer Medicine, Ninth Edition, offers a balanced view of the most current knowledge of cancer science and clinical oncology practice. This all-new edition is the consummate reference source for medical oncologists, radiation oncologists, internists, surgical oncologists, and others who treat cancer patients. A translational perspective throughout, integrating cancer biology with cancer management providing an in depth understanding of the disease An emphasis on multidisciplinary, research-driven patient care to improve outcomes and optimal use of all appropriate therapies Cutting-edge coverage of personalized cancer care, including molecular diagnostics and therapeutics Concise, readable, clinically relevant text with algorithms, guidelines and insight into the use of both conventional and novel drugs Includes free access to the Wiley Digital Edition providing search across the book, the full reference list with web links, illustrations and photographs, and post-publication updates