Adeno Associated Virus AAV Vectors in Gene Therapy

Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements.

Author: Kenneth I. Berns

Publisher: Springer

ISBN: 9783642802096

Category: Medical

Page: 173

View: 940

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).

Adeno Associated Virus Vectors

This volume provides a complete and timely guide to the use of adeno-associated virus (AAV) vectors for genetic manipulation of mammalian tissues.

Author: Michael J. Castle

Publisher: Humana Press

ISBN: 9781493991389

Category: Medical

Page: 426

View: 955

This volume provides a complete and timely guide to the use of adeno-associated virus (AAV) vectors for genetic manipulation of mammalian tissues. Beginning with methods for the design and characterization of AAV vectors, the book continues with protocols for AAV delivery to various components of the central nervous system, to a number of sensory systems, and to a broad range of other tissues. Novel techniques such as ultrasound-targeted delivery to the brain, subpial delivery to the spinal cord, and subILM delivery to the retina are accompanied by chapters that provide an overview and comparison of current methods for AAV delivery to tissues such as brain, heart, liver, and lung. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, readily reproducible step-by-step laboratory protocols, and tips for troubleshooting and avoiding known pitfalls. Authoritative and comprehensive, Adeno-Associated Virus Vectors: Design and Delivery aims to enhance the utility of AAV vectors for targeted gene transfer to living animals and continue the ongoing development of novel AAV-based gene therapies for human disease.



AAV Gene Therapy Immunology and Immunotherapeutics

Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma.

Author: Jose Martinez-Navio

Publisher: Frontiers Media SA

ISBN: 2889743063

Category: Medical

Page: 187

View: 440

Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme

Gene Transfer Vectors for Clinical Application

This volume of Methods in Enzymology looks at Gene Transfer Vectors for Clinical Application. The chapters provide an invaluable resource for academics, researchers and students alike.

Author: Theodore Friedmann

Publisher: Academic Press

ISBN: 0123865093

Category: Medical

Page: 450

View: 487

This volume of Methods in Enzymology looks at Gene Transfer Vectors for Clinical Application. The chapters provide an invaluable resource for academics, researchers and students alike. With an international board of authors, this volume covers such topics as General principles of retrovirus vector design, Chronic granulomatous disease (CGD), Gene therapy for blindness, and Retrovirus genetic strategy and vector design. Chapters provide an invaluable resource for academics, researchers and students alike International board of authors This volume covers such topics as general principles of retrovirus vector design, chronic granulomatous disease (CGD), gene therapy for blindness, and retrovirus genetic strategy and vector design

Adenoviruses Model and Vectors in Virus Host Interactions

After three volumes on adenoviruses in 1995 the past years have seen rapid progress in the field of adenovirus research. Moreover, adenoviruses have attracted considerable interest as vectors in gene transfer regimens.

Author: Walter Doerfler

Publisher: Springer

ISBN:

Category: Medical

Page: 476

View: 880

After three volumes on adenoviruses in 1995 the past years have seen rapid progress in the field of adenovirus research. Moreover, adenoviruses have attracted considerable interest as vectors in gene transfer regimens.


Viral Vectors for Treating Diseases of the Nervous System

Gene therapy offers considerable potential for the treatment of various incurable diseases of the nervous system.

Author: David S. Latchman

Publisher: Gulf Professional Publishing

ISBN: 9780123668561

Category: Medical

Page: 360

View: 304

Gene therapy offers considerable potential for the treatment of various incurable diseases of the nervous system. Viral Vectors for Treating Disease of the Nervous System describes a number of different viral vectors developed for achieving high efficiency gene delivery to the brain. Vectors described include those based on adenovirus, adeno-associated virus, Herpes Simplex Virus, lentivirus, and other retroviruses. It also discusses the potential application of such viruses in treating brain tumors, Parkinson's disease, and other diseases of the nervous system. Provides up-to-date account of gene therapy approaches for incurable neurological disorders Describes a range of gene delivery methods based on different viruses

Viral Vectors for Gene Therapy

While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, ...

Author: Otto-Wilhelm Merten

Publisher: Humana Press

ISBN: 9781617790942

Category: Medical

Page: 450

View: 207

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.