Lentiviral Vectors and Gene Therapy

Lentiviral Vectors and Gene Therapy PDF Author: David Escors
Publisher: Springer Science & Business Media
ISBN: 3034804024
Category : Medical
Languages : en
Pages : 110

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Book Description
Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success. This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials.

Lentiviral Vectors and Gene Therapy

Lentiviral Vectors and Gene Therapy PDF Author: David Escors
Publisher: Springer Science & Business Media
ISBN: 3034804024
Category : Medical
Languages : en
Pages : 110

Get Book Here

Book Description
Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success. This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials.

Lentiviral Vectors

Lentiviral Vectors PDF Author: Didier Trono
Publisher: Springer Science & Business Media
ISBN: 9783540421900
Category : Medical
Languages : en
Pages : 276

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Book Description
For the first time a compilation of chapters that depict the biological bases underlying the development of lentiviral vectors, the techniques involved in the manufacture of this new gene delivery tool, and its most promising applications.

Lentivirus Gene Engineering Protocols

Lentivirus Gene Engineering Protocols PDF Author: Maurizio Federico
Publisher: Springer Science & Business Media
ISBN: 1592593933
Category : Science
Languages : en
Pages : 310

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Book Description
Cell gene engineering is emerging as a field with outstanding impact, not only in medicine/biology, but also, and perhaps most importantly, in agriculture and in all those food sciences involved in the fight against world hunger. Lentivirus vector-based technologies represent the last frontier in the development of powerful and reliable methods for both in vitro and in vivo gene transfer in eukaryotic animal cells. Although the design of lentivirus vectors is closely reminiscent of those already successfully applied to the construction of oncoretroviral vectors, some unique features, e.g., the ef- ciency in transducing both postmitotic and stem cells, render the use of lentivirus vectors invaluable. It has been a great pleasure to edit Lentivirus Gene Engineering Pro- cols, owing in part to the high level of enthusiasm that the authors dem- strated in contributing to this book. The fact that so many outstanding scientists engaged in lentivirus vector research have provided articles renders it so- thing more than a technical handbook. In addition to detailed descriptions of the most innovative methodologies, the reader may find very informative ov- views concerning both theoretical and practical aspects of the origin and the development of diverse lentivirus vector types. This, in my opinion, rep- sents a unique added value of this volume, which should help our work resist the passage of time, to which books such as this are particularly sensitive.

Viral Gene Therapy

Viral Gene Therapy PDF Author: Ke Xu
Publisher:
ISBN: 9789535164388
Category :
Languages : en
Pages : 464

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Book Description
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.

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders PDF Author: Nicola Brunetti-Pierri
Publisher: Springer
ISBN: 3319534572
Category : Medical
Languages : en
Pages : 222

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Book Description
In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.

Viral Vectors for Gene Therapy

Viral Vectors for Gene Therapy PDF 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.

Challenges in Delivery of Therapeutic Genomics and Proteomics

Challenges in Delivery of Therapeutic Genomics and Proteomics PDF Author: Ambikanandan Misra
Publisher: Elsevier
ISBN: 0123849659
Category : Science
Languages : en
Pages : 686

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Book Description
Delivery of therapeutic proteomics and genomics represent an important area of drug delivery research. Genomics and proteomics approaches could be used to direct drug development processes by unearthing pathways involved in disease pathogenesis where intervention may be most successful. This book describes the basics of genomics and proteomics and highlights the various chemical, physical and biological approaches to protein and gene delivery. - Covers a diverse array of topics from basic sciences to therapeutic applications of proteomics and genomics delivery - Of interest to researchers in both academia and industry - Highlights what's currently known and where further research is needed

Chimeric Antigen Receptor T Cells

Chimeric Antigen Receptor T Cells PDF Author: Kamilla Swiech
Publisher:
ISBN: 9781071601464
Category : Antigens
Languages : en
Pages :

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Book Description


Gene and Cell Therapy

Gene and Cell Therapy PDF Author: Nancy Smyth Templeton
Publisher: CRC Press
ISBN: 0824758609
Category : Science
Languages : en
Pages : 894

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Book Description
This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field.

Viral Vectors for Gene Therapy

Viral Vectors for Gene Therapy PDF Author: Otto-Wilhelm Merten
Publisher: Humana
ISBN: 9781617790942
Category : Medical
Languages : en
Pages : 0

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Book Description
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.