[PDF] Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide Based Therapies eBook

Author : Daniel Scherman
Publisher : World Scientific
Page : 634 pages
File Size : 46,96 MB
Release : 2019-07-16
Category : Science
ISBN : 1786346893

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This unique advanced textbook provides a clear and comprehensive overview 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 second edition features new groundbreaking material on genome editing using the recently discovered CRISPR/Cas9 system and on cancer immunotherapy by CAR-T cells. It also presents the historical milestone of gene therapy application in the field of severe combined immunodeficiency, and other fields of gene therapy and molecular medicine.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 gene therapy, which is already revolutionizing medicine. In this advanced textbook, more than 30 leading scientists come together to explore these topics.This educational introduction provides the background material needed to further explore the subject as well as relevant research literature. It is an invaluable resource 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. The second edition'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.

Author : Daniel Scherman
Publisher :
Page : 617 pages
File Size : 15,2 MB
Release : 2019
Category : Electronic books
ISBN : 9781786346889

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This unique advanced textbook provides a clear and comprehensive overview 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 second edition features new groundbreaking material on genome editing using the recently discovered CRISPR/Cas9 system and on cancer immunotherapy by CAR-T cells. It also presents the historical milestone of gene therapy application in the field of severe combined immunodeficiency, and other fields of gene therapy and molecular medicine. 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 gene therapy, which is already revolutionizing medicine. In this advanced textbook, more than 30 leading scientists come together to explore these topics. This educational introduction provides the background material needed to further explore the subject as well as relevant research literature. It is an invaluable resource 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. The second edition'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.

Author : Alain Rolland
Publisher : CRC Press
Page : 300 pages
File Size : 44,7 MB
Release : 2003-09-02
Category : Science
ISBN : 0203303814

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A practical resource for everyone involved in the gene therapy field and in the design of effective gene delivery systems, this volume presents an overview and update of recent advances in the field of non-viral methods for the in vivo transfer of therapeutic genes to biological targets using conventional routes of administration. Methods to control the spatial and temporal modulation of gene function in vivo as well as the level, duration, specificity, and fidelity of gene expression are described. The rational design and the applications of a variety of non-viral gene delivery systems, such as cationic lipid-, polymer-, and (poly) peptide-based systems, are exemplified for the control of location of therapeutic genes administered by various routes. Current and potential clinical applications of gene-based medicines are presented for the prevention, correction or modulation of diseases. Examples of current applications of plasmid-based systems for genetic vaccination, treatment of genetic disorders such as cystic fibrosis, and treatment of acquired diseases such as cancer are also provided.

Author : Mansoor M. Amiji
Publisher : CRC Press
Page : 707 pages
File Size : 24,78 MB
Release : 2004-09-29
Category : Medical
ISBN : 0203500474

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To treat disease or correct genetic disorders using gene therapy, the most suitable vehicle must be able to deliver genes to the appropriate tissues and cells in the body in a specific as well as safe and effective manner. While viruses are the most popular vehicles to date, their disadvantages include toxicity, limited size of genes they can carry

Author : G. Subramanian
Publisher : Springer Science & Business Media
Page : 355 pages
File Size : 41,91 MB
Release : 2012-12-06
Category : Medical
ISBN : 1461513537

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Advances in molecular biology and recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches and methods are readily available for gene cloning and therapeutic vector construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus gene therapy has already shown that it holds great promise for the treatment of many diseases and disorders. In general it involves the delivery of recombinant genes or transgenes into somatic cells to replace proteins with a genetic defect or to transfer with the pathological process of an illness. The viral and non-viral delivery systems may hold the potential for future non-invasive, cost-effective oral therapy of genetically-based disorders. Recent years have seen considerable progress in the discovery and early clinical development of a variety of gene therapeutic products. The availability, validation, and implementation of gene therapeutic products has also enabled success in testing and evaluation. New challenges will need to be overcome to ensure that products will also be successful in later clinical development and ultimately for marketing authorisation. These new challenges will include improvements in delivery systems, better control of in-vivo targeting, increased level transduction and duration of expression of the gene, and manufacturing process efficiencies that enable reduction in production costs. Perhaps profound understanding of regulated gene design may result in innovative bioproducts exhibiting safety and efficacy profiles that are significantly superior to those achieved by the use of naturally occurring genes. This procedure may contribute considerably to fulfilling standards set by regulatory authorities. This book provides an overview of the current advances in the field of gene therapy and the methods that are being successfully applied in the manufacture of gene therapeutic products, and hopefully will stimulate further progress and advancement in this field to meet the ever-increasing demands.

Author : Alain Rolland
Publisher : CRC Press
Page : 442 pages
File Size : 19,35 MB
Release : 2003-04-11
Category : Medical
ISBN : 0824748581

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This volume examines the advantages and limitations of the major gene delivery systems and offers guidelines to select the most appropriate viral or synthetic delivery system for specific therapeutic applications. It discusses advances in the design, optimization, and adaptation of gene delivery systems for the treatment of cancerous, cardiovascular, pulmonary, genetic, and infectious diseases.

Author : Doaa Hashad
Publisher : BoD – Books on Demand
Page : 220 pages
File Size : 11,13 MB
Release : 2015-11-26
Category : Medical
ISBN : 9535122215

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Gene therapy is becoming a promising technology for the management of many human diseases. Hereditary and acquired disorders can both be tackled using the technique of gene therapy. This book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology. The development of new and efficient gene transfer vectors is of utmost importance in the progress of the field of gene therapy. Both viral and non-viral vectors are extensively discussed. A detailed chapter elaborates the problem of host immune rejection of transplanted donor cells or engineered tissue that can be avoided using the encapsulation of transgenic cells, thus avoiding the use of drugs that achieve immunosuppression.

Author : Blankenstein
Publisher : Birkhäuser
Page : 374 pages
File Size : 22,14 MB
Release : 2012-12-06
Category : Science
ISBN : 3034870116

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K. Sikora Gene therapy is one of the fastest developing areas in modern medical research. Transcending the classical preclinical and clinical disciplines, it is likely to have far reaching consequences in the practice of medicine, as we enter the next millennium. Currently, there are over 200 seperate active clinical trials with over 2,500 patients entered. These studies involve over 20 countries and include patients with a wide range of diseases, including cancer, HIV infection; cystic fibrosis (CF), haemophilia, diabetes, immune deficiencies, metabolic disorders, ischaemic heart disease and arthritis. Gene therapy can be defined as the deliberate transfer of DNA for therapeutic purposes. There is a further implication that only specific sequences containing rel evant genetic information are used; otherwise, transplantation procedures involving bone marrow, kidney or liver could be considered a form of gene therapy. The con cept of transfer of genetic information as a practical clinical tool arose from the gene-cloning technology, developed during the 1970s. Without the ability to isolate and replicate defined genetic sequences, it would be impossible to produce purified material for clinical use. The drive for the practical application of this technology came from the biotechnology industry with its quest for complex human biomole cules produced by recombinant techniques in bacteria. Within a decade, pharma ceutical-grade insulin, interferon, interleukin 2 and tumour necrosis factor were all involved in clinical trials. The next step was to obtain gene expression in vivo.

Author : Roland W Herzog
Publisher : World Scientific
Page : 415 pages
File Size : 32,21 MB
Release : 2010-06-18
Category : Science
ISBN : 9814467103

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Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy — though still in its infancy — remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ('vector') to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession.This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system. Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Author : Jon A. Wolff
Publisher : Springer Science & Business Media
Page : 431 pages
File Size : 47,82 MB
Release : 2012-12-06
Category : Medical
ISBN : 1468468227

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During the first half century of genetics, coinciding with the first half of this cen tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex tremely efficient in transferring their contents to the chromosomes of cultured cells.