Gene Delivery

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Gene Delivery

Vanessa Zimmer
Gene Delivery Book PDF

Author : Vanessa Zimmer
Publisher : Unknown
Page : 0 pages
File Size : 52,5 Mb
Release : 2019
Category : Gene therapy
ISBN : 153616268X

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Gene Delivery by Vanessa Zimmer Pdf

Gene Delivery: Methods and Applications provides a comprehensive overview on viral and non-viral methods used to genetically engineer human mesenchymal stromal cells. In addition, an update on ongoing and completed clinical studies with engineered mesenchymal stromal cells will be provided, as well as a snapshot of the advances and technical challenges yet to be addressed.Next, a variety of gene delivery systems including physical transfection techniques, virus-based delivery vectors, chemically engineered delivery systems and bio-inspired vehicles are reviewed and their strengths, shortcomings and biomedical applications are discussed.Selfish DNA called transposons capable of cutting out and pasting into the host genome are active throughout the phylogenetic kingdoms. Researchers have repurposed natural transposons for use in delivering a gene-of-interest, enabling for the study of a large and growing list of preclinical gene therapy applications. As such, the authors discuss the past achievements and future challenges of this early-stage technology.The closing chapter introduces cell-penetrating peptides as an efficient tool for DNA transfection. HR9, a designed cell-penetrating peptides, containing nona-arginine flanked by cysteine and penta-histidine displayed a high penetrating ability in mammalian cells.

Gene Delivery Systems

Yashwant Pathak
Gene Delivery Systems Book PDF

Author : Yashwant Pathak
Publisher : CRC Press
Page : 317 pages
File Size : 54,9 Mb
Release : 2022-07-01
Category : Medical
ISBN : 9781000580297

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Gene Delivery Systems by Yashwant Pathak Pdf

This unique volume in our Drugs and Pharmaceutical Sciences series covers the development of gene therapy today, the technology involved, clinical applications of siRNA, non-viral vector-based mRNA delivery using nanotechnology, and RNA based vaccines for treating the infectious diseases. It also presents the current application of the CRISPR/Cas9 gene-editing technique which has revolutionized genome editing and which was awarded the 2020 Nobel Prize in Chemistry. Several new drug delivery systems are explored for the applications of gene therapy. These are found to be useful in treating chronic illnesses, including cancer and infectious diseases. Key Features: Overview of the development of gene therapy Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing Presents CRISPR gene therapy recent trends and applications Discusses siRNA, mRNA, and DNA plasmids

Non-viral Gene Therapy

Kazunari Taira,Kazunori Kataoka,Takuro Niidome
Non viral Gene Therapy Book PDF

Author : Kazunari Taira,Kazunori Kataoka,Takuro Niidome
Publisher : Springer Science & Business Media
Page : 494 pages
File Size : 49,8 Mb
Release : 2006-03-20
Category : Science
ISBN : 9784431278795

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Non-viral Gene Therapy by Kazunari Taira,Kazunori Kataoka,Takuro Niidome Pdf

Presents information on non-viral gene-delivery techniques, covering a spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. This work is useful to researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.

Liposomes in Gene Delivery

Danilo D. Lasic
Liposomes in Gene Delivery Book PDF

Author : Danilo D. Lasic
Publisher : CRC Press
Page : 326 pages
File Size : 49,7 Mb
Release : 1997-03-13
Category : Medical
ISBN : 0849331099

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Liposomes in Gene Delivery by Danilo D. Lasic Pdf

Many specialists are not familiar with both drug delivery and the molecular biology of DNA vectors. Liposomes in Gene Delivery covers both-molecular biologists will gain a basic knowledge of lipids, liposomes, and other gene delivery vehicles; lipid and drug delivery scientists will better understand DNA, molecular biology, and DNA manipulation. Topics include an introduction to nucleic acids, a theoretical description of DNA, recombinant technology, lipids and liposomes, stability and interaction properties of lipids and liposomes, complexation of lipids and liposomes with DNA plasmids, gene expression of genosomes in various models, structure-activity relationships, and transfection models. This is an excellent introductory text for graduate students, scientists, and researchers in molecular and cell biology, genetics, biochemistry, physical chemistry, colloid science, pharmacology, molecular science, and medicine.

Polymers and Nanomaterials for Gene Therapy

Ravin Narain
Polymers and Nanomaterials for Gene Therapy Book PDF

Author : Ravin Narain
Publisher : Woodhead Publishing
Page : 302 pages
File Size : 51,7 Mb
Release : 2016-01-09
Category : Science
ISBN : 9780081005217

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Polymers and Nanomaterials for Gene Therapy by Ravin Narain Pdf

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. Explores current challenges in the research of genetic diseases Discusses polymers for gene therapy and their function in designing advanced materials Provides examples of organic and inorganic nanomaterials for gene therapy Includes labeling, targeting, and assays Looks at characterization, physico-(bio)chemical properties, and applications

Polymeric Gene Delivery

Mansoor M. Amiji
Polymeric Gene Delivery Book PDF

Author : Mansoor M. Amiji
Publisher : CRC Press
Page : 707 pages
File Size : 40,8 Mb
Release : 2004-09-29
Category : Medical
ISBN : 9780203500477

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Polymeric Gene Delivery by Mansoor M. Amiji Pdf

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

Challenges in Delivery of Therapeutic Genomics and Proteomics

Ambikanandan Misra
Challenges in Delivery of Therapeutic Genomics and Proteomics Book PDF

Author : Ambikanandan Misra
Publisher : Elsevier
Page : 686 pages
File Size : 51,6 Mb
Release : 2010-09-09
Category : Science
ISBN : 9780123849656

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Challenges in Delivery of Therapeutic Genomics and Proteomics by Ambikanandan Misra Pdf

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

Advanced Gene Delivery

Alain Rolland
Advanced Gene Delivery Book PDF

Author : Alain Rolland
Publisher : CRC Press
Page : 300 pages
File Size : 50,6 Mb
Release : 2003-09-02
Category : Science
ISBN : 9780203303818

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Advanced Gene Delivery by Alain Rolland Pdf

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.

Gene Delivery to Mammalian Cells

William C. Heiser
Gene Delivery to Mammalian Cells Book PDF

Author : William C. Heiser
Publisher : Springer Science & Business Media
Page : 561 pages
File Size : 47,7 Mb
Release : 2008-02-02
Category : Science
ISBN : 9781592596508

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Gene Delivery to Mammalian Cells by William C. Heiser Pdf

The efficiency of delivering DNA into mammalian cells has increased t- mendously since DEAE dextran was first shown to be capable of enhancing transfer of RNA into mammalian cells in culture. Not only have other chemical methods been developed and refined, but also very efficient physical and viral delivery methods have been established. The technique of introducing DNA into cells has developed from transfecting tissue culture cells to delivering DNA to specific cell types and organs in vivo. Moreover, two important areas of biology—assessment of gene function and gene therapy—require succe- ful DNA delivery to cells, driving the practical need to increase the efficiency and efficacy of gene transfer both in vitro and in vivo. TM These two volumes of the Methods in Molecular Biology series, Gene Del- ery to Mammalian Cells, are designed as a compendium of those techniques that have proven most useful in the expanding field of gene transfer in mammalian cells. It is intended that these volumes will provide a thorough background on chemical, physical, and viral methods of gene delivery, a synopsis of the myriad techniques currently available to introduce genes into mammalian cells, as well as a practical guide on how to accomplish this. It is my expectation that it will be useful to the novice in the field as well as to the scientist with expertise in gene delivery.

Pharmaceutical Gene Delivery Systems

Alain Rolland
Pharmaceutical Gene Delivery Systems Book PDF

Author : Alain Rolland
Publisher : CRC Press
Page : 443 pages
File Size : 49,7 Mb
Release : 2003-04-11
Category : Medical
ISBN : 9780203912331

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Pharmaceutical Gene Delivery Systems by Alain Rolland Pdf

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, cardiovascula

Translating Gene Therapy to the Clinic

Jeffrey Laurence,Michael Franklin
Translating Gene Therapy to the Clinic Book PDF

Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Page : 347 pages
File Size : 48,9 Mb
Release : 2014-11-14
Category : Science
ISBN : 9780128005644

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Translating Gene Therapy to the Clinic by Jeffrey Laurence,Michael Franklin Pdf

Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials

Gene Therapy

Mauro Giacca
Gene Therapy Book PDF

Author : Mauro Giacca
Publisher : Springer Science & Business Media
Page : 318 pages
File Size : 47,5 Mb
Release : 2010-11-01
Category : Medical
ISBN : 9788847016439

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Gene Therapy by Mauro Giacca Pdf

I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided.

Gene Therapy and Gene Delivery Systems

David V. Schaffer,Weichang Zhou
Gene Therapy and Gene Delivery Systems Book PDF

Author : David V. Schaffer,Weichang Zhou
Publisher : Springer Science & Business Media
Page : 304 pages
File Size : 45,6 Mb
Release : 2005-12-20
Category : Medical
ISBN : 3540284044

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Gene Therapy and Gene Delivery Systems by David V. Schaffer,Weichang Zhou Pdf

1 D.V. Schaffer, W. Zhou: Gene Therapy as Future Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- 3 M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From Design to Applications.- 4 S.R. Little, R. Langer: Non-Viral Delivery of Cancer Genetic Vaccines.- 5 J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications.- 6 J.H. Yu, D.V. Schaffer: Advanced Targeting Strategies for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- 8 N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: Production and Formulation of Adenovirus Vectors.-

Nonviral Vectors for Gene Therapy

Mark A. Findeis
Nonviral Vectors for Gene Therapy Book PDF

Author : Mark A. Findeis
Publisher : Springer Science & Business Media
Page : 399 pages
File Size : 51,7 Mb
Release : 2008-02-02
Category : Science
ISBN : 9781592591398

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Nonviral Vectors for Gene Therapy by Mark A. Findeis Pdf

The purpose of this volume of Methods in Molecular Medicine is to set forth examples of the great variety of techniques and applications that are now emerging in the field of nonviral gene therapy. The book emphasizes not only specific approaches to gene delivery but, in particular, the best current me- ods to prepare, handle, and characterize gene delivery agents. These topics are of very broad importance since gene therapy evolves from its mostly ac- emy-based experimental and clinical research to the ever increasing number of industry-driven programs directed toward commercial development. S- cessful introduction of nonviral gene therapy agents into the clinic should be expected to require rigorous manufacturing and analytical methods that readily meet the regulatory guidelines under which new drug candidates are reviewed for marketing approval. Exactly what those guidelines will prove to be c- tainly depends on the established guidelines for review of both biological and chemical therapeutics. Additionally, many new techniques are being devised and applied to gene therapy research; these techniques will be instrumental in developing and characterizing successful gene delivery agents. Nonviral Vectors for Gene Therapy: Methods and Protocols has two main sections. To start with, there is a series of chapters on specific protocols for the synthesis, characterization, and application of gene delivery agents. S- eral chapters address the topic of materials to bind with DNA to form the compact condensed phases that facilitate cellular delivery.

Gene Delivery to Mammalian Cells

William C. Heiser
Gene Delivery to Mammalian Cells Book PDF

Author : William C. Heiser
Publisher : Humana Press
Page : 301 pages
File Size : 48,9 Mb
Release : 2003-10-28
Category : Science
ISBN : 1588290867

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Gene Delivery to Mammalian Cells by William C. Heiser Pdf

Highly experienced researchers describe in step-by-step detail methods that have proven most useful in delivering genes to mammalian cells. Volume 1: Nonviral Gene Transfer Techniques focuses on gene delivery by a variety of chemical and physical methods, including ultrasound, biolistics, peptides, PNA clamps, liposomes, microinjection, electroporation, particle bombardment, dendrimers, and hydrodynamics. An accompanying volume, Volume 2: Viral Gene Transfer Techniques, details procedures for delivering genes to cells in vitro and in vivo, including the use of lentiviral vectors.