Gene Delivery Systems

Gene Delivery Systems PDF Author: Yashwant Pathak
Publisher: CRC Press
ISBN: 1000580318
Category : Medical
Languages : en
Pages : 307

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Book Description
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

Gene Delivery Systems

Gene Delivery Systems PDF Author: Yashwant Pathak
Publisher: CRC Press
ISBN: 1000580318
Category : Medical
Languages : en
Pages : 307

Get Book Here

Book Description
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

Polymers and Nanomaterials for Gene Therapy

Polymers and Nanomaterials for Gene Therapy PDF Author: Ravin Narain
Publisher: Woodhead Publishing
ISBN: 0081005210
Category : Science
Languages : en
Pages : 303

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Book Description
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

Liposomes in Gene Delivery

Liposomes in Gene Delivery PDF Author: Danilo D. Lasic
Publisher: CRC Press
ISBN: 9780849331091
Category : Medical
Languages : en
Pages : 326

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Book Description
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.

Holland-Frei Cancer Medicine

Holland-Frei Cancer Medicine PDF Author: Robert C. Bast, Jr.
Publisher: John Wiley & Sons
ISBN: 111900084X
Category : Medical
Languages : en
Pages : 2004

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Book Description
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

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

Gene Delivery to Mammalian Cells

Gene Delivery to Mammalian Cells PDF Author: William C. Heiser
Publisher: Springer Science & Business Media
ISBN: 1592596509
Category : Science
Languages : en
Pages : 561

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Book Description
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.

Oversight and Review of Clinical Gene Transfer Protocols

Oversight and Review of Clinical Gene Transfer Protocols PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 030929665X
Category : Medical
Languages : en
Pages : 78

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Book Description
Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

Polymeric Gene Delivery

Polymeric Gene Delivery PDF Author: Mansoor M. Amiji
Publisher: CRC Press
ISBN: 0203500474
Category : Medical
Languages : en
Pages : 707

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Book Description
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

Gene Therapy and Gene Delivery Systems

Gene Therapy and Gene Delivery Systems PDF Author: David V. Schaffer
Publisher: Springer Science & Business Media
ISBN: 9783540284048
Category : Medical
Languages : en
Pages : 304

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Book Description
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

Nonviral Vectors for Gene Therapy PDF Author: Mark A. Findeis
Publisher: Springer Science & Business Media
ISBN: 1592591396
Category : Science
Languages : en
Pages : 399

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Book Description
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.