Advanced Gene Delivery

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Advanced Gene Delivery

Alain Rolland
Advanced Gene Delivery Book PDF

Author : Alain Rolland
Publisher : CRC Press
Page : 300 pages
File Size : 48,8 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.

Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies

Daniel Scherman
Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies Book PDF

Author : Daniel Scherman
Publisher : World Scientific
Page : 556 pages
File Size : 52,6 Mb
Release : 2013-12-10
Category : Science
ISBN : 9781783263141

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Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies by Daniel Scherman Pdf

This unique advanced textbook provides a clear and comprehensive description of the field 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 use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable 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. Furthermore, the textbook'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.

Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies (Second Edition)

Daniel Scherman
Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies Second Edition Book PDF

Author : Daniel Scherman
Publisher : World Scientific
Page : 634 pages
File Size : 53,8 Mb
Release : 2019-07-16
Category : Science
ISBN : 9781786346896

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Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies (Second Edition) by Daniel Scherman Pdf

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.

Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology

Daniel Scherman
Advanced Textbook on Gene Transfer Gene Therapy and Genetic Pharmacology Book PDF

Author : Daniel Scherman
Publisher : Unknown
Page : 617 pages
File Size : 52,5 Mb
Release : 2019
Category : Electronic books
ISBN : 1786346885

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Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology by Daniel Scherman Pdf

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.

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 : 46,8 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.-

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 : 41,8 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

Pharmaceutical Biotechnology

Daan J. A. Crommelin,Robert D. Sindelar
Pharmaceutical Biotechnology Book PDF

Author : Daan J. A. Crommelin,Robert D. Sindelar
Publisher : CRC Press
Page : 456 pages
File Size : 53,7 Mb
Release : 2002-11-14
Category : Medical
ISBN : 0415285011

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Pharmaceutical Biotechnology by Daan J. A. Crommelin,Robert D. Sindelar Pdf

The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Advanced Drug Delivery Systems in the Management of Cancer

Kamal Dua,Meenu Mehta,Terezinha de Jesus Andreoli Pinto,Lisa G. Pont,Kylie A. Williams,Michael Rathbone
Advanced Drug Delivery Systems in the Management of Cancer Book PDF

Author : Kamal Dua,Meenu Mehta,Terezinha de Jesus Andreoli Pinto,Lisa G. Pont,Kylie A. Williams,Michael Rathbone
Publisher : Elsevier
Page : 572 pages
File Size : 47,5 Mb
Release : 2021-06-24
Category : Business & Economics
ISBN : 9780323900799

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Advanced Drug Delivery Systems in the Management of Cancer by Kamal Dua,Meenu Mehta,Terezinha de Jesus Andreoli Pinto,Lisa G. Pont,Kylie A. Williams,Michael Rathbone Pdf

Advanced Drug Delivery Systems in the Management of Cancer discusses recent developments in nanomedicine and nano-based drug delivery systems used in the treatment of cancers affecting the blood, lungs, brain, and kidneys. The research presented in this book includes international collaborations in the area of novel drug delivery for the treatment of cancer. Cancer therapy remains one of the greatest challenges in modern medicine, as successful treatment requires the elimination of malignant cells that are closely related to normal cells within the body. Advanced drug delivery systems are carriers for a wide range of pharmacotherapies used in many applications, including cancer treatment. The use of such carrier systems in cancer treatment is growing rapidly as they help overcome the limitations associated with conventional drug delivery systems. Some of the conventional limitations that these advanced drug delivery systems help overcome include nonspecific targeting, systemic toxicity, poor oral bioavailability, reduced efficacy, and low therapeutic index. This book begins with a brief introduction to cancer biology. This is followed by an overview of the current landscape in pharmacotherapy for the cancer management. The need for advanced drug delivery systems in oncology and cancer treatment is established, and the systems that can be used for several specific cancers are discussed. Several chapters of the book are devoted to discussing the latest technologies and advances in nanotechnology. These include practical solutions on how to design a more effective nanocarrier for the drugs used in cancer therapeutics. Each chapter is written with the goal of informing readers about the latest advancements in drug delivery system technologies while reinforcing understanding through various detailed tables, figures, and illustrations. Advanced Drug Delivery Systems in the Management of Cancer is a valuable resource for anyone working in the fields of cancer biology and drug delivery, whether in academia, research, or industry. The book will be especially useful for researchers in drug formulation and drug delivery as well as for biological and translational researchers working in the field of cancer. Presents an overview of the recent perspectives and challenges within the management and diagnosis of cancer Provides insights into how advanced drug delivery systems can effectively be used in the management of a wide range of cancers Includes up-to-date information on diagnostic methods and treatment strategies using controlled drug delivery systems

Nanopharmaceutical Advanced Delivery Systems

Vivek Dave,Nikita Gupta,Srija Sur
Nanopharmaceutical Advanced Delivery Systems Book PDF

Author : Vivek Dave,Nikita Gupta,Srija Sur
Publisher : John Wiley & Sons
Page : 467 pages
File Size : 54,5 Mb
Release : 2020-12-29
Category : Computers
ISBN : 9781119711681

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Nanopharmaceutical Advanced Delivery Systems by Vivek Dave,Nikita Gupta,Srija Sur Pdf

The book provides a single volume covering detailed descriptions about various delivery systems, their principles and how these are put in use for the treatment of multiple diseases. It is divided into four sections where the first section deals with the introduction and importance of novel drug delivery system. The second section deals with the most advanced drug delivery systems like microbubbles, dendrimers, lipid-based nanoparticles, nanofibers, microemulsions etc., describing the major principles and techniques of the preparations of the drug delivery systems. The third section elaborates on the treatments of diverse diseases like cancer, topical diseases, tuberculosis etc. The fourth and final section provides a brief informative description about the regulatory aspects of novel drug delivery system that is followed in various countries.

Oversight and Review of Clinical Gene Transfer Protocols

Institute of Medicine,Board on Health Sciences Policy,Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee
Oversight and Review of Clinical Gene Transfer Protocols Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee
Publisher : National Academies Press
Page : 78 pages
File Size : 48,7 Mb
Release : 2014-03-27
Category : Medical
ISBN : 9780309296656

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Oversight and Review of Clinical Gene Transfer Protocols by Institute of Medicine,Board on Health Sciences Policy,Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee Pdf

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.

Electrospun Materials and Their Allied Applications

Inamuddin,Rajender Boddula,Mohd Imran Ahamed,Abdullah M. Asiri
Electrospun Materials and Their Allied Applications Book PDF

Author : Inamuddin,Rajender Boddula,Mohd Imran Ahamed,Abdullah M. Asiri
Publisher : John Wiley & Sons
Page : 544 pages
File Size : 53,8 Mb
Release : 2020-05-27
Category : Technology & Engineering
ISBN : 9781119654865

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Electrospun Materials and Their Allied Applications by Inamuddin,Rajender Boddula,Mohd Imran Ahamed,Abdullah M. Asiri Pdf

The aim of this book is to explore the history, fundamentals, manufacturing processes, optimization parameters, and applications of electrospun materials. The book includes various types of electrospun materials such as antimicrobial, smart, bioinspired systems. It focuses on the many application areas for electrospun materials such as energy storage and harvesting, catalysis, biomedical including gene delivery and tissue engineering, separation, adsorption and water treatment technologies, packaging. The book emphasizes the enhanced sustainable properties of electrospun materials, with the challenges and future developments being discussed in detail. The chapters are written by top-class researchers and experts from throughout the world.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine
Exploring Novel Clinical Trial Designs for Gene Based Therapies Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine
Publisher : National Academies Press
Page : 127 pages
File Size : 41,7 Mb
Release : 2020-08-27
Category : Medical
ISBN : 9780309672986

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Exploring Novel Clinical Trial Designs for Gene-Based Therapies by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine Pdf

Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Maria Cristina Galli
Regulatory Aspects of Gene Therapy and Cell Therapy Products Book PDF

Author : Maria Cristina Galli
Publisher : Springer Nature
Page : 243 pages
File Size : 49,9 Mb
Release : 2023-08-01
Category : Science
ISBN : 9783031345678

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Regulatory Aspects of Gene Therapy and Cell Therapy Products by Maria Cristina Galli Pdf

This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Gene Delivery

Vanessa Zimmer
Gene Delivery Book PDF

Author : Vanessa Zimmer
Publisher : Unknown
Page : 0 pages
File Size : 45,9 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.

Immunopharmacology

Manzoor M. Khan
Immunopharmacology Book PDF

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
Page : 275 pages
File Size : 53,9 Mb
Release : 2008-12-19
Category : Medical
ISBN : 9780387779768

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Immunopharmacology by Manzoor M. Khan Pdf

During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.