Gene and Cell Therapy

Author: Nancy Smyth Templeton
Publisher: CRC Press
ISBN: 9780849387999
Format: PDF
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Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Advanced Textbook on Gene Transfer Gene Therapy and Genetic Pharmacology

Author: Daniel Scherman
Publisher: World Scientific
ISBN: 1783263148
Format: PDF, Mobi
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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. Contents:Basic Definitions and Principles:Introduction (Thierry VandenDriessche)Basic Definitions and General Principles (Daniel Scherman)History of Gene Therapy (Serge Braun)Genetic Pharmacology Using Synthetic Deoxyribonucleotides (Jean-Christophe François and Carine Giovannangeli)Principles of RNAi Trigger Expression for Gene Therapy (Lisa J Scherer and John J Rossi)On Demand Alternative Splicing for Gene Rescue (Stéphanie Lorain and Luis Garcia)Nuclease-Mediated Targeted Genetic Correction (Dieter C Gruenert, Hamid Emamekhoo and R Geoffrey Sargent)Vectors and Gene Delivery Techniques:γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy (Caroline Duros and Odile Cohen-Haguenauer)Adenovirus Vectors (Stefan Kochanek)Adeno-Associated Virus (AAV) Vectors (Aurélie Ploquin, Hildegard Büning and Anna Salvetti)Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination (Matias E Melendez, Aldo Pourchet, Anna Greco and Alberto L Epstein)Non-Viral DNA Vectors (Martin Schleef)Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery (Mark Ericson, Kevin Rice and Guy Zuber)Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery (Virginie Escriou, Nathalie Mignet and Andrew Miller)Hydrodynamic-Pressure-Based Non-Viral Nucleic Acid Delivery (Takeshi Suda, Kenya Kamimura, Guisheng Zhang and Dexi Liu)Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery (Pascal Bigey, Richard Heller and Daniel Scherman)Imaging of Gene Delivery (Georges Vassaux, Peggy Richard-Fiardo, Béatrice Cambien and Philippe Franken)Therapeutic Applications:Oncolytic Adenoviruses for Cancer Gene Therapy (Gunnel Hallden, Yaohe Wang, Han-Hsi Wong and Nick R Lemoine)Progress in DNA Vaccine Approaches for Cancer Immunotherapy (Geoffrey D Hannigan and David B Weiner)Gene Therapy of the Beta-Hemoglobinopathies (Emmanuel Payen, Charlotte Colomb, Olivier Negre, Marina Cavazzana-Calvo, Salima Hacein-Bey-Abina, Yves Beuzard and Philippe Leboulch)Gene Therapy for Hemophilia A and B (Nisha Nair, Marinee Chuah and Thierry VandenDriessche)Experimental and Clinical Ocular Gene Therapy (Alexis-Pierre Bemelmans and José-Alain Sahel)Gene Therapy of Neurological Diseases (Lisa M Stanek, Lamya S Shihabuddin and Seng H Cheng)Genetic Therapy of Muscle Diseases: Duchenne Muscular Dystrophy (Takis Athanasopoulos, Susan Jarmin, Helen Foster, Keith Foster, Jagjeet Kang, Taeyoung Koo, Alberto Malerba, Linda Popplewell, Daniel Scherman and George Dickson)Induced Pluripotent Stem Cells and Gene Targeting for Regenerative Medicine (Jizhong Zou and Linzhao Cheng)Gene Vector Production:Production and Purification of Viral Vectors and Safety Considerations Related to Their Use (Otto-Wilhelm Merten, Matthias Schweizer, Parminder Chahal and Amine Kamen)Production and Purification of Plasmid Vectors (Martin Schleef) Readership: Master, PhD or MD students, post-doctoral scientists or medical doctors, and any scientists using gene transfer techniques or implementing gene therapy strategies. Key Features:The advanced textbook addresses a broader audience of Master degree students than existing literature dedicated to more specialized scientistsThe advanced textbook might be purchased by non-specialists, such as MDs who want to follow the evolution of modern medicineThe global field of nucleotide-based therapies and nucleotide delivery are presented in this comprehensive textbook in simple words, giving easy access to key concepts and technologiesThe textbook also aims at helping non-specialized scientists choose an adequate gene transfer and/or gene therapy strategyKeywords:Gene Transfer;Gene Therapy;Gene Delivery;Genetic Pharmacology;Genetic Diseases;Vectors;Nucleotide;Therapy

Gene Therapy

Author: Nancy Smyth Templeton
Publisher: CRC Press
ISBN:
Format: PDF, Kindle
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This text covers all aspects of gene therapy, including basic principles, viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents.

Multifunctional Nanocarriers for Contemporary Healthcare Applications

Author: Barkat, Md. Abul
Publisher: IGI Global
ISBN: 1522547827
Format: PDF, ePub, Docs
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Advances in technology permeates every aspect of life, including the healthcare system. Nanotechnology based systems have gained popularity based upon their promise, size, and other characteristics. Multifunctional Nanocarriers for Contemporary Healthcare Applications is a critical academic publication that explores advancements in nanostructured systems, applications of these systems in healthcare, and biomedical applications of these systems. Featuring coverage on a wide range of topics, such as hydrogels, controlled drug delivery systems, and nanomedicine, this book is geared toward researchers, students, and academicians seeking current research on advancements and applications of nanostructured systems in the healthcare industry.

A Guide to Human Gene Therapy

Author: Roland W. Herzog
Publisher: World Scientific
ISBN: 9814280917
Format: PDF, ePub
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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.

Jasper s Basic Mechanisms of the Epilepsies

Author: Jeffrey Noebels
Publisher: Oxford University Press
ISBN: 0199842590
Format: PDF, Kindle
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H.H. Jasper, A.A. Ward, A. Pope and H.H. Merritt, chair of the Public Health Service Advisory Committee on the Epilepsies, National Institutes of Health, published the first volume on Basic Mechanisms of the Epilepsies (BME) in 1969. Their ultimate goal was to search for a "better understanding of the epilepsies and seek more rational methods of their prevention and treatment." Since then, basic and clinical researchers in epilepsy have gathered together every decade and a half with these goals in mind -- assessing where epilepsy research has been, what it has accomplished, and where it should go. In 1999, the third volume of BME was named in honor of H.H. Jasper. In line with the enormous expansion in the understanding of basic epilepsy mechanisms over the past four decades, this fourth edition of Jasper's BME is the most ambitious yet. In 90 chapters, the book considers the role of interactions between neurons, synapses, and glia in the initiation, spread and arrest of seizures. It examines mechanisms of excitability, synchronization, seizure susceptibility, and ultimately epileptogenesis. It provides a framework for expanding the epilepsy genome and understanding the complex heredity responsible for common epilepsies as it explores disease mechanisms of ion channelopathies and developmental epilepsy genes. It considers the mechanisms of conditions of epilepsy comorbidities. And, for the first time, this 4th edition describes the current efforts to translate the discoveries in epilepsy disease mechanisms into new therapeutic strategies. This book, considered the 'bible' of basic epilepsy research, is essential for the student, the clinician scientist and all research scientists who conduct laboratory-based experimental epilepsy research using cellular, brain slice and animal models, as well as for those interested in related disciplines of neuronal oscillations, network plasticity, and signaling in brain strucutres that include the cortex, hippocampus, and thalamus. In keeping with the 1969 goals, the book is now of practical importance to the clinical neurologist and epileptologist as the progress of research in molecular genetics and modern efforts to design antiepileptic drugs, cures and repairs in the epilepsies converge and impact clinical care.

Cellular Therapy for Neurological Injury

Author: Charles S., Jr. Cox
Publisher: CRC Press
ISBN: 148222593X
Format: PDF, ePub, Docs
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Cellular Therapy for Neurological Injury discusses the current status of cellular therapy for neurological disorders. The primary areas of focus include traumatic brain injury, stroke (ischemic and hemorrhagic), and spinal cord injury. The book explores cell therapy approaches to these and other conditions, while discussing current advances and a literature review in the context of a field that is moving rapidly. The book presents a translational focus, addressing barriers and opportunities to moving forward. The mechanisms of injury are explored, as well as how these mechanisms influence the type of cell therapy used, the route of delivery, and dosing regimen. Written by leaders in the field, this is an invaluable resource for clinicians and researchers alike.

A Roadmap to Nonhematopoietic Stem Cell Based Therapeutics

Author: Xiao-Dong Chen
Publisher: Academic Press
ISBN: 0128119217
Format: PDF, Mobi
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A Roadmap to Non-hematopoietic Stem Cell-Based Therapeutics: From the Bench to the Clinic is a resource that provides an overview of the principles of stem cell therapy, the promises and challenges of using stem cells for treating various clinical conditions, and future perspectives. The overall goal is to facilitate the translation of basic research on stem cells to clinical applications. The properties of stem cells from various sources are reviewed and the advantages and disadvantages of each for clinical use are discussed. Modifying stem cell properties through preconditioning strategies using physical, chemical, genetic, and molecular manipulation to improve cell survival, increase cell differentiation potential, enhance production of paracrine factors, and facilitate homing to the site of injury or disease upon transplantation are reviewed. Various routes of stem cell administration and dosing, and the duration of effects, are explored. Individual chapters are written by experts in the field and focus on the use of stem cells in treating various degenerative diseases, autoimmune diseases, wound healing, cardiovascular disease, spinal cord injury, oral and dental diseases, and skeletal disorders. Finally, experts in the regulatory arena discuss mechanisms used in different countries for approving the use of stem cells to treat diseases and many common issues that are typically encountered while seeking approval for this class of therapeutic agent. Offers advanced students, as well as new researchers, an overview of the principles of stem cell therapy Discusses a wide array of pressing clinical issues with stem cell-based therapies so that new ideas in the laboratory can be efficiently translated to the clinic through better designed clinical trials Helps clarify current regulatory mechanisms so that the safe use of stem cells for treating a variety of diseases can move forward Fosters cross-disciplinary dialogue between research scientists and physicians to accelerate the safe implementation of efficacious cell therapies

Molecular Biology of Cancer

Author: Lauren Pecorino
Publisher: Oxford University Press
ISBN: 019957717X
Format: PDF, Mobi
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The third edition of The Molecular Biology of Cancer: Mechanisms, Targets, and Therapeutics offers a fresh approach to the study of the molecular basis of cancer, by showing how our understanding of the defective mechanisms which drive cancer is leading to the development of new targeted therapeutic agents.

Principles of Tissue Engineering

Author: Robert Lanza
Publisher: Academic Press
ISBN: 0123983703
Format: PDF, ePub, Mobi
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Now in its fourth edition, Principles of Tissue Engineering has been the definite resource in the field of tissue engineering for more than a decade. The fourth edition provides an update on this rapidly progressing field, combining the prerequisites for a general understanding of tissue growth and development, the tools and theoretical information needed to design tissues and organs, as well as a presentation by the world’s experts of what is currently known about each specific organ system. As in previous editions, this book creates a comprehensive work that strikes a balance among the diversity of subjects that are related to tissue engineering, including biology, chemistry, material science, and engineering, among others, while also emphasizing those research areas that are likely to be of clinical value in the future. This edition includes greatly expanded focus on stem cells, including induced pluripotent stem (iPS) cells, stem cell niches, and blood components from stem cells. This research has already produced applications in disease modeling, toxicity testing, drug development, and clinical therapies. This up-to-date coverage of stem cell biology and other emerging technologies –such as brain-machine interfaces for controlling bionics and neuroprostheses– is complemented by a series of new and updated chapters on recent clinical experience in applying tissue engineering, as well as a new section on the application of tissue-engineering techniques for food production. The result is a comprehensive textbook that will be useful to students and experts alike. Includes new chapters on biomaterial-protein interactions, nanocomposite and three-dimensional scaffolds, skin substitutes, spinal cord, vision enhancement, and heart valves Offers expanded coverage of adult and embryonic stem cells of the cardiovascular, hematopoietic, musculoskeletal, nervous, and other organ systems Full-color presentation throughout