Gene And Cell Therapy

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Gene and Cell Therapy: Biology and Applications

Giridhara R. Jayandharan
Gene and Cell Therapy Biology and Applications Book PDF

Author : Giridhara R. Jayandharan
Publisher : Springer
Page : 305 pages
File Size : 43,5 Mb
Release : 2018-09-12
Category : Medical
ISBN : 9789811304811

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Gene and Cell Therapy: Biology and Applications by Giridhara R. Jayandharan Pdf

Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics.

Gene and Cell Therapy

Nancy Smyth Templeton
Gene and Cell Therapy Book PDF

Author : Nancy Smyth Templeton
Publisher : CRC Press
Page : 894 pages
File Size : 42,8 Mb
Release : 2003-12-17
Category : Science
ISBN : 9780824758608

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Gene and Cell Therapy by Nancy Smyth Templeton Pdf

This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field.

A Handbook of Gene and Cell Therapy

Clévio Nóbrega,Liliana Mendonça,Carlos A. Matos
A Handbook of Gene and Cell Therapy Book PDF

Author : Clévio Nóbrega,Liliana Mendonça,Carlos A. Matos
Publisher : Springer Nature
Page : 195 pages
File Size : 43,9 Mb
Release : 2020-06-27
Category : Medical
ISBN : 9783030413330

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A Handbook of Gene and Cell Therapy by Clévio Nóbrega,Liliana Mendonça,Carlos A. Matos Pdf

This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field.

Gene and Cell Therapy

Nancy Smyth Templeton
Gene and Cell Therapy Book PDF

Author : Nancy Smyth Templeton
Publisher : CRC Press
Page : 1154 pages
File Size : 45,5 Mb
Release : 2008-10-06
Category : Science
ISBN : 9780849387999

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Gene and Cell Therapy by Nancy Smyth Templeton Pdf

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.

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 : 45,9 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.

Stem Cell and Gene Therapy for Cardiovascular Disease

Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Stem Cell and Gene Therapy for Cardiovascular Disease Book PDF

Author : Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Publisher : Academic Press
Page : 538 pages
File Size : 40,8 Mb
Release : 2015-08-21
Category : Science
ISBN : 9780128018637

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Stem Cell and Gene Therapy for Cardiovascular Disease by Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson Pdf

Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI Covers the prevailing opinions on how to harness the body’s natural repair mechanisms Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals

Gene and Cell Therapies

Eve Hanna,Mondher Toumi
Gene and Cell Therapies Book PDF

Author : Eve Hanna,Mondher Toumi
Publisher : CRC Press
Page : 168 pages
File Size : 41,5 Mb
Release : 2020-05-19
Category : Medical
ISBN : 9781000068474

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Gene and Cell Therapies by Eve Hanna,Mondher Toumi Pdf

The major advances in the field of biotechnology and molecular biology in the twenty-first century have led to a better understanding of the pathophysiology of diseases. A new generation of biopharmaceuticals has emerged, including a wide and heterogeneous range of innovative cell and gene therapies. These therapies aim to prevent or treat chronic and serious life-threatening diseases, previously considered incurable. This book describes the evolution and adaptation of the regulatory environment to assess these therapies in contrast with the resistance of health technology assessment (HTA) agencies and payers to acknowledge the specificity of cell and gene therapies and the need to adapt existing decision-making frameworks. This book provides insights on the learnings from the experience of current cell and gene therapies (regulatory approval, HTA, and market access), in addition to future trends to enhance patient access to these therapies. Key Features: Describes the potential change of treatment paradigm and the specificity of cell and gene therapies, including the gradual move from repeated treatment administration to one-time single administration with the potential to be definite cure Highlights the challenges at the HTA level Discusses the affordability of future cell and gene therapies and the possible challenges for health insurance systems Provides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of healthcare systems

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 : 41,9 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.

Gene and Cell Therapy

Nancy Smyth Templeton
Gene and Cell Therapy Book PDF

Author : Nancy Smyth Templeton
Publisher : CRC Press
Page : 1337 pages
File Size : 42,5 Mb
Release : 2015-01-20
Category : Science
ISBN : 9781466572003

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Gene and Cell Therapy by Nancy Smyth Templeton Pdf

The Most Comprehensive, State-of-the-Art Book on Using Gene and Cell Therapy in Clinical MedicineGene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. T

Gene Therapy of Cancer

Stanton L. Gerson,Edmund C. Lattime
Gene Therapy of Cancer Book PDF

Author : Stanton L. Gerson,Edmund C. Lattime
Publisher : Elsevier
Page : 534 pages
File Size : 50,5 Mb
Release : 2002-04-04
Category : Science
ISBN : 0080491367

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Gene Therapy of Cancer by Stanton L. Gerson,Edmund C. Lattime Pdf

The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

Molecular and Cellular Therapies for Motor Neuron Diseases

Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante
Molecular and Cellular Therapies for Motor Neuron Diseases Book PDF

Author : Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante
Publisher : Academic Press
Page : 336 pages
File Size : 53,9 Mb
Release : 2017-01-18
Category : Psychology
ISBN : 9780128025246

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Molecular and Cellular Therapies for Motor Neuron Diseases by Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante Pdf

Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians. Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig’s Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials. Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy

Stem Cell Biology and Gene Therapy

Peter J. Quesenberry,Gary S. Stein,Bernard G. Forget,Sherman M. Weissman
Stem Cell Biology and Gene Therapy Book PDF

Author : Peter J. Quesenberry,Gary S. Stein,Bernard G. Forget,Sherman M. Weissman
Publisher : John Wiley & Sons
Page : 586 pages
File Size : 54,7 Mb
Release : 1998-09-10
Category : Science
ISBN : 0471146560

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Stem Cell Biology and Gene Therapy by Peter J. Quesenberry,Gary S. Stein,Bernard G. Forget,Sherman M. Weissman Pdf

STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine.

Emerging Trends in Cell and Gene Therapy

Michael K. Danquah,Ram I. Mahato
Emerging Trends in Cell and Gene Therapy Book PDF

Author : Michael K. Danquah,Ram I. Mahato
Publisher : Springer Science & Business Media
Page : 705 pages
File Size : 54,5 Mb
Release : 2013-06-14
Category : Science
ISBN : 9781627034173

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Emerging Trends in Cell and Gene Therapy by Michael K. Danquah,Ram I. Mahato Pdf

Examples from various organs and diseases illustrate the potential benefit obtained when both therapeutic approaches are combined with delivery strategies. Representing the combined effort of several leading international research and clinical experts, this book, Emerging Trends in Cell and Gene Therapy, provides a complete account on and brings into sharp focus current trends and state-of-the-art in important areas at the interface of cell- and gene-based therapies. This book addresses the current fragmented understanding regarding these two research areas and fills the vast unmet educational need and interest of both students and researchers in academia and industry. Main features of the book: · Biological aspects of stem cell sources, differentiation and engineering. · Application of microfluidics to study stem cell dynamics · Potential clinical application of stem cells and gene therapy to specific human disease. · Utilization of biomaterials and stem cells in regenerative medicine with particular emphasis on spinal cord repair, ligament and bone tissue engineering. · Biomimetic multiscale topography for cell alignment.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

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

Author : Maria Cristina Galli,Mercedes Serabian
Publisher : Springer
Page : 230 pages
File Size : 54,8 Mb
Release : 2015-09-15
Category : Medical
ISBN : 9783319186184

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

This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). 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 for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, 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 and 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.

Cell Therapy

Adrian Gee
Cell Therapy Book PDF

Author : Adrian Gee
Publisher : Springer Science & Business Media
Page : 256 pages
File Size : 52,7 Mb
Release : 2009-09-18
Category : Science
ISBN : 9780387895840

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Cell Therapy by Adrian Gee Pdf

Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professional’s library. Additional topics in Cell Therapy: cGMP Facilities and Manufacturing...Standard operating procedures - Supply management - Facility equipment - Product manufacturing, review, release and administration - Facility master file.