Second Generation Cell and Gene Based Therapies

Second Generation Cell and Gene Based Therapies
Author: Alain Vertes,Nathan J. Dowden,Devyn Smith,Nasib Qureshi
Publsiher: Academic Press
Total Pages: 846
Release: 2020-02-07
Genre: Science
ISBN: 9780128120330

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Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

A Handbook of Gene and Cell Therapy

A Handbook of Gene and Cell Therapy
Author: Clévio Nóbrega,Liliana Mendonça,Carlos A. Matos
Publsiher: Springer Nature
Total Pages: 188
Release: 2020-06-27
Genre: Medical
ISBN: 9783030413330

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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.

Cell and Gene Therapies

Cell and Gene Therapies
Author: Miguel-Angel Perales,Syed A. Abutalib,Catherine Bollard
Publsiher: Springer
Total Pages: 288
Release: 2018-11-27
Genre: Medical
ISBN: 9783319543680

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In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

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

Advanced Textbook On Gene Transfer  Gene Therapy And Genetic Pharmacology  Principles  Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies
Author: Daniel Scherman
Publsiher: World Scientific
Total Pages: 556
Release: 2013-12-10
Genre: Science
ISBN: 9781783263141

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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.

Cell Therapy

Cell Therapy
Author: Adrian Gee
Publsiher: Springer Science & Business Media
Total Pages: 256
Release: 2009-09-18
Genre: Science
ISBN: 9780387895840

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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.

Adenoviral Vectors for Gene Therapy

Adenoviral Vectors for Gene Therapy
Author: David T. Curiel
Publsiher: Academic Press
Total Pages: 868
Release: 2016-03-10
Genre: Science
ISBN: 9780128005101

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Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Exploring Novel Clinical Trial Designs for Gene Based Therapies

Exploring Novel Clinical Trial Designs for Gene Based Therapies
Author: National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine
Publsiher: National Academies Press
Total Pages: 126
Release: 2020-08-27
Genre: Medical
ISBN: 9780309672986

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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 Therapies

Gene and Cell Therapies
Author: Eve Hanna,Mondher Toumi
Publsiher: CRC Press
Total Pages: 146
Release: 2020-05-19
Genre: Medical
ISBN: 9781000068474

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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

Somatic Gene Therapy

Somatic Gene Therapy
Author: P.L. Chang
Publsiher: CRC Press
Total Pages: 313
Release: 2018-01-17
Genre: Science
ISBN: 9781351093668

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As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

Advances In Pharmaceutical Cell Therapy Principles Of Cell based Biopharmaceuticals

Advances In Pharmaceutical Cell Therapy  Principles Of Cell based Biopharmaceuticals
Author: Huss Ralf,Guenther Christine,Hauser Andrea Josefine
Publsiher: World Scientific
Total Pages: 564
Release: 2015-09-23
Genre: Medical
ISBN: 9789814616805

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This textbook is a comprehensive overview of the development of cell-based biopharmaceuticals. Beginning with the underlying biology of stem cell and cell-based products, it traces the long and complex journey from preclinical concept to initiation of a pivotal clinical trial and the potential business model behind it.The book also takes into consideration the different regulatory landscapes and their continuous evolution in Europe, North America and other parts of the world. The authors describe a path to manufacture a clinical grade therapeutic that passes all necessary quality measures as a robust and marketable product including an outlook on next generation products and innovative strategies.This reference book is a must-have guide for any professional already active in biopharmaceuticals and anyone interested in getting involved in a scientific, medical or business capacity.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products
Author: Maria Cristina Galli,Mercedes Serabian
Publsiher: Springer
Total Pages: 230
Release: 2015-09-15
Genre: Medical
ISBN: 9783319186184

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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.

Stem Cell and Gene Therapy for Cardiovascular Disease

Stem Cell and Gene Therapy for Cardiovascular Disease
Author: Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
Publsiher: Academic Press
Total Pages: 538
Release: 2015-08-21
Genre: Science
ISBN: 9780128018637

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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

A Guide to Human Gene Therapy

A Guide to Human Gene Therapy
Author: Roland W. Herzog,Sergei Zolotukhin
Publsiher: World Scientific
Total Pages: 388
Release: 2010
Genre: Medical
ISBN: 9789814280914

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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.

Cancer Gene Therapy

Cancer Gene Therapy
Author: David T. Curiel,Joanne T. Douglas
Publsiher: Springer Science & Business Media
Total Pages: 490
Release: 2007-11-03
Genre: Medical
ISBN: 9781592597857

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A complete introduction and guide to the latest developments in cancer gene therapy-from bench to bedside. The authors comprehensively review the anticancer genes and gene delivery methods currently available for cancer gene therapy, including the transfer of genetic material into the cancer cells, stimulation of the immune system to recognize and eliminate cancer cells, and the targeting of the nonmalignant stromal cells that support their growth. They also thoroughly examine the advantages and limitations of the different therapies and detail strategies to overcome obstacles to their clinical implementation. Topics of special interest include vector-targeting techniques, the lessons learned to date from clinical trials of cancer gene therapy, and the regulatory guidelines for future trials. Noninvasive techniques to monitor the extent of gene transfer and disease regression during the course of treatment are also discussed.

Advanced Wound Repair Therapies

Advanced Wound Repair Therapies
Author: David Farrar
Publsiher: Elsevier
Total Pages: 672
Release: 2011-06-21
Genre: Medical
ISBN: 9780857093301

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Wound repair is an important and growing sector of the medical industry with increasingly sophisticated biomaterials and strategies being developed to treat wounds. Advanced wound repair therapies provides readers with up-to-date information on current and emerging biomaterials and advanced therapies concerned with healing surgical and chronic wounds. Part one provides an introduction to chronic wounds, with chapters covering dysfunctional wound healing, scarring and scarless wound healing and monitoring of wounds. Part two covers biomaterial therapies for chronic wounds, including chapters on functional requirements of wound repair biomaterials, polymeric materials for wound dressings and interfacial phenomena in wound healing. In part three, molecular therapies for chronic wounds are discussed, with chapters on topics such as drug delivery, molecular and gene therapies and antimicrobial dressings. Part four focuses on biologically-derived and cell-based therapies for chronic wounds, including engineered tissues, biologically-derived scaffolds and stem cell therapies for wound repair. Finally, part five covers physical stimulation therapies for chronic wounds, including electrical stimulation, negative pressure therapy and mechanical debriding devices. With its distinguished editor and international team of contributors, Advanced wound repair therapies is an essential reference for researchers and materials scientists in the wound repair industry, as well as clinicians and those with an academic research interest in the subject. Provides readers with up-to-date information on current and emerging biomaterials and advanced therapies concerned with healing surgical and chronic wounds Chapters include the role of micro-organisms and biofilms in dysfunctional wound healing, tissue-biomaterial interaction and electrical stimulation for wound healing Covers biologically-derived and cell-based therapies for chronic wounds, including engineered tissues, biologically-derived scaffolds and stem cell therapies for wound repair