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Handbook of Cell and Gene Therapy

Handbook of Cell and Gene Therapy
Author: Hazel Aranha
Publisher: CRC Press
Total Pages: 444
Release: 2023-03-17
Genre: Science
ISBN: 1000864251

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This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.


A Handbook of Gene and Cell Therapy

A Handbook of Gene and Cell Therapy
Author: Clévio Nóbrega
Publisher: Springer Nature
Total Pages: 195
Release: 2020-06-27
Genre: Medical
ISBN: 3030413330

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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.


Gene Therapy

Gene Therapy
Author: Kenneth W. Culver
Publisher: Mary Ann Liebert
Total Pages: 144
Release: 1994
Genre: Medical
ISBN:

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Gene and Cell Therapy

Gene and Cell Therapy
Author: Nancy Smyth Templeton
Publisher: CRC Press
Total Pages: 1337
Release: 2015-01-20
Genre: Science
ISBN: 1466572000

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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


Handbook of Cell and Gene Therapy

Handbook of Cell and Gene Therapy
Author: Hazel Aranha
Publisher: CRC Press
Total Pages: 372
Release: 2023-03-17
Genre: Science
ISBN: 1000864200

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This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.


Cell Therapy

Cell Therapy
Author: Adrian Gee
Publisher: Springer Science & Business Media
Total Pages: 258
Release: 2009-09-18
Genre: Science
ISBN: 0387895841

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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.


Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy
Author: Peter J. Quesenberry
Publisher: John Wiley & Sons
Total Pages: 586
Release: 1998-09-10
Genre: Science
ISBN: 9780471146568

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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.


Stem Cells Handbook

Stem Cells Handbook
Author: Stewart Sell
Publisher: Springer Science & Business Media
Total Pages: 498
Release: 2003-10-22
Genre: Medical
ISBN: 1592594115

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The power of stem cells for tissue development, regeneration, and renewal has been well known by embryologists and developmental biologists for many years. Those presently active in research in the stem cell field owe much to previous work by embryologists and cancer researchers for their insights into what stem cells can do. In the last 4- 5 years, the rapid expansion of the concept of adult tissue stem cells as pluripotent progenitors for various tissues has led to an even greater appreciation of the power of stem cells. The demonstration that both embryonic and adult tissue stem cells have the ability to produce progenitor cells for tissue renewal has opened vast possibilities for treatment of congenital deficiency diseases as well as for regeneration of damaged tissues. Older concepts of determination leading to loss of potential during differentiation of adult tissues are being replaced by newer ideas that cells with multiple potential exist in different forms in various adult organs and that cells thought to be restricted to differentiation to one cell type may be able to "transdifferentiate" into other tissue cell types. Thus, the concept of "embryonic rests" in adult tissues, hypothesized to be the cellular origin of cancer by Durante and Conheim in the 1870s, now can be expanded to include survival of pluripotential embryonic-like stem cells in adult tissues.


A Guide To Human Gene Therapy

A Guide To Human Gene Therapy
Author: Roland W Herzog
Publisher: World Scientific
Total Pages: 415
Release: 2010-06-18
Genre: Science
ISBN: 9814467103

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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.


Gene and Cell Therapies

Gene and Cell Therapies
Author: Eve Hanna
Publisher: CRC Press
Total Pages: 168
Release: 2020-05-19
Genre: Medical
ISBN: 1000068471

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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