Gene and Cell Therapy


Book Description

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


Book Description

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: Biology and Applications


Book Description

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.




Regulatory Aspects of Gene Therapy and Cell Therapy Products


Book Description

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.




Exploring Novel Clinical Trial Designs for Gene-Based Therapies


Book Description

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.




Stem Cell and Gene Therapy for Cardiovascular Disease


Book Description

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 Therapy


Book Description

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.




Gene and Cellular Immunotherapy for Cancer


Book Description

Clinical and preclinical exploration of gene and cellular immunotherapy have seen rapid growth and interest with the development and approval of five Chimeric Antigen Receptor T-cell (CAR-T) products for lymphoma and myeloma and one Bispecific T-Cell Engager (BiTE) for acute lymphoblastic leukemia (ALL). These advances have dramatically improved the management of patients with relapsed refractory lymphoma, myeloma and leukemia. Gene and Cellular Immunotherapy for Cancer offers readers a comprehensive review of current cellular and gene-based immunotherapies. Divided into eighteen cohesive chapters, this book provides an in-depth and detailed look into cellular-based immunotherapies including CAR-T, TCR-T, TIL, Viral CTLs, NK cells in addition to T/NK cell engagers, focusing on their historical perspectives, biology, development and manufacturing, toxicities and more. Edited by two leading experts on gene and cellular immunotherapy, the book will feature chapters written by a diverse collection of recognized and up-and-coming experts and researchers in the field, providing oncologists, immunologists, researchers and clinical and basic science trainees with a bench to bedside view of the latest developments in the field.




Molecular and Cellular Therapies for Motor Neuron Diseases


Book Description

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


Book Description

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.