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Molecular and Cellular Therapeutics aims to bring together key developments in the areas of molecular diagnostics, therapeutics and drug discovery. The book covers topics including diagnostics, therapeutics, model systems, clinical trials and drug discovery. The developing approaches to molecular and cellular therapies, diagnostics and drug discovery are presented in the context of the pathologies they are devised to treat.
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
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.
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.
"This book should be useful to lecturers who teach senior undergraduates, graduate students, and students in the biomedical sciences in general. More globally, Greenwell and McCulley's book should encourage academicians of any stripe who for some time have been honing their lectures in a niche subject area to turn their courses into textbooks." –Biochemistry and Molecular Biology Education Molecular therapeutics refers to the developments in molecular biology that are focused on treating disease with new molecular-based drugs. By taking advantage of recent advances and increased understanding in the field of human genetics, this book provides essential background knowledge and key literature on a broad range of novel approaches and disciplines. These include making recombinant proteins, xenotransplantation gene therapy and therapeutic cloning. Molecular Therapeutics: 21st Century Medicine describes the techniques - including their associated benefits, problems, pitfalls - and discusses their applicability with respect to treating microbial, inherited, multifactoral and acquired diseases. The book also pays specific attention to the ethical issues associated with this new field. Cutting-edge topics and clinically relevant materials engage and maintain student interest Self-assessment questions are included throughout the book Features an additional web site, with a web forum, regular updates and PowerPoint slides of figures from the book Molecular Therapeutics: 21st Century Medicine is a comprehensive, accessible and engaging guide to the rapidly developing field of molecular therapeutics. It is essential reading for all students in this area of research and also of interest for health professionals involved in these novel therapies.
Molecular and Cellular Therapeutics aims to bring together key developments in the areas of molecular diagnostics, therapeutics and drug discovery. The book covers topics including diagnostics, therapeutics, model systems, clinical trials and drug discovery. The developing approaches to molecular and cellular therapies, diagnostics and drug discovery are presented in the context of the pathologies they are devised to treat.
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
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.
advanced metastatic disease of solid tumors, dictates that each tumor mass, indeed each individual metastasis, will have a unique antigen and cytokine environment and hence unique response to immune modu lation. A differential response to immunotherapy is thus inevitable. 4. Many of the human trials described are not randomized and report survival or response against historical controls. Most tumors described are immunogenic human tumors: renal cell cancer and melanoma are most common. In order to avoid the well-described inter-patient vari ation and rare incidence of spontaneous response among patient samples as well as selection bias and changes in practice over time, randomized trials are required. 5. Immunological treatment is unlike conventional chemotherapy in its endpoint. Most chemotherapeutic regimes require a complete response or a good partial response for cure or good palliation. There are now many cases where immunotherapy has provided long-term palliation without massive tumor reduction. Immunity may be stimulated to a degree which holds tumorigenicity in check and most importantly, pro vides good palliation for the patient in a manner that differs essentially from chemotherapy.