Tightly linked to tissue engineering and regenerative medicine, genetic and cellular therapies have drawn universal attention. Since genetic sequencing is critical for precision medicine, the combination of bioinformatics and therapies is important to conduct prescriptive and predictive analytics based on genetic sequencing data. Bioinformatics and computational approaches have been widely considered for the development of genetic and cellular therapies. Therefore, the current research topic aims to cover recent advances in studies such as functional genomics, proteomics, metabolomics, and bioinformatics that bring in new perspectives on genetic therapies. The central goal of regenerative medicine is to replace damaged or diseased tissue with cells that are integrated and functioning optimally. Stem cell-based therapy is restricted by the limited availability of cell sources, the excessive cost, and the anticipated difficulties of clinical translation and ethical approval. Some promising alternatives are to incite the patient's innate ability of tissue repairs such as in situ tissue regeneration, which has the potential to provide new therapeutic options for tissue engineering and regenerative medicine. These innovative cellular therapies are also contained in our research topic.

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.

The development, clinical translation and recent efficacy of novel gene therapies targeting refractory malignancies has led to research that extends this technology to a variety of infectious and rheumatological diseases. Unlike conventional drugs or antibodies, T cells have the potential to target and exert effector function in response to disease in a dynamic manner, acting as a “living drug”. The most efficacious form of gene-modified T cells to date is the chimeric antigen receptor (CAR)-modified T cell, which redirects the specificity of T cells to an antigen expressed by tumor cells. Clinical experience with autologous CAR-T cells, primarily in hematologic malignancies, has underscored the feasibility and safety of the approach, while also demonstrating dramatic and sustained antitumor effects through mechanisms orthogonal to those of traditional anticancer therapies. However, several challenging obstacles must be surmounted in order to improve the broader efficacy of this approach.

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.

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.

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.

Cells are the building blocks of life and some cells (stem cells) have the ability to produce other cells through the processes of cell division and cell differentiation. Stem cell research has now progressed dramatically and there are countless studies published every year in scientific journals. Stem cell technology is being used to create new cell lines with edited genes and to regenerate cell based tissues for biological and medical purposes. This ebook presents a brief snapshot of clinical research in stem cell research and regenerative medicine. The concise reference is intended to be an introduction for biology students to current standards and new technologies in these fields.

This book presents a detailed overview of the most modern and promising developments in pain management. Chronic pain affects millions of people worldwide and poses a major challenge for medicine, as conventional treatment approaches often offer only limited relief. This book provides an insight into innovative therapeutic options that have the potential to make a lasting difference to the lives of sufferers. From neuromodulation to spinal cord stimulation and targeted brain stimulation, the book shows how modern technologies are used to influence pain signals directly in the nervous system, thus achieving effective pain relief. In addition, the latest developments in pharmacology are highlighted, including new classes of active ingredients and medications that act specifically on pain receptors, thus enabling targeted treatment with fewer side effects. Another focus is on advances in gene and cell therapy. With a deep understanding of the molecular mechanisms that cause pain, new treatment options are emerging that address the cause of the pain directly. Stem cell therapies, genetic approaches to pain reduction and innovative biotechnological methods could revolutionise the way pain is treated. In addition to the scientific background and current clinical studies, the book provides practical insights into the application of the new therapies. It highlights both the effects on patients' quality of life and the ethical, legal and social aspects that play a role in the development and implementation of innovative pain treatment approaches. This provides a comprehensive picture of the current state of research and future prospects in pain medicine.

These proceedings gather the best papers presented at the “10th International Scientific and Practical Conference – the 21st Century from the Positions of Modern Science: Intellectual, Digital and Innovative Aspects,” which was organized by the non-profit organization “Institute of Scientific Communications.” The conference took place on May 23–24 in Nizhny Novgorod, Russia, with support from Minin Nizhny Novgorod State Pedagogical University. The chief advantage of these proceedings are their multidisciplinary character – they include articles and empirical studies addressing various fields, including economics, the social sciences, and law. Accordingly, the target audience is broad, covering scholars, researchers, independent experts, entrepreneurs, and government workers, who are interested in issues concerning: measuring and accelerating socio-economic development; the formation and evolution of the digital society and digital economy; the role of economic systems and economic subjects in the 21st-century technological revolution (the fourth industrial revolution); development and implementation of AI; development and application of intellectual resources in economic activities; and innovations in the economy.

New Frontiers and Applications of Synthetic Biology presents a collection of chapters from eminent synthetic biologists across the globe who have established experience and expertise working with synthetic biology. This book offers several important areas of synthetic biology which allow us to read and understand easily. It covers the introduction of synthetic biology and design of promoter, new DNA synthesis and sequencing technology, genome assembly, minimal cells, small synthetic RNA, directed evolution, protein engineering, computational tools, de novo synthesis, phage engineering, a sensor for microorganisms, next-generation diagnostic tools, CRISPR-Cas systems, and more. This book is a good source for not only researchers in designing synthetic biology, but also for researchers, students, synthetic biologists, metabolic engineers, genome engineers, clinicians, industrialists, stakeholders and policymakers interested in harnessing the potential of synthetic biology in many areas. - Offers basic understanding and knowledge in several aspects of synthetic biology - Covers state-of-the-art tools and technologies of synthetic biology, including promoter design, DNA synthesis, DNA sequencing, genome design, directed evolution, protein engineering, computational tools, phage design, CRISPR-Cas systems, and more - Discusses the applications of synthetic biology for smart drugs, vaccines, therapeutics, drug discovery, self-assembled materials, cell free systems, microfluidics, and more