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The purpose of this book is to highlight some of latest developments and applications of CRISPR, RNA, and DNA to treat diseases ranging from cancers to cardiovascular and degenerative disorders. It also features innovations of the delivery methods for nucleic acids ranging from nanodevices made from DNA and pseudo amino acids to viral vectors. This is an ideal book for academics, clinicians, and students interested in gene therapy.

Much research has focused on the basic cellular and molecular biological aspects of stem cells. Much of this research has been fueled by their potential for use in regenerative medicine applications, which has in turn spurred growing numbers of translational and clinical studies. However, more work is needed if the potential is to be realized for improvement of the lives and well-being of patients with numerous diseases and conditions.This book series'Cell Biology and Translational Medicine (CBTMED)'as part of SpringerNature's longstanding and very successful Advances in Experimental Medicine and Biology book series, has the goal to accelerate advances by timely information exchange. Emerging areas of regenerative medicine and translational aspects of stem cells are covered in each volume. Outstanding researchers are recruited to highlight developments and remaining challenges in both the basic research and clinical arenas. This current book is the seventh volume of a continuing series.Chapter “Application of iPSC to Modelling of Respiratory Diseases” is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.

This book offers an updated overview of the most recent research advances in the field, a comparison of established techniques and methods, a discussion on current experimental and translational challenges, and a commentary on potential opportunities and future directions.Dedicated chapters address and review the preclinical and clinical state-of-the-art of gene therapies for the reconstructive and regenerative surgery of skin and wounds, pathological scars, cartilage, tendons, skeletal muscles, and bio-engineered flaps. A brief guide to developing gene therapy clinical trials in the context of reconstructive and regenerative surgery is also provided.Biomedical and technological innovations are transforming our capacity to use gene therapies to safely and effectively repair, reconstruct, and regenerate tissues that are deficient or have been damaged by trauma and diseases. The targeted and controlled modulation of gene expression in tissues represents a game-changing, next-generation therapeutic tool for the modern reconstructive surgeon, expanding the horizon of regenerative surgery and tissue engineering. Through gene therapies, surgeons can direct (stem) cell differentiation and cell function, modulate the release of growth/transcriptional factors, affect the biological properties of regenerative scaffolds, control tissue inflammation, or induce immune-suppression in composite tissue allotransplants and xenotransplants.Written by renowned reconstructive surgeons and leading experts in each of these fields - from top academic institutions around the globe, the book provides an initial practical guide for veteran and newcomer surgeons alike, as well as for researchers interested in exploring the latest gene-based therapeutic strategies for reconstructive and regenerative surgery.

Much research has focused on the basic cellular and molecular biological aspects of stem cells. Much of this research has been fueled by their potential for use in regenerative medicine applications, which has in turn spurred growing numbers of translational and clinical studies. However, more work is needed if the potential is to be realized for improvement of the lives and well-being of patients with numerous diseases and conditions.This book series'Cell Biology and Translational Medicine (CBTMED)'as part of SpringerNature's longstanding and very successful Advances in Experimental Medicine and Biology book series, has the goal to accelerate advances by timely information exchange. Emerging areas of regenerative medicine and translational aspects of stem cells are covered in each volume. Outstanding researchers are recruited to highlight developments and remaining challenges in both the basic research and clinical arenas. This current book is the third volume of a continuing series.

Much research has focused on the basic cellular and molecular biological aspects of stem cells. Much of this research has been fueled by their potential for use in regenerative medicine applications, which has in turn spurred growing numbers of translational and clinical studies. However, more work is needed if the potential is to be realized for improvement of the lives and well-being of patients with numerous diseases and conditions.This online first book series'Cell Biology and Translational Medicine (CBTMED)'as part of SpringerNature's longstanding and very successful Advances in Experimental Medicine and Biology book series, has the goal to accelerate advances by timely information exchange. Emerging areas of regenerative medicine and translational aspects of stem cells are covered in each volume. Outstanding researchers are recruited to highlight developments and remaining challenges in both the basic research and clinical arenas. This current book is the second volume of a continuing series.

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.

'Gene Therapy for Cartilage and Bone Tissue Engineering'outlines the tissue engineering and possible applications of gene therapy in the field of biomedical engineering as well as basic principles of gene therapy, vectors and gene delivery, specifically for cartilage and bone engineering. It is intended for tissue engineers, cell therapists, regenerative medicine scientists and engineers, gene therapist and virologists. Dr. Yu-Chen Hu is a Distinguished Professor at the Department of Chemical Engineering, National Tsing Hua University and has received the Outstanding Research Award (National Science Council), Asia Research Award (Society of Chemical Engineers, Japan) and Professor Tsai-Teh Lai Award (Taiwan Institute of Chemical Engineers). He is also a fellow of the American Institute for Medical and Biological Engineering (AIMBE) and a member of the Tissue Engineering International & Regenerative Medicine Society (TERMIS)-Asia Pacific Council.

This book introduces fundamental principles and practical application of techniques used in the scalable production of biopharmaceuticals with animal cell cultures. A broad spectrum of subjects relevant to biologics production and manufacturing are reviewed, including the generation of robust cell lines, a survey of functional genomics for a better understanding of cell lines and processes, as well as advances in regulatory compliant upstream and downstream development. The book is an essential reference for all those interested in translational animal cell-based pharmaceutical biotechnology.

This first title on the topic provides complete coverage, including the molecular basis, production and possible biomedical applications. Written by the most prominent academic researchers in the field as well as by researchers at one of the world's leading companies in industrial production of minicircle DNA, this practical book is aimed at everyone who is directly or indirectly involved in the development of gene therapies.

Examples from various organs and diseases illustrate the potential benefit obtained when both therapeutic approaches are combined with delivery strategies. Representing the combined effort of several leading international research and clinical experts, this book, Emerging Trends in Cell and Gene Therapy, provides a complete account on and brings into sharp focus current trends and state-of-the-art in important areas at the interface of cell- and gene-based therapies. This book addresses the current fragmented understanding regarding these two research areas and fills the vast unmet educational need and interest of both students and researchers in academia and industry. Main features of the book:· Biological aspects of stem cell sources, differentiation and engineering. · Application of microfluidics to study stem cell dynamics· Potential clinical application of stem cells and gene therapy to specific human disease. · Utilization of biomaterials and stem cells in regenerative medicine with particular emphasis on spinal cord repair, ligament and bone tissue engineering. · Biomimetic multiscale topography for cell alignment.

According to Greek mythology Pandora was sent down to earth upon the orders of Zeus. She was given a mysterious box which she was not allowed to open. However, Pandora was very curious and when she arrived on earth she couldn't help taking a peek inside the box. She saw that it was filled with gifts and calamities and to her astonishment they all escaped and spread throughout humanity, with all the dire consequences thereof. Only hope was left at the bottom. Figuratively speaking, Pandora's box today represents a source of much suffering. Is modern biotechnology just such a Pandora's box, as the anti-biotechnology lobby would have us believe? Or can we selectively release the gifts and turn this new Pandora's box into a Panacea? Modern biotechnology makes use of the recombinant DNA technology to genetically modify microorganisms, plants and animals in order to make them more suitable for all kinds of applications, such as cultivating food crops, baking bread, making wine, antibiotics and hormones, xenotransplantation, and gene- and stem cell therapy. The book also particularly addresses the controversial aspects of these applications. Some quotes taken from reviews of the Dutch version of Modern biotechnology: In short, this is a good book if you want to know more about the subject and if you want to develop a well-founded opinion. (Marijke Domis, NVOX) Altogether this has lead to a very accessible book. [...] Furthermore, we would like to mention that the book contains highly amusing illustrations which also contribute to the vast amount of information the book provides. (Ina Eggink, De Telegraaf) Completely clear and not one word too many, that is the first contribution to the readability. [...] For everyone who wants to gain a comprehensive view of the material this book is practically obligatory literature. (Fred van Hessen, NPT procestechnologie) Johannes (Hans) Tramper was born in 1949 in's Gravenpolder, the Netherlands. He later studied at the Technical University of Delft, Purdue University in the US and Wageningen University. Since 1987 he has been a professor of Bioprocess Technology at the latter institute. In addition to his main activities in education and research, he also spends a lot of time and effort informing the wider public about modern biotechnology. For more than 20 years he has traversed the Netherlands with his Pandora's box, visiting schools and libraries, attending women's societies, Rotary and Lions clubs, student associations, teacher's gatherings and using many other occasions, to promote discussions on the social and ethical aspects of biotechnology. As a reward for his work in this area, in 2003 Hans Tramper was awarded an honorary membership of the Dutch Biotechnology Association. Since March 2007 he has occupied a place on the ISI list of highly cited authors. Yang Zhu was born in Shanghai in 1955. After the cultural revolution in China, he followed a university course in Bioprocess Technology at East China University of Science and Technology. After obtaining his degree, he stayed on at the university as a lecturer. In 1986, Yang Zhu was sent as a guest researcher by the Chinese Ministry of Education to the Institute for Microbiology at Munster University, Germany. Since 1993 he has worked in the Netherlands, firstly as a scientific researcher at TNO Voeding in Zeist. In 1997 he obtained a PhD in bioprocess technology from the Agricultural University of Wageningen, as it was then called. He is now a senior project manager in the Business Unit Biosciences at TNO Quality of Life in Zeist.

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.

This book describes medical applications of recombinant proteins and monoclonal antibodies, some of which have already been on the market for several years while others have only recently been launched. It also highlights the manufacturing processes for individual products, the strategies that were taken by companies in the clinical development, and the hurdles that were encountered in clinical trials and had to be overcome before approval by regulatory authorities. Finally, this book illustrates strategies to modify and improve the pharmacodynamic and pharmacokinetic properties of naturally occurring proteins thus paving the way for a new era in biotechnology. Foreword written by Jurgen Drews.

This is the first book specializing in plasmids and their biomedical use, including all relevant aspects of production, applications, quality, and regulations. Readers will discover clinical applications for the wide range of preventive and therapeutic applications using plasmid DNA. The book describes modified vector systems based on plasmids, as well as the potency of genomic research and vector design by informatics. Using the example of fish vaccination, the application of DNA vaccination in veterinary health care is reviewed, followed by a detailed overview of plasmid production technology on an industrial scale. Finally, the book considers regulatory and quality assurance aspects of such new drugs plus thire market potential.