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.

This book reviews recent knowledge of the role of stem cells in the gastrointestinal system. It covers extensive topics for each organ, including the pancreas, esophagus, liver, and colon, while also discussing the contributions of stem cells to therapeutic approaches toward gastrointestinal diseases, including inflammatory bowel diseases. Comprehensive and cutting-edge, Digestive System Diseases: Stem Cell Mechanisms and Therapies deepens a reader's theoretical expertise in gastrointestinal stem cell biology. It furthers scientists' understanding of gastrointestinal stem cells and, most importantly, the development of novel therapeutic targets. Graduate and postdoctoral students, medical doctors (including gastroenterologists and surgeons), and principal investigators in both academia and industry will benefit from this book. In particular, it is a valuable resource for professionals within the fields of gastrointestinal research, pharmaceutical science, molecular biology, regenerative medicine, and genetics.

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. With a goal to accelerate advances by timely information exchange, this new 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 is launched. Emerging areas of regenerative medicine and translational aspects of stem cells will be 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 first volume of a continuing series.

Due to continuous technical developments and new insights into the high complexity of neurological diseases, there is an increasing need for the application of proteomic technologies which can yield potential biomarker readouts for improved clinical management as well as for the development of new drugs by struggling pharmaceutical companies. This book describes the step-by-step use of proteomic methods such as two-dimensional gel electrophoresis, multiplex immunoassay, liquid chromatography mass spectrometry (LC-MS) and selective reaction monitoring MS, to increase our understanding of these diseases, with the ultimate aim of improving patient care. The volume will be of high interest to clinical scientists, physicians and pharmaceutical company scientists as it gives insights into the latest technologies enabling the revolution of personalized medicine. It is of direct interest to both technical and bench biomarker scientists as it gives step by step instructions on how to carry out each of the protocols. It is also of interest to researchers as each technique will be presented in the context of a specific neurological disorder, including Alzheimer's disease, multiple sclerosis, autism spectrum disorders, schizophrenia, major depressive disorder and bipolar disorder. Finally, it will also highlight the future research efforts in this field, which are endeavoring to convert proteomic platforms to the form of hand held devices which can be used in a point of care setting and return diagnostic results within the timeframe of a visit to the general practitioner.

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 work was compiled to serve as a convenient source that covers a number of techniques (and details of their use) in the rather large field of nanomedicine, with special attention paid to gene delivery. As principal investigators working in the field of nanomedicine, we sought to put together the most current and relevant topics in gene delivery, imaging and evaluation systems. We expect the work to serve very well for scientists and graduate students in the nanomedicine field.

Molecules to Medicine with mTOR: Translating Critical Pathways into Novel Therapeutic Strategies is a one-stop reference that thoroughly covers the mechanistic target of rapamycin (mTOR). mTOR, also known as the mammalian target of rapamycin, is a 289-kDa serine/threonine protein kinase that is ubiquitous throughout the body and has a critical role in gene transcription and protein formation, stem cell development, cell survival and senescence, aging, immunity, tissue regeneration and repair, metabolism, tumorigenesis, oxidative stress, and pathways of programmed cell death that include apoptosis and autophagy. Incorporating a translational medicine approach, this important reference highlights the basic cellular biology of mTOR pathways, presents the role of mTOR during normal physiologic function and disease, and illustrates how the mechanisms of mTOR can be targeted for current and future therapeutic treatment strategies. Coverage of mTOR signaling includes the entire life cycle of cells that impacts multiple systems of the body including those of nervous, cardiovascular, immune, musculoskeletal, endocrine, reproductive, renal, and respiratory origin.

This book provides current, comprehensive, and clear explanations of the physics behind medical and biomedical applications of shock waves. Extracorporeal shock wave lithotripsy is one of the greatest medical advances of our time, and its techniques and clinical devices are continuously evolving. Further research continues to improve the understanding of calculi fragmentation and tissue-damaging mechanisms. Shock waves are also used in orthopedics and traumatology. Possible applications in oncology, cardiology, dentistry, gene therapy, cell transfection, transformation of fungi and bacteria, as well as the inactivation of microorganisms are promising approaches for clinical treatment, industrial applications and research. Medical and Biomedical Applications of Shock Waves is useful as a guide for students, technicians and researchers working in universities and laboratories. Chemists, biologists, physicians and veterinarians, involved in research or clinical practice will find useful advice, but also engineers and physicists may benefit from the overview of current research endeavors and future directions. Furthermore, it may also serve to direct manufacturers towards the design of more efficient and safer clinical, industrial and laboratory equipment.

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy.

Is inheritable genetic modification the new dividing line in gene therapy? The editors of this searching investigation, representing clinical medicine, public health and biomedical ethics, have established a distinguished team of scientists and scholars to address the issues from the perspectives of biological and social science, law and ethics, including an intriguing Foreword from Peter Singer. Their purpose is to consider how society might deal with the ethical concerns raised by inheritable genetic modification, and to re-examine prevailing views about whether these procedures will ever be ethically and socially justifiable. The book also provides background to define the field, and discusses the biological and technological potential for inheritable genetic modification, its limitations, and its connection with gene therapy, cloning, and other reproductive interventions. For scientists, bioethicists, clinicians, counsellors and public commentators, this is an essential contribution to one of the critical debates in current genetics.

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 professionala (TM)s library.

Additional topics in Cell Therapy: cGMP Facilities and Manufacturinga ]

• Standard operating procedures
• Supply management
• Facility equipment
• Product manufacturing, review, release and administration
• Facility master file

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.

Over the past decade a complex role for DNA damage response (DDR) in tumorigenesis has emerged. A proficient DDR has been shown to be a primary cause for cellular resistance to the very many DNA damaging drugs, and IR, that are widely used as standard-of-care across multiple cancer types. It has also been shown that defects in this network, predominantly within the ATM mediated signaling pathway, are commonly observed in cancers and may be a primary event during tumorigenesis. Such defects may promote a genomically unstable environment, facilitating the persistence of mutations, any of which may provide a growth or survival advantage to the developing tumor. In addition, these somatic defects provide opportunities to exploit a reliance on remaining repair pathways for survival, a process which has been termed synthetic lethality. As a result of all these observations there has been a great interest in targeting the DDR to provide anti-cancer agents that may have benefit as monotherapy in cancers with high background DNA damage levels or as a means to increase the efficacy of DNA damaging drugs and IR. In this book we will review a series of important topics that are of great interest to a broad range of academic, industrial and clinical researchers, including the basic science of the DDR, its role in tumorigenesis and in dictating response to DNA damaging drugs and IR. Additionally, we will focus on the several proteins that have been targeted in attempts to provide drug candidates, each of which appear to have quite distinct profiles and could represent very different opportunities to provide patient benefit.

This book reviews the current state of ocular drug therapy and future therapeutic opportunities for a wide variety of conditions, including Age-related Macular Degeneration, Diabetic Retinopathy and Macular Edema, Glaucoma, and Inherited Retinal Diseases. Retinal diseases are major contributors to moderate or severe vision impairment in adults aged 50 years and older. The respective patient populations for many of these indications is expected to significantly increase as the world population continues to grow older. An improved understanding of the etiological underpinnings of ocular degenerative diseases over the past decade has significantly bolstered ophthalmic drug discovery. In this volume, contributions from leading experts explore the unique challenges faced for ocular drug discovery and delivery providing the reader with detailed information on ocular pharmacokinetics, in vitro, ex vivo and in vivo models for retinal disease pathology and emerging gene therapy treatments. The book is intended for all researchers and clinicians who wish to increase their knowledge on the latest findings in ocular drug therapy.

This detailed volume explores the methods used for most of the recent approaches to suicide gene therapy of cancer, which exploits promoters that are specific to cancer cells, thereby ensuring (or greatly increasing the likelihood) that the therapeutic gene is expressed only in cancer cells. The book also contains chapters describing methods to improve the safety of cell therapy and techniques utilizing bone marrow mesenchymal cells. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Suicide Gene Therapy: Methods and Protocols serves as an ideal guide for researchers expanding upon our knowledge and application of this vital form of cancer therapy.

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 issue is part two of a two-part issue involving the emerging field of stem cell transplantation.? Articles include: BMT for profound T cell Dysfunction (combined immunodeficiency); BMT and other management of HLA Class II deficiency; Genetic manipulation of stem cells - Past and future; Gene therapy for ADA deficiency; Haemopoietic stem cell biology; and Indications for BMT.

The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy.This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions.In"Prenatal Gene Therapy: Concepts, Methods and Protocols, "expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These includedetailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and forthe methods of gene transfer analysis. Written in the highly successful"Methods in Molecular Biology "series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.

Thorough and intuitive, "Prenatal Gene Therapy: Concepts, Methods and Protocols"seeks to aid scientists in the further study ofprenatal disease and gene therapy projects beyond the scope of fetal medicine.

This book provides a unique and innovative perspective on the controversial phenomenon of 'stem cell tourism'. A growing number of patients are embarking on stem cell treatments that are clinically unproven and yet available in clinics and hospitals around the world. The authors offer a cutting-edge multi-dimensional perspective on this complex and rapidly changing phenomenon, including an analysis of the experiences of those who have undertaken or have contemplated undertaking a stem cell treatment, as well as examination of the views of those who undertake research or advise on or provide stem cell treatments. Developing the concept of 'the political economy of hope', and referencing case studies of the stem cell treatment market in China, Germany, and Australia, this book argues for a reframing of 'stem cell tourism' to understand why patients and families pursue these treatments and whether authorities' concerns are justified and whether their responses are appropriate and proportionate to the alleged risks.

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 sixth volume of a continuing series.

This book explores critical principles and new concepts in bioengineering, integrating the biological, physical and chemical laws and principles that provide a foundation for the field. Both biological and engineering perspectives are included, with key topics such as the physical-chemical properties of cells, tissues and organs; principles of molecules; composition and interplay in physiological scenarios; and the complex physiological functions of heart, neuronal cells, muscle cells and tissues. Chapters evaluate the emerging fields of nanotechnology, drug delivery concepts, biomaterials, and regenerative therapy. The leading individuals and events are introduced along with their critical research. Bioengineering: A Conceptual Approach is a valuable resource for professionals or researchers interested in understanding the central elements of bioengineering. Advanced-level students in biomedical engineering and computer science will also find this book valuable as a secondary textbook or reference.

This book discusses the emergence of a new class of genes with a specific anticancer activity. These genes, recently defined as “Anticancer Genes”, are reviewed in individual chapters on their mode of action, the specific cell death signals they induce, and the status of attempts to translate them into clinical application. Anticancer Genes provides an overview of this nascent field, its genesis, current state, and prospect. It discusses how Anticancer Genes might lead to the identification of a repertoire of signaling pathways directed against cellular alterations that are specific for tumor cells. With contributions from experts worldwide, Anticancer Genes is an essential guide to this dynamic topic for researchers and students in cancer research, molecular medicine, pharmacology and toxicology and genetics as well as clinicians and clinical researchers interested in the therapeutic potential of this exciting new field.

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular Biology (TM) series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

This book will contain the proceedings of the XV International Symposium on Retinal Degeneration (RD2012). A majority of those who will speak and present posters at the meeting will contribute to this volume. The blinding diseases of inherited retinal degenerations have no treatments, and age-related macular degeneration has no cures, despite the fact that it is an epidemic among the elderly, with 1 in 3-4 affected by the age of 70. The RD Symposium will focus on the exciting new developments aimed at understanding these diseases and providing therapies for them. Since most major scientists in the field of retinal degenerations attend the biennial RD Symposia, they are known by most as the "best" and "most important" meetings in the field. The volume will present representative state-of-the-art research in almost all areas of retinal degenerations, ranging from cytopathologic, physiologic, diagnostic and clinical aspects; animal models; mechanisms of cell death; candidate genes, cloning, mapping and other aspects of molecular genetics; and developing potential therapeutic measures such as gene therapy and neuroprotective agents for potential pharmaceutical therapy. While advances in these areas of retinal degenerations will be described, there will be many new topics that either were in their infancy or did not exist at the time of the last RD Symposium, RD2010. These include the role of inflammation and immunity, as well as other basic mechanisms, in age-related macular degeneration, several new aspects of gene therapy, and revolutionary new imaging and functional testing that will have a huge impact on the diagnosis and following the course of retinal degenerations, as well as to provide new quantitative endpoints for clinical trials. The retina is an approachable part of the central nervous system (CNS), and there is a major interest in neuroprotective and gene therapy for CNS diseases and neurodegenerations, in general. It should be noted that with successful and exciting initial clinical trials in neuroprotective and gene therapy, including the restoration of sight in blind children, the retinal degeneration therapies are leading the way towards new therapeutic measures for neurodegenerations of the CNS. Many of the successes recently reported in these areas of retinal degeneration sprang from collaborations established at previous RD Symposia, and many of those will be reported at the RD2010 meeting and included in the proposed volume. We anticipate the excitement of those working in the field and those afflicted with retinal degenerations will be reflected in the volume.