Stem Cell Therapy In Lysosomal Storage Diseases
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| Author | : Jaap Jan Boelens |
| Publisher | : Springer Science & Business Media |
| Total Pages | : 178 |
| Release | : 2013-10-10 |
| Genre | : Science |
| ISBN | : 1461483573 |
Stem Cell therapy for lysosomal diseases (LSDs) is developing rapidly. This volume discusses the history, current practice and future perspectives of stem cells in inborn errors of metabolism (IEM) and provides an international perspective on progress, limitations, and future directions (e.g. gene therapy, iPS, ES) in the field. Beginning with an overview of these diseases, the book covers the breadth of this topic from treatment options, bone marrow transplantation, and alternative treatment options, through long-term outcomes and future perspectives.
| Author | : Steven U. Walkley |
| Publisher | : |
| Total Pages | : 496 |
| Release | : 2004 |
| Genre | : Medical |
| ISBN | : |
Lysosomal storage diseases are inherited metabolic disorders characterized by severe pathology, typically involving the brain. Although individually rare, they collectively represent a significant group of diseases that primarily present in early infancy or childhood. In recent yearsconsiderable progress has been made in understanding the molecular mechanisms that lead to disordered function of the lysosomal system and to lysosomal storage. Unravelling the basis for these diseases is providing unique insight into the normal biology of cells and pointing the way to thedevelopment of therapeutic strategies for their treatment. Lysosomal Disorders of Brain details recent advances in the molecular and cellular pathologies of these diseases and in the development of effective therapies. After an overview of the biology of the endosomal-lysosomal system and the types of diseases resulting from defects in this system, thebook describes in detail the molecular mechanisms of storage, model systems and pathophysiological mechanisms, and finally, new advances toward treatment. With each chapter written by leading experts in their field, this book will be valuable for scientists and clinicians in helping them understandthe role of lysosomes in normal cells and mechanisms underlying these disorders, how they can be diagnosed, and the treatment options that are currently available.
| Author | : Michelle Kenyon |
| Publisher | : Springer |
| Total Pages | : 318 |
| Release | : 2018-03-14 |
| Genre | : Medical |
| ISBN | : 3319500260 |
This book is open access under a CC BY 4.0 license. This textbook, endorsed by the European Society for Blood and Marrow Transplantation (EBMT), provides adult and paediatric nurses with a full and informative guide covering all aspects of transplant nursing, from basic principles to advanced concepts. It takes the reader on a journey through the history of transplant nursing, including essential and progressive elements to help nurses improve their knowledge and benefit the patient experience, as well as a comprehensive introduction to research and auditing methods. This new volume specifically intended for nurses, complements the ESH-EBMT reference title, a popular educational resource originally developed in 2003 for physicians to accompany an annual training course also serving as an educational tool in its own right. This title is designed to develop the knowledge of nurses in transplantation. It is the first book of its kind specifically targeted at nurses in this specialist field and acknowledges the valuable contribution that nursing makes in this area. This volume presents information that is essential for the education of nurses new to transplantation, while also offering a valuable resource for more experienced nurses who wish to update their knowledge.
| Author | : Anthony H. Futerman |
| Publisher | : CRC Press |
| Total Pages | : 553 |
| Release | : 2006-07-07 |
| Genre | : Medical |
| ISBN | : 1420005502 |
In September of 2007 Gaucher Disease received a commendation in the Haematology category of the 2007 British Medical Association Medical Book Competition! Although rare in the general population, Gaucher disease is the most prevalent of the lysosomal storage disorders, making research into this particular orphan disorder an invaluable proto
| Author | : Yaoliang Tang |
| Publisher | : Academic Press |
| Total Pages | : 287 |
| Release | : 2015-09-02 |
| Genre | : Science |
| ISBN | : 0128004975 |
Mesenchymal stem cell-derived exosomes are at the forefront of research in two of the most high profile and funded scientific areas – cardiovascular research and stem cells. Mesenchymal Stem Cell Derived Exosomes provides insight into the biofunction and molecular mechanisms, practical tools for research, and a look toward the clinical applications of this exciting phenomenon which is emerging as an effective diagnostic. Primarily focused on the cardiovascular applications where there have been the greatest advancements toward the clinic, this is the first compendium for clinical and biomedical researchers who are interested in integrating MSC-derived exosomes as a diagnostic and therapeutic tool. - Introduces the MSC-exosome mediated cell-cell communication - Covers the major functional benefits in current MSC-derived exosome studies - Discusses strategies for the use of MSC-derived exosomes in cardiovascular therapies
| Author | : Sara Mole |
| Publisher | : OUP Oxford |
| Total Pages | : 772 |
| Release | : 2011-03-10 |
| Genre | : Medical |
| ISBN | : 019101558X |
The neuronal ceroid lipofuscinoses are an extremely rare group of inherited neurodegenerative diseases that primarily affect children. Core symptoms of these conditions typically include epilepsy, cognitive decline and visual failure. These diseases are so rare that professionals who come into contact with them need a consultative reference work that enables them to become expert, or identify who to contact for more details. Fully updated and revised, this second edition continues to be the definitive volume on this devastating group of disorders. Written by an international collection of authorities in the field, it provides invaluable advice on their diagnosis, patient care, and new treatments that are available. This new edition of the definitive reference text on the neuronal ceroid lipofuscinoses will prove useful for clinicians, family physicians, research scientists, diagnostic laboratories, families affected by the disease as well as by workers in industry planning translational research.
| Author | : Allison R. Kermode |
| Publisher | : John Wiley & Sons |
| Total Pages | : 496 |
| Release | : 2018-03-12 |
| Genre | : Science |
| ISBN | : 1118801482 |
A single volume collection that surveys the exciting field of plant-made pharmaceuticals and industrial proteins This comprehensive book communicates the recent advances and exciting potential for the expanding area of plant biotechnology and is divided into six sections. The first three sections look at the current status of the field, and advances in plant platforms and strategies for improving yields, downstream processing, and controlling post-translational modifications of plant-made recombinant proteins. Section four reviews high-value industrial and pharmacological proteins that are successfully being produced in established and emerging plant platforms. The fifth section looks at regulatory challenges facing the expansion of the field. The final section turns its focus toward small molecule therapeutics, drug screening, plant specialized metabolites, and plants as model organisms to study human disease processes. Molecular Pharming: Applications, Challenges and Emerging Areas offers in-depth coverage of molecular biology of plant expression systems and manipulation of glycosylation processes in plants; plant platforms, subcellular targeting, recovery, and downstream processing; plant-derived protein pharmaceuticals and case studies; regulatory issues; and emerging areas. It is a valuable resource for researchers that are in the field of plant molecular pharming, as well as for those conducting basic research in gene expression, protein quality control, and other subjects relevant to molecular and cellular biology. Broad ranging coverage of a key area of plant biotechnology Describes efforts to produce pharmaceutical and industrial proteins in plants Provides reviews of recent advances and technology breakthroughs Assesses realities of regulatory and cost hurdles Forward looking with coverage of small molecule technologies and the use of plants as models of human disease processes Providing wide-ranging and unique coverage, Molecular Pharming: Applications, Challenges and Emerging Areas will be of great interest to the plant science, plant biotechnology, protein science, and pharmacological communities.
| Author | : Alain A. Vertes |
| Publisher | : John Wiley & Sons |
| Total Pages | : 788 |
| Release | : 2015-09-14 |
| Genre | : Science |
| ISBN | : 1118846214 |
This book is a unique guide to emerging stem cell technologies and the opportunities for their commercialisation. It provides in-depth analyses of the science, business, legal, and financing fundamentals of stem cell technologies, offering a holistic assessment of this emerging and dynamic segment of the field of regenerative medicine. • Reviews the very latest advances in the technology and business of stem cells used for therapy, research, and diagnostics • Identifies key challenges to the commercialisation of stem cell technology and avenues to overcome problems in the pipeline • Written by an expert team with extensive experience in the business, basic and applied science of stem cell research This comprehensive volume is essential reading for researchers in cell biology, biotechnology, regenerative medicine, and tissue engineering, including scientists and professionals, looking to enter commercial biotechnology fields.
| Author | : Frederick J. Suchy |
| Publisher | : Cambridge University Press |
| Total Pages | : 995 |
| Release | : 2007-05-07 |
| Genre | : Medical |
| ISBN | : 1139464035 |
Completely revised new edition of the premier reference on pediatric liver disease. Liver Disease in Children, 3rd Edition provides authoritative coverage of every aspect of liver disease affecting infants, children, and adolescents. The book offers an integrated approach to the science and clinical practice of pediatric hepatology and charts the substantial progress in understanding and treating these diseases. Chapters are written by international experts and address the unique pathophysiology, manifestations, and management of these disorders in the pediatric population. The third edition has been thoroughly updated and features new contributions on liver development, cholestatic and autoimmune disorders, fatty liver disease, and inborn errors of metabolism. With the continued evolution of pediatric hepatology as a discipline, this text remains an essential reference for all physicians involved in the care of children with liver disease.
| Author | : Tanja Zigova |
| Publisher | : Springer Science & Business Media |
| Total Pages | : 438 |
| Release | : 2002-11-05 |
| Genre | : Medical |
| ISBN | : 1592592988 |
Active neuroscientists survey NSCs as potential tools for central nervous system and spinal cord repair by explaining their clinically significant fundamental properties, manipulations, and potential therapeutic paradigms. Their discussion of the fundamental biology of NSCs illustrates the signaling pathways that regulate stem cell division and differentiation, and defines the methods of NSC expansion and propagation, neuromorphogenesis, the factors determining cell fate both in vitro and in situ, and the induction of self-reparative processes within the brain. They also present strategies that may lead to fruitful clinical applications in the near future. These range from the replacement of degenerated, dysfunctional, or maldeveloped cells to the provision of factors that may protect, correct, recruit, promote self-repair, or mediate the connectivity of host cells.
