Download or read book Ex Vivo Gene Therapy for Lysosomal Storage Disease Using IPSC derived Neural Stem Cells written by Tagan Aaron Griffin and published by . This book was released on 2015 with total page 174 pages. Available in PDF, EPUB and Kindle. Book excerpt: Diseases affecting the central nervous system (CNS) pose a formidable obstacle to the delivery of effective therapeutics. A tight-knit collection of cells and macromolecules known as the blood-brain-barrier (BBB) prevents most substances from entering the brain. One intriguing approach to overcoming this obstacle involves transplanting neural stem cells (NSCs), the precursor cells to neurons and glia in the brain, as vehicles for the delivery of therapeutic proteins in their native environment. Notably, this strategy has already been successfully applied to several lysosomal storage diseases caused by genetic deficiencies in one of the many lysosomal hydrolases expressed throughout the body. A major drawback to this approach is that foreign NSCs, e.g. immortalized cell lines and primary fetal NSCs can be tumorigenic and immunogenic. Recently developed induced pluripotent stem cell (iPSC) technologies, combined with pluripotent stem cell differentiation techniques, have the potential to overcome these obstacles. This approach was evaluated using a comprehensive strategy targeting a prototypical lysosomal storage disease, Sly disease (MPS VII). MPS VII patient fibroblasts were transduced with retroviral vectors expressing the transcription factors Oct4, Sox2, Klf4, and c-Myc. Patient fibroblasts were reprogrammed into embryonic stem cell-like iPSCs that demonstrated hallmarks of pluripotency. Patient iPSCs, alongside iPSCs derived from an unaffected individual, were subjected to a stepwise differentiation protocol, yielding a relatively homogenous population of NSCs. Following in vitro characterization, patient iPSCs were genetically corrected using a DNA transposon-based vector. Transplantation of NSCs into neonatal MPS VII mice revealed that these cells could migrate long distances and survive for several months. However, corrected grafts expressing physiological levels of the missing enzyme, ?-glucuronidase, were too sparse to significantly ameliorate pathology. In contrast, the same cells transplanted into the post-symptomatic adult MPS VII striatum were restricted to the injection site. Corrected, but not uncorrected patient iPSC-NSCs, were able to restore pathologically activated microglia to a normal quiescent state in a zone surrounding the graft. Together, these results provide evidence that ex vivo NSC gene therapy may be a viable option for many lysosomal storage diseases using easily attainable, non-neural patient tissue.
Download or read book Stem Cell Therapy in Lysosomal Storage Diseases written by Jaap Jan Boelens and published by Springer Science & Business Media. This book was released on 2013-10-10 with total page 178 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Gene Correction written by Francesca Storici and published by Humana. This book was released on 2016-09-03 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene correction is a technology that gives us the tools for both repairing and mutating DNA, for discovering gene functions and for engineering new genetic variants. Gene Correction: Methods and Protocols provides a user friendly, detailed and up-to-date collection of strategies and methodologies utilized for generating specific sequence changes in the DNA of cells in the laboratory, while also tackling the major problems that the field of gene correction faces. This volume brings together many experts in the field of gene correction to disclose a wide and varied array of specific gene correction protocols for engineering mutations in DNA, for delivering correcting DNA to target cells, and for improving the accuracy and safety of the gene correction process. Written in the 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 protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and easily accessible, Gene Correction: Methods and Protocols seeks to serve scientists of all backgrounds interested in the area of gene targeting/recombination/therapy.
Download or read book The Neuronal Ceroid Lipofuscinoses Batten Disease written by Sara Mole and published by OUP Oxford. This book was released on 2011-03-10 with total page 480 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Gene Therapy of the Central Nervous System From Bench to Bedside written by Michael G. Kaplitt and published by Elsevier. This book was released on 2005-11-11 with total page 372 pages. Available in PDF, EPUB and Kindle. Book excerpt: Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use
Download or read book Recent Advances in iPSC Disease Modeling written by Alexander Birbrair and published by Academic Press. This book was released on 2020-07-16 with total page 322 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recent Advances in iPSC Disease Modeling, Volume One addresses how induced pluripotent stem cells can be used to model various diseases. This new volume teaches readers about current advances in the field, describing the use of induced pluripotent stem cells to model several diseases in vitro, and thus enabling us to study the cellular and molecular mechanisms involved in different pathologies. Further insights into these mechanisms will have important implications for our understanding of disease appearance, development and progression. The volume is written for researchers and scientists in stem cell therapy, cell biology, regenerative medicine and organ transplantation specialists. In recent years, remarkable progress has been made in the obtention of induced pluripotent stem cells and their differentiation into several cell types, tissues and organs using state-of-art techniques. Hence, these advantages have facilitated the identification of key targets and further defining on the molecular basis of several disorders. Provides an overview on the fast-moving field of induced pluripotent stem cell technology, regenerative medicine and therapeutics Covers the following diseases: severe congenital neutropenia, sickle cell and Diamond-Blackfan anemias, muscular dystrophies, Bernard-Soulier syndrome, familial hypercholesterolemia type II A, Werner syndrome, lysosomal storage diseases, and more Contains descriptions of cutting-edge research on the development of disease-specific human pluripotent stem cells
Download or read book Curing Genetic Diseases through Genome Reprogramming written by and published by Academic Press. This book was released on 2021-06-24 with total page 560 pages. Available in PDF, EPUB and Kindle. Book excerpt: Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations
Download or read book Polyglutamine Disorders written by Clévio Nóbrega and published by Springer. This book was released on 2018-02-09 with total page 467 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a cutting-edge review of polyglutamine disorders. It primarily focuses on two main aspects: (1) the mechanisms underlying the pathologies’ development and progression, and (2) the therapeutic strategies that are currently being explored to stop or delay disease progression. Polyglutamine (polyQ) disorders are a group of inherited neurodegenerative diseases with a fatal outcome that are caused by an abnormal expansion of a coding trinucleotide repeat (CAG), which is then translated in an abnormal protein with an elongated glutamine tract (Q). To date, nine polyQ disorders have been identified and described: dentatorubral-pallidoluysian atrophy (DRPLA); Huntington’s disease (HD); spinal–bulbar muscular atrophy (SBMA); and six spinocerebellar ataxias (SCA 1, 2, 3, 6, 7, and 17). The genetic basis of polyQ disorders is well established and described, and despite important advances that have opened up the possibility of generating genetic models of the disease, the mechanisms that cause neuronal degeneration are still largely unknown and there is currently no treatment available for these disorders. Further, it is believed that the different polyQ may share some mechanisms and pathways contributing to neurodegeneration and disease progression.
Download or read book The Fibroblast Growth Factor Family written by Andrew Baird and published by . This book was released on 1991 with total page 542 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gene Therapy in Neurological Disorders written by Mingjie Li and published by Academic Press. This book was released on 2018-05-25 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders. Each chapter gives an in-depth introduction to the relevant field before diving into the specific tool or application. The book aims to help investigators, students and research staff better understand the principles of gene therapy and its application in the nervous system. Provides background information and experimental details of gene therapy tools applied for neuroscience research and neurological disorders Covers a broad range of gene delivery and regulation tools, therapeutic agents, and target cells, including emerging new technologies such as CRISPR/Cas9 genome editing Discusses applications of gene therapy tools to neurological disorders including neurodegeneration, muscular dystrophy, trauma and chronic pain, and neoplastic diseases
Download or read book Regenerative Pharmacology written by George J. Christ and published by Cambridge University Press. This book was released on 2013-04-15 with total page 361 pages. Available in PDF, EPUB and Kindle. Book excerpt: A state-of-the-art primer on the role of pharmacological sciences in regenerative medicine, for advanced students, postdoctoral fellows, and researchers.
Download or read book Cell Biology and Translational Medicine Volume 14 written by Kursad Turksen and published by Springer Nature. This book was released on 2022-01-01 with total page 225 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 Springer Nature’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 14th volume of a continuing series.
Download or read book Extracellular Matrix in Development written by Douglas W. DeSimone and published by Springer Science & Business Media. This book was released on 2013-03-15 with total page 260 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cells in the developing embryo depend on signals from the extracellular environment to help guide their differentiation. An important mediator in this process is the extracellular matrix – secreted macromolecules that interact to form large protein networks outside the cell. During development, the extracellular matrix serves to separate adjacent cell groups, participates in establishing morphogenic gradients, and, through its ability to interact directly will cell-surface receptors, provides developmental clocks and positional information. This volume discusses how the extracellular matrix influences fundamental developmental processes and how model systems can be used to elucidate ECM function. The topics addressed range from how ECM influences early development as well as repair processes in the adult that recapitulate developmental pathways.
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Download or read book Stem Cell Engineering written by Gerhard M. Artmann and published by Springer Science & Business Media. This book was released on 2010-10-29 with total page 566 pages. Available in PDF, EPUB and Kindle. Book excerpt: The potential of stem cells for healing and disease prevention in all fields of medicine is tremendous and has revolutionized the high-tech biomedical research. In this book, many of the most prominent researchers discuss the challenging topics of stem cell engineering, for example: Ethical issues of stem cell research; technological challenges, stem cell growth and differentiation, therapeutic applications, bioreactors and bioprocesses, high throughput and microfluidic screening platforms, stem cell identification and sorting, intercellular signaling and engineered niches, novel approaches for embryonic and adult stem cell growth and differentiation, stem cells and drug discovery, screening platforms. Stem Cell Engineering offers valuable background and reference for both the public and professionals including industrial staffers, faculty, researchers, engineers, students and scientific journalists.
Download or read book Mesenchymal Stem Cell Therapy written by Lucas G. Chase and published by Springer Science & Business Media. This book was released on 2012-12-12 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over the past decade, significant efforts have been made to develop stem cell-based therapies for difficult to treat diseases. Multipotent mesenchymal stromal cells, also referred to as mesenchymal stem cells (MSCs), appear to hold great promise in regards to a regenerative cell-based therapy for the treatment of these diseases. Currently, more than 200 clinical trials are underway worldwide exploring the use of MSCs for the treatment of a wide range of disorders including bone, cartilage and tendon damage, myocardial infarction, graft-versus-host disease, Crohn’s disease, diabetes, multiple sclerosis, critical limb ischemia and many others. MSCs were first identified by Friendenstein and colleagues as an adherent stromal cell population within the bone marrow with the ability to form clonogenic colonies in vitro. In regards to the basic biology associated with MSCs, there has been tremendous progress towards understanding this cell population’s phenotype and function from a range of tissue sources. Despite enormous progress and an overall increased understanding of MSCs at the molecular and cellular level, several critical questions remain to be answered in regards to the use of these cells in therapeutic applications. Clinically, both autologous and allogenic approaches for the transplantation of MSCs are being explored. Several of the processing steps needed for the clinical application of MSCs, including isolation from various tissues, scalable in vitro expansion, cell banking, dose preparation, quality control parameters, delivery methods and numerous others are being extensively studied. Despite a significant number of ongoing clinical trials, none of the current therapeutic approaches have, at this point, become a standard of care treatment. Although exceptionally promising, the clinical translation of MSC-based therapies is still a work in progress. The extensive number of ongoing clinical trials is expected to provide a clearer path forward for the realization and implementation of MSCs in regenerative medicine. Towards this end, reviews of current clinical trial results and discussions of relevant topics association with the clinical application of MSCs are compiled in this book from some of the leading researchers in this exciting and rapidly advancing field. Although not absolutely all-inclusive, we hope the chapters within this book can promote and enable a better understanding of the translation of MSCs from bench-to-bedside and inspire researchers to further explore this promising and quickly evolving field.
Download or read book Stem Cells and Regenerative Medicine written by Walter C. Low and published by World Scientific. This book was released on 2008 with total page 571 pages. Available in PDF, EPUB and Kindle. Book excerpt: The commercialization of biotechnology has resulted in an intensive search for new biological resources for the purposes of increasing food productivity, medicinal applications, energy production, and various other applications. Although biotechnology has produced many benefits for humanity, the exploitation of the planet's natural resources has also resulted in some undesirable consequences such as diminished species biodiversity, climate change, environmental contamination, and intellectual property right and patent concerns.This book discusses the role of biological, ecological, environmental, ethical, and economic issues in the interaction between biotechnology and biodiversity, using different contexts. No other book has discussed all of these issues in a comprehensive manner. Of special interest is their impact when biotechnology is shared between developed and developing countries, and the lack of recognition of the rights of indigenous populations and traditional farmers in developing countries by large multinational corporations.
