Gene Therapy in the CNS – Progress and Prospects for Novel Therapies

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies PDF Author: Casper René Gøtzsche
Publisher: Frontiers Media SA
ISBN: 2889718522
Category : Science
Languages : en
Pages : 188

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Book Description

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies PDF Author: Casper René Gøtzsche
Publisher: Frontiers Media SA
ISBN: 2889718522
Category : Science
Languages : en
Pages : 188

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Book Description


Neurotherapy

Neurotherapy PDF Author: Joost Verhaagen
Publisher: Elsevier
ISBN: 0080922961
Category : Medical
Languages : en
Pages : 546

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Book Description
This book focuses on the exciting recent progress in restorative neurology and neuroscience. The book includes chapters on major neurodegenerative disorders of the brain and the visual system, including Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis, Huntington's disease, macular degeneration, retinitis pigmentosa, glaucoma, spinal cord trauma, and multiple sclerosis. The primary goal of the book is to give an overview of new developments in translational research and in potential therapeutic strategies, including stem cell therapy, immunotherapy, gene therapy, pharmacotherapy, neuroprostheses and deep brain stimulation. - Provides the reader with a unique overview over all aspects of new advances in the therapy of neurological and psychiatric disorders - Covers all levels of biological organization including novel molecular and cellular targets, electrophysiological, anatomical and behavioural substrates of neurodegeneration and the application of whole brain in vivo imaging - Broad focus with contributions by the top scientists worldwide in the respective disciplines

A Guide to Human Gene Therapy

A Guide to Human Gene Therapy PDF Author: Roland W. Herzog
Publisher: World Scientific
ISBN: 9814280917
Category : Medical
Languages : en
Pages : 415

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Book Description
1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Handbook of Innovations in Central Nervous System Regenerative Medicine

Handbook of Innovations in Central Nervous System Regenerative Medicine PDF Author: António Salgado
Publisher: Elsevier
ISBN: 0128180854
Category : Medical
Languages : en
Pages : 712

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Book Description
Handbook of Innovations in CNS Regenerative Medicine provides a comprehensive overview of the CNS regenerative medicine field. The book describes the basic biology and anatomy of the CNS and how injury and disease affect its balance and the limitations of the present therapies used in the clinics. It also introduces recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies. Finally, the book presents successful cases of translation of basic research to first-in-human trials and the steps needed to follow this path. Areas such as cell transplantation approaches, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies are key in regenerative medicine are covered in the book, along with regulatory and ethical issues. - Describes the basic biology and anatomy of the CNS and how injury and disease affect its balance - Discusses the limitations of present therapies used in the clinics - Introduces the recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies, and enabling technologies - Presents successful cases of translation of basic research to first-in-human trials, along with the steps needed to follow this path

Translational Neuroscience

Translational Neuroscience PDF Author: Mark H. Tuszynski
Publisher: Springer
ISBN: 148997654X
Category : Medical
Languages : en
Pages : 584

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Book Description
Translational Neuroscience offers a far-reaching and insightful series of perspectives on the effort to bring potentially revolutionary new classes of therapies to the clinic, thereby transforming the treatment of human nervous system disorders. Great advances in the fields of basic neuroscience, molecular biology, genomics, gene therapy, cell therapy, stem cell biology, information technology, neuro devices, rehabilitation and others over the last 20 years have generated unprecedented opportunities to treat heretofore untreatable disorders of the nervous system. This book provides a wide-ranging yet detailed sample of many of these efforts, together with the methods for pursuing clinical translation and assessing clinical outcomes. Among the topics covered are Alzheimer’s disease, Parkinson’s disease, stroke, multiple sclerosis, epilepsy, motor neuron disease, pain, inborn errors of metabolism, brain tumors, spinal cord injury, neuroprosthetics, rehabilitation and clinical trial design/consideration. Translational Neuroscience is aimed at basic neuroscientists, translational neuroscientists and clinicians who seek to gain a perspective on the nature and promise of translational therapies in the current era. Both students and established professionals will benefit from the content.

Gene Therapy of Autoimmune Disease

Gene Therapy of Autoimmune Disease PDF Author: Gerald J. Prud'homme
Publisher: Springer
ISBN: 9780306479915
Category : Medical
Languages : en
Pages : 149

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Book Description
Autoimmune diseases are diverse and responsible for considerable morbidity. Their etiology remains largely unknown, and current therapy with anti-inflammatory drugs is prone to adverse effects, and rarely curative. New therapies with anti-cytokine antibodies or receptors are promising, but require frequent administration of expensive protein drugs. Gene Therapy of Autoimmune Diseases comprehensively reviews research in gene therapy for autoimmune diseases with viral or non-viral vectors. Gene therapy offers the possibility of long-term, continuous delivery of a wide variety of immunosuppressive, anti-inflammatory, or tolerance-inducing agents. Moreover, highly specific genetically modified cells can be produced. This book discusses the most promising avenues in this exciting new field.

Leucine-Rich Repeat Kinase 2 (LRRK2)

Leucine-Rich Repeat Kinase 2 (LRRK2) PDF Author: Hardy J. Rideout
Publisher: Springer
ISBN: 3319499696
Category : Medical
Languages : en
Pages : 280

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Book Description
This is the first book to assemble the leading researchers in the field of LRRK2 biology and neurology and provide a snapshot of the current state of knowledge, encompassing all major aspects of its function and dysfunction. The contributors are experts in cell biology and physiology, neurobiology, and medicinal chemistry, bringing a multidisciplinary perspective on the gene and its role in disease. The book covers the identification of LRRK2 as a major contributor to the pathogenesis of Parkinson's Disease. It also discusses the current state of the field after a decade of research, putative normal physiological roles of LRRK2, and the various pathways that have been identified in the search for the mechanism(s) of its induction of neurodegeneration.

Multiple Sclerosis

Multiple Sclerosis PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 030917130X
Category : Medical
Languages : en
Pages : 456

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Book Description
Multiple sclerosis is a chronic and often disabling disease of the nervous system, affecting about 1 million people worldwide. Even though it has been known for over a hundred years, no cause or cure has yet been discovered-but now there is hope. New therapies have been shown to slow the disease progress in some patients, and the pace of discoveries about the cellular machinery of the brain and spinal cord has accelerated. This book presents a comprehensive overview of multiple sclerosis today, as researchers seek to understand its processes, develop therapies that will slow or halt the disease and perhaps repair damage, offer relief for specific symptoms, and improve the abilities of MS patients to function in their daily lives. The panel reviews existing knowledge and identifies key research questions, focusing on: Research strategies that have the greatest potential to understand the biological mechanisms of recovery and to translate findings into specific strategies for therapy. How people adapt to MS and the research needed to improve the lives of people with MS. Management of disease symptoms (cognitive impairment, depression, spasticity, vision problems, and others). The committee also discusses ways to build and financially support the MS research enterprise, including a look at challenges inherent in designing clinical trials. This book will be important to MS researchers, research funders, health care advocates for MS research and treatment, and interested patients and their families.

Central Nervous System Metastases

Central Nervous System Metastases PDF Author: Manmeet Ahluwalia
Publisher: Springer Nature
ISBN: 3030234177
Category : Medical
Languages : en
Pages : 411

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Book Description
This book provides a comprehensive overview of brain metastases, from the molecular biology aspects to therapeutic management and perspectives. Due to the increasing incidence of these tumors and the urgent need to effectively control brain metastatic diseases in these patients, new therapeutic strategies have emerged in recent years. The volume discusses all these innovative approaches combined with new surgical techniques (fluorescence, functional mapping, integrated navigation), novel radiation therapy techniques (stereotactic radiosurgery) and new systemic treatment approaches such as targeted- and immunotherapy. These combination strategies represent a new therapeutic model in brain metastatic patients in which each medical practitioner (neurosurgeon, neurologist, medical oncologist, radiation oncologist) plays a pivotal role in defining the optimal treatment in a multidisciplinary approach. Written by recognized experts in the field, this book is a valuable tool for neurosurgeons, neuro-oncologists, neuroradiologists, medical oncologists, radiation oncologists, cognitive therapists, basic scientists and students working in the area of brain tumors.

Neuronal Degeneration and Regeneration: From Basic Mechanisms to Prospects for Therapy

Neuronal Degeneration and Regeneration: From Basic Mechanisms to Prospects for Therapy PDF Author: F.W. Van Leeuwen
Publisher: Elsevier
ISBN: 008086242X
Category : Psychology
Languages : en
Pages : 551

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Book Description
This book is the result of the 20th International Summer School in Brain Research, organized in August 1997 in Amsterdam, by the Netherlands Institute for Brain Research at the Royal Netherlands Academy of Sciences. It is the first book that provides a complete overview of the field of neurodegeneration and regeneration including spinal cord injury, neurodegenerative diseases and therapy. Divided into five sections, the first two sections give an overview of fundamental research on nerve cell death, neuronal survival, neurite outgrowth and guidance. Extensive attention is given to the role of neurotrophins, their receptor tyrosine kinases and cell-adhesion molecules in development and regeneration of the nervous system. The third section of the book is devoted to research involving human neurodegenerative diseases and emerging treatment strategies. Section four focusses on recent advances in the understanding of pathophysiological mechanisms underlying neurodegenerative diseases, including Alzheimer's, Parkinson's and Huntington's diseases along with prion diseases. Novel insights into the neuropathological hallmarks of these diseases, as well as into transgenic animal models, the involvement of environmental factors, and genomic and mRNA changes that can cause neurodegeneration. The final section of this volume reveals recent developments in the use of cell and gene therapy to treat neurodegenerative disease and lesion-related deficits. Implantation of genetically modified cells, direct gene transfer with viral vectors and the first clinical trials with encapsulated genetically modified cells in patients suffering from amyotrophic lateral scelerosis are examples of new therapeutic strategies treating neurodegenerative diseases.The book is of particular interest to departments of neuroscience, neurological clinics and departments, the pharmalogical industry and medical libraries.