Gene Therapy in the CNS – Progress and Prospects for Novel Therapies
Author: Casper René Gøtzsche
Publisher: Frontiers Media SA
Published: 2021-12-21
Total Pages: 188
ISBN-13: 2889718522
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Gene Therapy of the Central Nervous System: From Bench to Bedside
Author: Michael G. Kaplitt
Publisher: Gulf Professional Publishing
Published: 2006
Total Pages: 380
ISBN-13: 9780123976321
DOWNLOAD EBOOK →Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology 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 in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who 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 which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.
Handbook of Innovations in Central Nervous System Regenerative Medicine
Author: António Salgado
Publisher: Elsevier
Published: 2020-06-10
Total Pages: 712
ISBN-13: 0128180854
DOWNLOAD EBOOK →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
Author: Mark H. Tuszynski
Publisher: Springer
Published: 2018-04-08
Total Pages: 584
ISBN-13: 9781493979424
DOWNLOAD EBOOK →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.
A Guide to Human Gene Therapy
Author: Roland W. Herzog
Publisher: World Scientific
Published: 2010
Total Pages: 415
ISBN-13: 9814280917
DOWNLOAD EBOOK →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
Leucine-Rich Repeat Kinase 2 (LRRK2)
Author: Hardy J. Rideout
Publisher: Springer
Published: 2017-03-28
Total Pages: 271
ISBN-13: 3319499696
DOWNLOAD EBOOK →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.
Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies
Author: Daniel Scherman
Publisher: World Scientific
Published: 2013-12-10
Total Pages: 557
ISBN-13: 1783263148
DOWNLOAD EBOOK →This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.
Central Nervous System Metastases
Author: Manmeet Ahluwalia
Publisher: Springer Nature
Published: 2019-11-05
Total Pages: 421
ISBN-13: 3030234177
DOWNLOAD EBOOK →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.
Modern Neurosurgery
Author: Dennis A. Turner
Publisher: CRC Press
Published: 2004-06-25
Total Pages: 341
ISBN-13: 0203503996
DOWNLOAD EBOOK →Focusing on how increased understanding of brain function affects clinical neuroscience, this incisive text explores the interface between neuroscience and clinical neuroscience advances by examining the hypotheses that drive this evolution. The author reviews the relevant underpinnings of new neurosurgical techniques, treatments, and conceptual ap
Textbook of Stereotactic and Functional Neurosurgery
Author: Andres M. Lozano
Publisher: Springer Science & Business Media
Published: 2009-06-22
Total Pages: 3247
ISBN-13: 3540699597
DOWNLOAD EBOOK →This book covers stereotactic principles as well as functional stereotaxis, covering the history and uses of the techniques, treatments for specific conditions, and future developments. Includes a DVD demonstrating surgical procedures.
