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Drug and Gene Delivery to the Central Nervous System for Neuroprotection

Drug and Gene Delivery to the Central Nervous System for Neuroprotection
Author: Hari S. Sharma
Publisher: Springer
Total Pages: 237
Release: 2017-06-05
Genre: Medical
ISBN: 3319576968
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This book summarizes the latest research on drug and gene delivery to the central nervous system (CNS). The chapters address safety concerns regarding the nanotechnology that is needed to develop nanomedicine for clinical practice. Particular focus is given to new technologies that have emerged in recent years to deliver therapeutic materials, such as genes, drugs, and other agents using nanotechnologies of diverse origin. This is an ideal book for students, teachers, researchers, and clinicians interested in a deeper understanding of nanotechnological advances in therapeutic medicine. This book also: Broadens readers’ understanding of viral vector gene delivery to the brain for treating neurogenetic diseases as well as targeted gene delivery into the brain using microbubble-facilitated focused ultrasound Covers in detail the latest developments in delivering therapeutic materials, such as siRNA delivery to the brain for treating neurological diseases, neuroprotective effects of gelatin nanoparticles in stroke, and nanowired drug delivery for brain diseases, heat stroke, and CNS injury Enriches understanding of new technologies for delivering therapeutic materials treating Alzheimer’s Disease, including targeted nanodrug delivery through the blood-brain barrier and the superior neuroprotective effects of nanowired drug delivery in Alzheimer’s Disease

Gene Therapy of the Central Nervous System: From Bench to Bedside

Gene Therapy of the Central Nervous System: From Bench to Bedside
Author: Michael G. Kaplitt
Publisher: Gulf Professional Publishing
Total Pages: 380
Release: 2006
Genre: Medical
ISBN: 9780123976321
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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.

Gene Therapy for the Central and Peripheral Nervous System

Gene Therapy for the Central and Peripheral Nervous System
Author: Andrew P. Tosolini
Publisher: Frontiers Media SA
Total Pages: 220
Release: 2018-05-10
Genre:
ISBN: 2889454754
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Gene therapy is at the forefront of current techniques that aim to re-establish functional connectivity, after an insult to the brain, spinal cord or peripheral nerves. Gene therapy makes the most of the existing cellular machinery and anatomical networks to facilitate molecular changes in DNA, RNA and proteins aiming to repair these disrupted connections. For instance, gene therapy is currently being used to target genes in conditions including spinal cord injury, amyotrophic lateral sclerosis, spinal muscular atrophy, stroke and multiple sclerosis, amongst others. The various delivery routes include viral-vectors, genetically modified cellular implants, naked DNA/RNA, liposomes, Cre-Lox recombination, optogenetics and nanoparticles. In particular, gene therapy aims to restore function by augmenting the expression of neuroprotective/axonal growth-promoting neurotrophic factors (e.g., BDNF, CNTF, NGF and GDNF, etc.). Furthermore, the downstream intracellular signalling pathways after receptor activation can also be targeted (e.g., mTor, MAPK, etc.). On the other hand, gene therapy can also be used to downregulate and/or remove faulty mutated genes, such as those contributing to disease progression or that inhibit axonal regeneration (e.g., SOD-1, TDP-43, Nogo-A, MAG, OmGP, etc.). Depending on the methodology, these genes, for instance, can be silenced, removed or replaced to alleviate the underlying pathology. As such, gene therapy can transform a largely toxic and inhibitory milieu surrounding a neuronal/axonal insult into a growth-permissive environment that will ultimately aid neuronal survival and functional regeneration. Moreover, gene therapy has the capacity to target non-neuronal cells and can be even used for neuroanatomical tract tracing. Ultimately, the principal outcome of gene therapy is to functionally restore damaged neuronal and/or axonal connections irrespective of the system it is being introduced in to. This Research Topic is devoted to work using gene therapy for the both the central and/or peripheral nervous system.

Drug Delivery to the Central Nervous System

Drug Delivery to the Central Nervous System
Author: Kewal K. Jain
Publisher: Humana
Total Pages: 321
Release: 2016-08-23
Genre: Medical
ISBN: 9781493961597
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Playing an important role in the treatment of neurological disorders, the delivery of drugs to central nervous system (CNS), both administered directly and administered systematically for targeted action, encounters a major challenge in the form of the blood-brain barrier (BBB), which limits the access of drugs to the brain substance. In Drug Delivery to the Central Nervous System, experts in the field present essential methods used to deliver therapeutics across the BBB, both in experimental animals and in humans. In addition to those methods, several overviews of innovative methods and their applications are presented in order to give a glimpse of the future of this research. As a volume in the successful Neuromethods series, this book presents its protocols with the kind of detailed description and implementation advice that is crucial for getting optimal results. Authoritative and cutting-edge, Drug Delivery to the Central Nervous System serves as an ideal guide to scientists continuing to pursue knowledge of the delicate interactions between pharmaceuticals and the brain.

Drug Delivery to the Brain

Drug Delivery to the Brain
Author: Margareta Hammarlund-Udenaes
Publisher: Springer Science & Business Media
Total Pages: 737
Release: 2013-12-03
Genre: Medical
ISBN: 1461491053
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The development of new CNS drugs is notoriously difficult. Drugs must reach CNS target sites for action and these sites are protected by a number of barriers, the most important being the blood –brain barrier (BBB). Many factors are therefore critical to consider for CNS drug delivery, e.g. active/passive transport across the BBB, intra-brain distribution, and central/systemic pharmacokinetics, to name a few. Neurological disease and trauma conditions add further complexity because CNS barriers, drug distribution and pharmacokinetics are dynamic and often changed by disease/trauma. Knowledge of all these factors and their interplay in different conditions is of utmost importance for proper CNS drug development and disease treatment. In recent years much information has become available for a better understanding of the many factors important for CNS drug delivery and how they interact to affect drug action. This book describes small and large drug delivery to the brain with an emphasis on the physiology of the BBB and the principles and concepts for drug delivery across the BBB and distribution within the brain. It contains methods descriptions for studying drug delivery, routes and approaches of administering drugs into the brain, the influence of disease, and drug industry perspectives. Therewith, it contributes to an in-depth understanding of the interplay between brain (patho)-physiology and drug characteristics. Furthermore, the content is designed to be both cutting-edge and educational, so that the book can be used in high-level training of academic and industry scientists with full references to original publications. ​

Gene Transfer and Therapy in the Nervous System

Gene Transfer and Therapy in the Nervous System
Author: Fred H. Gage
Publisher: Springer Science & Business Media
Total Pages: 226
Release: 2012-12-06
Genre: Medical
ISBN: 3642848427
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Gene transfer technology is a powerful tool for increasing our understandingof brain functions. It is also the basis of gene therapy, which is now technically possible for the correction of many human diseases, including several disorders of the nervous (and muscular) system such as Alzheimer's disease, Parkinson's disease, and dystrophy. This volume,which contains the proceedings of a symposium of the Fondation Ipsen, provides a unique view of the state of the art on different transgenes, vectors, target cells, and clinical applications related to the nervous system.

Gene Delivery Into the Central Nervous System (CNS) Using AAV Vectors

Gene Delivery Into the Central Nervous System (CNS) Using AAV Vectors
Author: Kōichi Miyake
Publisher:
Total Pages:
Release: 2015
Genre: Medicine
ISBN:
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Application of gene therapies is a promising approach to the treatment of various neurological disorders, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lysosomal storage disorders, which are not treatable by any other means. However, the blood-brain barrier (BBB) is a key obstacle to gene delivery to the central nervous system (CNS). Adeno-associated virus (AAV) vectors have emerged as a promising tool for gene delivery to the CNS, thanks to their safety and ability to transduce non-dividing neuronal cells. In this chapter, we discuss strategies for delivering genes across the BBB, focusing especially on potential routes of administration of AAV vectors and promising applications of AAV vectors to the treatment of CNS disorders.

Handbook of Innovations in Central Nervous System Regenerative Medicine

Handbook of Innovations in Central Nervous System Regenerative Medicine
Author: António Salgado
Publisher: Elsevier
Total Pages: 712
Release: 2020-06-10
Genre: Medical
ISBN: 0128180854
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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

New Perspectives of Central Nervous System Injury and Neuroprotection

New Perspectives of Central Nervous System Injury and Neuroprotection
Author:
Publisher: Academic Press
Total Pages: 419
Release: 2012-10-22
Genre: Medical
ISBN: 012387016X
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Central nervous system (CNS) diseases such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis affect a large number of populations worldwide for which no suitable cure is currently available. In addition, stroke, nanoparticle intoxication, diabetes, hypertension, and psychostimulant abuse either alone or in combination are capable of inducing potential brain damage. Thus, there is an urgent need to expand our knowledge to find suitable therapeutic strategies to enhance neurorepair processes in such diseases.This volume presents neuroprotection and novel therapeutic strategies developed in the last 5 years by 12 world leaders in the field. The term neuroprotection means rescuing neuronal and non-neuronal cells together. The cerebral endothelium that constitutes the anatomical and physiological site of the blood-brain barrier (BBB) is one of the most important non-neural cells in the CNS. Any distortion of the BBB leads to brain diseases and restoration of the barrier results in neuroprotection. Thus, the BBB appears to be the "gateway" for neurological diseases and neurorepair. However, to treat brain tumors or infarcts, new therapeutic strategies are needed to enhance brain drug delivery using nanotechnology. In addition, apart from conventional drugs, restoration of BBB function could also be achieved by means of antibodies directed against specific proteins, neurotransmitters or exogenous supplement of neurotrophic factors. Since co-morbidity factors e.g., hypertension, diabetes, and exposure of nanoparticles could complicate the pathogenesis of neurological disorders either an enhanced dose of the drug or nanodelivery of a combination of several drugs is needed to achieve neuroprotection.This volume of International Review of Neurobiology is the first to discuss novel therapeutic strategies in situations of neurological disorders in combination with different co-morbidity factors. Reviews written by experts in such a way that provides basic knowledge for beginners and advanced information for researchers and experts New aspects of Neurodegenerative diseases such as; Alzheimer’s Disease, Parkinson’s Disease, Amyotrophic Lateral Sclerosis are presented with the latest therapeutic measures Exacerbation of brain pathology in hypertension or diabetes is discussed for the first time

Protocols for Gene Transfer in Neuroscience

Protocols for Gene Transfer in Neuroscience
Author: P. R. Lowenstein
Publisher:
Total Pages: 468
Release: 1996-07-25
Genre: Medical
ISBN:
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Protocols for Gene Transfer in Neuroscience Towards Gene Therapy of Neurological Disorders Edited by P. R. Lowenstein University of Manchester, UK and L. W. Enquist Princeton University, USA The brain and the cells that compose it have always presented unique barriers to any kind of outside interventions, including drug delivery, surgical procedures and the genetic manipulation of cells. However, these barriers are now being bypassed by the new technology of gene delivery. Protocols for Gene Transfer in Neuroscience is the first practical guide to gene delivery, providing over 150 easy to follow, step-by-step protocols, together with troubleshooting hints and the theoretical background needed for neuroscientists to understand, adapt and expand the technology as needed. Written by a team of international expert neuroscientists, geneticists, molecular biologists and biochemists, Protocols for Gene Transfer in Neuroscience describes the latest techniques from the top gene-delivery labs, ranging from physical transfection techniques, through the use of a wide range of viral vectors, to retrovirally transduced cell and bone marrow transplantation. Several variations of a single protocol are given, thus demonstrating how different labs have solved a common problem in different ways. The protocols are also indexed and cross-referenced so that an investigator can find all the details required for a particular problem.