Antisense Technology In The Central Nervous System
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Antisense Technology In The Central Nervous System
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Author : Ronald A. Leslie
language : en
Publisher: Oxford University Press, USA
Release Date : 1999
Antisense Technology In The Central Nervous System written by Ronald A. Leslie and has been published by Oxford University Press, USA this book supported file pdf, txt, epub, kindle and other format this book has been release on 1999 with Medical categories.
"Antisense Technology in the Central Nervous System describes the promises and the pitfalls of this approach, and compares it to other approaches. The practical experience of leading researchers is combined with a thorough description of the theoretical foundation of the technique, making the book ideal for researchers and clinicians alike."--BOOK JACKET.
Intrathecal Delivery Of Antisense Oligonucleotide Therapeutics
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Author : Brynna Wilken-Resman
language : en
Publisher:
Release Date : 2019
Intrathecal Delivery Of Antisense Oligonucleotide Therapeutics written by Brynna Wilken-Resman and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019 with categories.
Antisense oligonucleotide (ASO) therapies for central nervous system (CNS) indications have advanced significantly within the past three years, including FDA approval for an ASO therapy for spinal muscular atrophy and clinical trials for several other neurological disorders. ASOs are ~6-8 kDa, single-stranded DNA or RNA oligomers and are attractive therapeutic candidates for diseases caused by known genetic abnormalities because they are disease-modifying therapeutics that can interact with target RNA to modify protein production. ASOs are unable to cross the blood-brain barrier on their own, so they are administered centrally, typically achieved by intrathecal administration. Despite the clinical use of CSF-administered ASOs, there is a paucity of knowledge concerning CSF-to-brain transport mechanisms and the resulting CNS biodistribution. Here, we investigated ASO transport and distribution in the CNS following intrathecal administration, focusing on the effects of different ASO chemistries and two transport mechanisms: diffusion in the extracellular spaces and convection/dispersion in perivascular spaces (PVS) surrounding the cerebral vasculature. We have demonstrated that intrathecal administration of fluorescently-labeled ASOs in rats leads to limited diffusion at CSF-brain and CSF-spinal cord interfaces and rapid distribution within the PVS. ASOs distributed to the PVS surrounding vessels of all type and caliber. We have also demonstrated that the extent to which perivascular distribution occurs differs in a sequence- and chemical modification-specific manner, a finding not yet reported in the literature. Furthermore, we have found that ASOs with a nucleotide sequence that allowed a random coil conformation had a significantly smaller hydrodynamic size compared to ASOs that formed secondary structures and that smaller hydrodynamic size correlated with more extensive diffusion and perivascular access. The difference in perivascular signal between the PS-ASO and the 2'MOE-ASO was significant when quantified and was particularly apparent in the dorsal cortex as well as in subcortical brain regions such as the striatum and hippocampus. Biodistribution and transport studies such as those conducted in the present work may provide an improved framework for understanding ASO delivery to brain and spinal cord targets. It is our hope that this framework may assist in developing ASO therapies for optimal CNS distribution to improve treatment results for devastating neurological conditions.
Genetic Manipulation Of The Nervous System
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Author :
language : en
Publisher: Elsevier
Release Date : 1995-11-24
Genetic Manipulation Of The Nervous System written by and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 1995-11-24 with Science categories.
Neuroscience Perspectives provides multidisciplinary reviews of topics in one of the most diverse and rapidly advancing fields in the life sciences. Whether you are a new recruit to neuroscience, or an established expert, look to this series for 'one-stop' sources of the historical, physiological, pharmacological, biochemical, molecular biological and therapeutic aspects of chosen research areas. The recent development of Gene Therapy procedures which allow specific genes to be delivered to human patients who lack functional copies of them is of major therapeutic importance. In addition such gene delivery methods can be used in other organisms to define the function of particular genes. These studies are of particular interest in the nervous system where there are many incurable diseases like Alzheimer's and Parkinson's diseases which may benefit from therapies of this kind. Unfortunately gene delivery methods for use in the nervous system have lagged behind those in other systems due to the fact that the methods developed in other systems are often not applicable to cells like neurons which do not divide. This book discusses a wide range of methods which have now been developed to overcome these problems and allow safe and efficient delivery of particular genes to the brain. Methods discussed include virological methods, physical methods (such as liposomes) and the transplantation of genetically modified cells. In a single volume therefore this book provides a complete view of these methods and indicates how they can be applied to the development of therapies for treating previously incurable neurological disorders.
Antisense Technology Part B Applications
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Author :
language : en
Publisher: Elsevier
Release Date : 1999-11-10
Antisense Technology Part B Applications written by and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 1999-11-10 with Science categories.
Antisense technology is the ability to manipulate gene expression within mammalian cells providing powerful experimental approaches for the study of gene function and gene regulation. For example, methods which inhibit gene expression permit studies probing the normal function of a specific product within a cell. Such methodology can be used in many disciplines such as pharmacology, oncology, genetics, cell biology, developmental biology, molecular biology, biochemistry, and neurosciences. This volume will be a truly important tool in biomedically-oriented research. The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today-truly an essential publication for researchers in all fields of life sciences.
Polyglutamine Disorders
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Author : Clévio Nóbrega
language : en
Publisher: Springer
Release Date : 2018-02-09
Polyglutamine Disorders written by Clévio Nóbrega and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-02-09 with Medical categories.
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.
Antisense Technology Part A General Methods Methods Of Delivery And Rna Studies
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Author :
language : en
Publisher: Elsevier
Release Date : 1999-11-01
Antisense Technology Part A General Methods Methods Of Delivery And Rna Studies written by and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 1999-11-01 with Science categories.
Antisense technology is the ability to manipulate gene expression within mammalian cells providing powerful experimental approaches for the study of gene function and gene regulation. For example, methods that inhibit gene expression permit studies which probe the normal function of a specific product within a cell. Such methodology can be used in many disciplines such as pharmacology, oncology, genetics, cell biology, developmental biology, molecular biology, biochemistry, and neurosciences. This volume will be a truly important tool in biomedical-oriented research. The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today--truly an essential publication for researchers in all fields of life sciences.
Antisense Drug Technology
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Author : Stanley T. Crooke
language : en
Publisher: CRC Press
Release Date : 2001-09-14
Antisense Drug Technology written by Stanley T. Crooke and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2001-09-14 with Medical categories.
This state-of-the-art reference provides comprehensive coverage of the development of antisense oligonucleotides to inhibit cancer cells as well as those involved in infectious, inflammatory, and immune-mediated diseases-highlighting new tools and technologies in medicinal chemistry, RNA biochemistry, and molecular and cellular biology to produce new therapeutic compounds. Presents previously unpublished data on the use of antisense technology to dissect pharmacological processes and confirm the roles of various genes! Showcasing the benefits of antisense drug use, including reduced toxicity and earlier disease detection, Antisense Drug Technology discusses novel formulations of antisense drugs practical methods to design effective isotype selective inhibitors molecular mechanisms of antisense drugs mRNA as a current biological template modern postreceptor binding mechanisms and more! With contributions by over 60 seasoned experts in the field and containing more than 3000 helpful references, tables, drawings, and photographs, Antisense Drug Technology is an illuminating source for organic, medicinal, and pharmaceutical chemists; biochemists; geneticists; hematologists; oncologists; molecular and cell biologists; virologists; immunologists; and medical school and graduate students in these disciplines.
Central Nervous System Diseases
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Author : Dwaine F. Emerich
language : en
Publisher: Springer Science & Business Media
Release Date : 1999-11-24
Central Nervous System Diseases written by Dwaine F. Emerich and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 1999-11-24 with Medical categories.
Prominent experimentalists critically review the animal models widely used in developing powerful new therapies for central nervous system diseases. Coverage includes novel uses of animal models of Alzheimer's, Parkinson's, and Huntington's diseases, and studies of aging. Techniques that rely heavily on behavioral analyses, as well as models developed from infusions of neurotoxins and from advances in molecular biology, are thoroughly explicated, as are models developed for more acute neurological conditions, including traumatic brain injury and stroke. Comprehensive and authoritative, Central Nervous System Diseases: Innovative Animal Models from Lab to Clinic offers neuroscientists, pharmacologists, and interested clinicians a unique survey of the most productive animal models of the leading neurological diseases currently employed to develop today's innovative drug therapies.
Antisense Therapeutics
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Author : Sudhir Agrawal
language : en
Publisher:
Release Date : 1996
Antisense Therapeutics written by Sudhir Agrawal and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1996 with Antisense nucleic acids categories.
Annotation Dr. Agrawal's newest book is of significant importance since oligonucleotide-based drugs have the advantage of retaining their recognition for the target RNA, and provide the tools needed to modulate the expression of specific genes, making Antisense Therapeutics an essential resource for all antisense researchers.
Central Nervous System Diseases New Insights For The Healthcare Professional 2012 Edition
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Author :
language : en
Publisher: ScholarlyEditions
Release Date : 2012-12-10
Central Nervous System Diseases New Insights For The Healthcare Professional 2012 Edition written by and has been published by ScholarlyEditions this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-12-10 with Medical categories.
Central Nervous System Diseases: New Insights for the Healthcare Professional / 2012 Edition is a ScholarlyEditions™ eBook that delivers timely, authoritative, and comprehensive information about Central Nervous System Diseases. The editors have built Central Nervous System Diseases: New Insights for the Healthcare Professional / 2012 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Central Nervous System Diseases in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Central Nervous System Diseases: New Insights for the Healthcare Professional / 2012 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.