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Gene Silencing And Editing Strategies For Neurodegenerative Diseases
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Book Synopsis Gene Silencing and Editing Strategies for Neurodegenerative Diseases by : Clévio Nóbrega
Download or read book Gene Silencing and Editing Strategies for Neurodegenerative Diseases written by Clévio Nóbrega and published by Frontiers Media SA. This book was released on 2018-08-31 with total page 115 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Gene Silencing and Editing Strategies for Neurodegenerative Diseases by :
Download or read book Gene Silencing and Editing Strategies for Neurodegenerative Diseases written by and published by . This book was released on 2018 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs. The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult. Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology.
Book Synopsis Genome Editing for Neurodegenerative Diseases by : Sandeep Kumar Singh
Download or read book Genome Editing for Neurodegenerative Diseases written by Sandeep Kumar Singh and published by Elsevier. This book was released on 2024-11-01 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials examines recent advances in neurodegenerative disease research and clinical challenges in practical applications. With an emphasis on genome editing tools such as ZFNs, TALENSs and CRISPR-Cas, this book sheds light on the possibilities to counteract genetic and neurodegenerative diseases. It will be of interest to researchers in neuroscience and biopharmaceuticals, as well as clinicians specializing in neurodegenerative diseases. Covers basic mechanisms of genome-editing tools and their therapeutic potential Highlights current research progress and therapeutic strategies to use genome-editing tools against neurodegenerative diseases Includes research on genome-editing tool development, from basic concept to clinical trials
Book Synopsis Ocular neurodegenerative diseases: Novel mechanisms, diagnosis, and therapeutic strategies by : Dan Wen
Download or read book Ocular neurodegenerative diseases: Novel mechanisms, diagnosis, and therapeutic strategies written by Dan Wen and published by Frontiers Media SA. This book was released on 2023-11-06 with total page 187 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Neurodegenerative Diseases by : Nagehan Ersoy Tunalı
Download or read book Neurodegenerative Diseases written by Nagehan Ersoy Tunalı and published by BoD – Books on Demand. This book was released on 2021-01-20 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurodegenerative diseases represent a very large group of heterogeneous disorders affecting specific subtypes of neurons in the brain. This book contributes insight both to the awareness of the brain and its neurodegenerative states. The chapters present current knowledge regarding genetics, molecular mechanisms, and new therapeutic strategies against neurodegenerative disorders. The book is intended to serve as a source to aid clinicians and researchers in the field, and also life science readers to increase their understanding and awareness of the clinical correlations, genetic aspects, neuropathological findings, and current therapeutic interventions in neurodegenerative diseases. I believe that this book will enlighten the curiosity for neurodegeneration and also encourage researchers to work on potentially effective molecular therapies for still mysterious neurodegenerative disorders.
Book Synopsis Disease-Modifying Targets in Neurodegenerative Disorders by : Veerle Baekelandt
Download or read book Disease-Modifying Targets in Neurodegenerative Disorders written by Veerle Baekelandt and published by Academic Press. This book was released on 2017-03-31 with total page 324 pages. Available in PDF, EPUB and Kindle. Book excerpt: Disease-Modifying Targets in Neurodegenerative Disorders: Paving the Way for Disease-Modifying Therapies examines specific neurodegenerative disorders in comprehensive chapters written by experts in the respective fields. Each chapter contains a summary of the disease management field, subsequently elaborating on the molecular mechanisms and promising new targets for disease-modifying therapies. This overview is ideal for neuroscientists, biomedical researchers, medical doctors, and caregivers, not only providing readers with a summary of the way patients are treated today, but also offering a glance at the future of neurodegenerative disorder treatment. Provides a comprehensive overview of how key proteins in neurodegenerative disorders can be used as targets to modify disease progress Summarizes how patients are treated today, providing a glance at future disease management Includes intelligible and informative information that is perfect for non-specialists, medical practitioners, and scientists Written and peer reviewed by outstanding scientists in their respective fields
Book Synopsis Neurodegenerative Diseases by : Philip Beart
Download or read book Neurodegenerative Diseases written by Philip Beart and published by Springer. This book was released on 2017-07-03 with total page 542 pages. Available in PDF, EPUB and Kindle. Book excerpt: Provides a timely overview of critical advances in molecular and cellular neurobiology, covers key methodologies driving progress, and highlights key future directions for research on neuronal injury and neurodegeneration relevant to neuronal brain pathologies. The editors bring together contributions from internationally recognized workers in the field to provide an up to date account of how and why molecular and cellular neurobiology is such an important area for clinical neuroscience. Understanding the molecular aspects of a number of neurodegenerative conditions such as Parkinson's or Alzheimer's disease for the purpose of improving patient management remains a major challenge of neurobiology be it from the basic or clinical perspective. A strategic evaluation of research contributions and the power of modern methods will help advance knowledge over the next years.
Book Synopsis The Molecular and Cellular Basis of Neurodegenerative Diseases by : Michael S. Wolfe
Download or read book The Molecular and Cellular Basis of Neurodegenerative Diseases written by Michael S. Wolfe and published by Academic Press. This book was released on 2018-03-29 with total page 560 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Molecular and Cellular Basis of Neurodegenerative Diseases: Underlying Mechanisms presents the pathology, genetics, biochemistry and cell biology of the major human neurodegenerative diseases, including Alzheimer’s, Parkinson’s, frontotemporal dementia, ALS, Huntington’s, and prion diseases. Edited and authored by internationally recognized leaders in the field, the book's chapters explore their pathogenic commonalities and differences, also including discussions of animal models and prospects for therapeutics. Diseases are presented first, with common mechanisms later. Individual chapters discuss each major neurodegenerative disease, integrating this information to offer multiple molecular and cellular mechanisms that diseases may have in common. This book provides readers with a timely update on this rapidly advancing area of investigation, presenting an invaluable resource for researchers in the field. Covers the spectrum of neurodegenerative diseases and their complex genetic, pathological, biochemical and cellular features Focuses on leading hypotheses regarding the biochemical and cellular dysfunctions that cause neurodegeneration Details features, advantages and limitations of animal models, as well as prospects for therapeutic development Authored by internationally recognized leaders in the field Includes illustrations that help clarify and consolidate complex concepts
Book Synopsis Handbook of Innovations in Central Nervous System Regenerative Medicine by : António Salgado
Download or read book Handbook of Innovations in Central Nervous System Regenerative Medicine written by António Salgado and published by Elsevier. This book was released on 2020-06-10 with total page 712 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Direct Nose-to-Brain Drug Delivery by : Chandrakantsing Pardeshi
Download or read book Direct Nose-to-Brain Drug Delivery written by Chandrakantsing Pardeshi and published by Academic Press. This book was released on 2021-06-16 with total page 556 pages. Available in PDF, EPUB and Kindle. Book excerpt: Direct Nose-to-Brain Drug Delivery provides the reader with precise knowledge about the strategies and approaches for enhanced nose-to-brain drug delivery. It highlights the development of novel nanocarrier-based drug delivery systems for targeted drug delivery to the brain microenvironments with a focus on the technological advances in the development of the novel drug delivery devices for intranasal administration, including special emphasis on brain targeting through nose. This book explores the various quantification parameters to assess the brain targeting efficiency following intranasal administration and includes an overview on the toxicity aspects of the various materials used to develop the direct nose-to-brain drug delivery vehicles and of the regulatory aspects including patents and current clinical status of the potential neurotherapeutics for the effective management of neuro-ailments. Technological advances in new drug delivery systems with diverse applications in pharmaceutical, biomedical, biomaterials, and biotechnological fields are also explained. This book is a crucial source that will assist the veteran scientists, industrial technologists, and clinical research professionals to develop new drug delivery systems and novel drug administration devices for the treatment of neuro-ailments. Explains the targeting approaches for enhanced brain targeting following intranasal drug administration Explores the various nanocarriers developed to date for neurotherapeutic delivery via nose-to-brain Discusses pharmaceutical and biomedical applications after nose-to-brain delivery of therapeutic pharmaceuticals and biologicals
Book Synopsis Public Health and Toxicology Issues in Drug Research, Volume 2 by : Rakesh Kumar Tekade
Download or read book Public Health and Toxicology Issues in Drug Research, Volume 2 written by Rakesh Kumar Tekade and published by Elsevier. This book was released on 2024-04-05 with total page 854 pages. Available in PDF, EPUB and Kindle. Book excerpt: Toxicodynamics in Drug Research, Volume 2: Public Health and Toxicology Issues examines the implications of public health issues and the impact of pharmaceuticals, chemical and food toxicants, dietary phytochemicals, and medical treatments on human health. Volume 2: Public Health and Toxicology Issues in Drug Research: Toxicity and Toxicodynamics covers topics on pharmacokinetics and toxicokinetics such as population pharmacokinetics/toxicokinetics, the design of toxicokinetic studies, and the use of toxicokinetic data in preclinical safety assessments. The book investigates the health effect caused by the bioaccumulation of pharmaceutical and personal care products and the impact of drug-induced toxicity on different systems of the body. It discusses the mechanistic pathways of food toxicants and illustrates the molecular mechanisms of the chemopreventive role of dietary phytochemicals. It also delves into the toxic effects of medical treatments and materials. Ethical, legal, societal, and professional issues in toxicology round off the coverage providing a valuable resource to interested in learning more about the health impact and public health issues related to the toxicity of pharmaceuticals, dietary supplements, personal care products, and medical treatments. Discusses the impact of pharmaceuticals, food, and chemical toxicants on human health Examines the toxic effects of medical treatments, clinical administrations, and materials Explores public health issues around drug safety and toxicology
Book Synopsis Neurogenetics for the Practitioner by : Gregory M. Pastores
Download or read book Neurogenetics for the Practitioner written by Gregory M. Pastores and published by Elsevier. This book was released on 2024-04-26 with total page 512 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurogenetics is a growing field, providing a clear link between clinical characteristics of phenotypes and exact molecular tests to reach a specific diagnosis. Neurogenetics for the Practitioner provides clinicians with a navigation tool to help diagnose and treat patients with neurological disorders using neurogenetics. The first section introduces the reader to an overview of genetic principles, including practical applications in relation to diagnosis and current limitations. Additional chapters highlight how to workup patients presenting with certain features including cerebral palsy/intellectual disability, congenital muscular dystrophy, cognitive decline/dementia, peripheral neuropathy, and paroxysmal disorder. The final section explores therapeutic strategies based on genetic interventions and genetic counselling options. Internationally contributed, this book will become the essential reference guide for neurologist. Reviews genetic testing for diagnostic confirmation, including carrier testing and prenatal diagnosis Explores various therapeutic strategies based on genetic interventions Discusses when a neurologic problem may have an underlying genetic cause
Book Synopsis Mechanism and Genetic Susceptibility of Neurological Disorders by : Andleeb Khan
Download or read book Mechanism and Genetic Susceptibility of Neurological Disorders written by Andleeb Khan and published by Springer Nature. This book was released on with total page 425 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Gene Therapy in the CNS – Progress and Prospects for Novel Therapies by : Casper René Gøtzsche
Download or read book Gene Therapy in the CNS – Progress and Prospects for Novel Therapies written by Casper René Gøtzsche and published by Frontiers Media SA. This book was released on 2021-12-21 with total page 188 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Neuroepigenomics in Aging and Disease by : Raul Delgado-Morales
Download or read book Neuroepigenomics in Aging and Disease written by Raul Delgado-Morales and published by Springer. This book was released on 2017-05-18 with total page 525 pages. Available in PDF, EPUB and Kindle. Book excerpt: Epigenetic mechanisms (DNA modifications, histone alterations and non-coding RNAs) are crucial for transcriptional regulation and alterations of the “physiological epigenome” are increasingly associated with human diseases. During the last decade the emerging field of neuroepigenomics have started to impact tremendously in areas such learning and memory, addiction or neurodegeneration. This expert volume covers the role of epigenetic molecular mechanism in regulation of central nervous system’s function, one of the most exciting areas of contemporary molecular neuroscience. The book describes the current knowledge on the epigenetic basis of human disease covering the complete lifespan: from neurodevelopment/childhood (Rett Syndrome, Rubinstein-Taybi, autism), adolescence (eating disorders, drug addiction, anxiety), adulthood (depression, schizophrenia, amyotrophic lateral sclerosis, Huntington’s disease) and elderly (Alzheimer’s disease, Parkinson’s disease). The book also covers the three major players on neuroepigenomic mechanisms: histones alterations, DNA modifications and non-coding RNAs, their roles at the molecular and cellular level and the impact of their alterations on neuronal function and behavior. Finally, a special chapter on state-of-the-art technologies helps the reader not only to understand epigenetic driven changes in human cognition and diseases but also the methodology that will help to generate paradigm shifts on our understanding of brain function and the role of the neuroepigenome in human diseases.
Book Synopsis Polyglutamine Disorders by : Clévio Nóbrega
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 469 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.
Book Synopsis Gene Therapy in Neurological Disorders by : Mingjie Li
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
