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Genome Editing For Neurodegenerative Diseases
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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 Academic Press. This book was released on 2024-09-01 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.
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 in Neurosciences by : Rudolf Jaenisch
Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by . This book was released on 2020-10-08 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.
Book Synopsis Genome Editing in Neurosciences by : Rudolf Jaenisch
Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by Springer. This book was released on 2017-09-14 with total page 129 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is open access under a CC BY 4.0 license. CRISPR-Cas9 is a rapid, efficient, versatile and relatively cheap method for dissecting the molecular pathways that are the basis of life, as well as for investigating and potentially rectifying faults in these pathways that result in disease. This book reviews how CRISPR-Cas9 and other genome editing techniques are advancing our understanding of development and function in the nervous system, uncovering the molecular causes of neurological disorders and providing tools for gene therapy.
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
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 561 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 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 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 Neurodegeneration: From Genetics to Molecules by : Victoria Campos-Peña
Download or read book Neurodegeneration: From Genetics to Molecules written by Victoria Campos-Peña and published by Frontiers Media SA. This book was released on 2016-11-15 with total page 266 pages. Available in PDF, EPUB and Kindle. Book excerpt: Chronic degenerative diseases are one of the major public health problems, particularly those affecting the nervous system. They are characterized by the degeneration of specific cell populations that include several pathologies which contribute significantly to morbidity and mortality in the elderly population. Therefore, in recent years, the study of neuroscience has gained significant importance. Most of these neurodegenerative disorders are the result of a complex interaction between genetic and environmental factors that generate progression and can even determine its severity. The presence of mutations in genes as LRRK2, SNCA, PARK7, PARK2 or PINK1 is associated with Parkinson's disease. Mutations in genes such as APP, PS1 and PS2 are associated with familial Alzheimer's disease; while HTT gene mutations are the cause of Huntington's disease. In most cases, this condition is inherited in an autosomal dominant pattern, which means one copy of the altered gene in each cell is sufficient to cause the disorder. It is known that these mutations can also alter the proteins function; however, it has not yet been possible to fully understand how some genetic changes cause the disease or influence the risk of developing these disorders. Most symptoms seen in these conditions occurs when specific nerve cells are damaged or die generating a loss in brain communication. Also many of these mutations generate aggregation of intracellular or extracellular proteins affecting cell function and eventually causing neuronal death. It is unclear whether the presence of these aggregates play an important role in nerve cell death during the development of neurodegenerative diseases, or if they are simply part of the response of cells to the disease. Other mutations affect the mitochondrial function generating alterations in energy production and promoting the formation of unstable molecules such as free radicals. Under normal conditions, the harmful effects caused by free radicals, are offset within the cell. However, in pathological conditions, the presence of mutations can alter this process by allowing the accumulation of radicals and damaging or killing cells. On the other hand, we also know that these diseases may not have a direct genetic component, thus, the study of sporadic type neurodegenerative diseases is much more complex. Histopathological lesions as well as the cellular and molecular alterations are generally indistinguishable from familial cases. For this reason, it is important to understand the genetic and molecular mechanisms associated with this type of pathologies. In this sense, this issue aims to understand the molecular processes that occur in the brain, and how these are influenced by the environment, genetics and behavior.
Book Synopsis Mutation-Specific Gene Editing for Blood Disorders by : Carsten Werner Lederer
Download or read book Mutation-Specific Gene Editing for Blood Disorders written by Carsten Werner Lederer and published by Frontiers Media SA. This book was released on 2021-11-25 with total page 218 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.
Book Synopsis Genetic Aberrancies and Neurodegenerative Disorders by : M.P. Mattson
Download or read book Genetic Aberrancies and Neurodegenerative Disorders written by M.P. Mattson and published by Elsevier. This book was released on 2000-01-28 with total page 443 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genetic Aberrancies and Neurodegenerative Disorders presents critical reviews and emerging findings concerning the roles of genetic mutations and polymorphisms in the pathogenesis of a range of neurological disorders including Alzheimer's and Parkinson's diseases, trinucleotide repeat disorders, stroke, epilepsy and others. This volume, written by leading experts, brings together fundamental information concerning the roles of inherited traits in the pathogenesis of different neurodegenerative disorders. In addition to providing a catalogue of the known genetic alterations that are linked to specific neurodegenerative disorders, the chapters detail the current state of understanding of the cellular and biochemical mechanisms whereby the genetic aberrancies lead to neuronal dysfunction and degeneration. The emerging picture of each disorder, painted by pathological, biochemical and molecular brushes, suggests that they share key mechanisms including increased levels of oxidative stress, perturbed ion homeostasis, mitochondrial dysfunction and apoptotic proteolytic cascades. The existence of specific molecular defects provides the opportunity to design experiments that can establish the precise pathogenic mechanism of a specific mutation or genetic risk factor. The value of this approach is exemplified by recent studies of how mutations in Cu/Zn-superoxide dismutase cause amyotrophic lateral sclerosis and how presenilin mutations result in early-onset Alzheimer's disease. A theme developed among the different chapters is that events that occur during aging predispose neurons to genetic aberrancies that promote degenerative cascades, and that specific genetic defects exert their influence on particular populations of neurons in a disorder-specific manner. The chapters in this volume will stimulate readers to generate new hypotheses concerning the pathogenic mechanisms of genetic aberrancies that can be experimentally tested.
Book Synopsis RNA Metabolism in Neurodegenerative Diseases by : Rita Sattler
Download or read book RNA Metabolism in Neurodegenerative Diseases written by Rita Sattler and published by Springer. This book was released on 2018-06-18 with total page 321 pages. Available in PDF, EPUB and Kindle. Book excerpt: It has become evident over the last years that abnormalities in RNA processing play a fundamental part in the pathogenesis of neurodegenerative diseases. Cellular viability depends on proper regulation of RNA metabolism and subsequent protein synthesis, which requires the interplay of many processes including transcription, pre--‐mRNA splicing, mRNA editing as well as mRNA stability, transport and translation. Dysfunction in any of these processes, often caused by mutations in the coding and non--‐ coding RNAs, can be very destructive to the cellular environment and consequently impair neural viability. The result of this RNA toxicity can lead to a toxic gain of function or a loss of function, depending on the nature of the mutation. For example, in repeat expansion disorders, such as the newly discovered hexanucleotide repeat expansion in theC9orf72 gene found in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a toxic gain of function leads to the formation of RNA foci and the sequestration of RNA binding proteins (RBPs). This in return leads to a loss of function of those RBPs, which is hypothesized to play a significant part in the disease progression of ALS and FTD. Other toxicities arising from repeat expansions are the formation of RNA foci, bi--‐directional transcription and production of repeat associated non--‐ATG (RAN) translation products. This book will touch upon most of these disease mechanisms triggered by aberrant RNA metabolism and will therefore provide a broad perspective of the role of RNA processing and its dysfunction in a variety of neurodegenerative disorders, including ALS, FTD, Alzheimer’s disease, Huntington’s disease, spinal muscular atrophy, myotonic dystrophy and ataxias. The proposed authors are leading scientists in the field and are expected to not only discuss their own work, but to be inclusive of historic as well as late breaking discoveries. The compiled chapters will therefore provide a unique collection of novel studies and hypotheses aimed to describe the consequences of altered RNA processing events and its newest molecular players and pathways.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309452880 Total Pages :329 pages Book Rating :4.3/5 (94 download)
Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Book Synopsis Leucine-Rich Repeat Kinase 2 (LRRK2) by : Hardy J. Rideout
Download or read book Leucine-Rich Repeat Kinase 2 (LRRK2) written by Hardy J. Rideout and published by Springer. This book was released on 2017-03-28 with total page 280 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Gene Therapy for Neurological Disorders by : Fredric P. Manfredsson
Download or read book Gene Therapy for Neurological Disorders written by Fredric P. Manfredsson and published by Humana. This book was released on 2015-11-26 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols and tips on troubleshooting and avoiding known pitfalls. Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.
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 467 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.
