Genome Editing in Neurosciences (Research and Perspectives in Neurosciences)

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Author : Rudolf Jaenisch
Genre : Medical

Summary : 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.

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Author : Rudolf Jaenisch
Genre : Medical

Summary : 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.

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Author : Simon G. Sprecher
Genre : Science

Summary : This book provides a thorough introduction to widely used techniques for the study of the intersection between developmental biology and neuroscience, an exceptional area to address and investigate impacting biological questions. The fully updated volume examines cutting-edge techniques on a representative range of animals, including widely used genetic model systems, such as the fruit fly, zebra fish, chicken, and mouse, as well as non-canonical experimental systems opened up through the advent of genome editing. Written for the highly successful Methods in Molecular Biology series, 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 up-to-date, Brain Development: Methods and Protocols, Second Edition is an ideal guide for researchers interested in utilizing recent technical advances in molecular genetics for the study of the brain.

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Author : Jacques Mallet
Genre : Medical

Summary : In this new volume in the series Research and Perspectives in Neurosciences the authors have presented and discussed their findings in the fields of speech and language disorders, X-linked mental retardation, gene therapy in the CNS, memory and learning disorders and other fields.

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Author : Malcolm R. Dando
Genre : Medical

Summary : This book discusses recent brain research and the potentially dangerous dual-use applications of the findings of these research projects. The book is divided into three sections: Part I examines the rise in dual-use concerns within various state’s chemical and biological non-proliferation regime’s during this century, as well as the rapid technologically driven advances in neuroscience and the associated possible misuse considerations in the same period. Part II reviews the brain research projects in the EU, USA, Japan, China and several other countries with regard to their objectives, achievements and measures to deal with the problem of dual-use. Part III assesses the extent to which the results of this civil neuroscience work, which is intended to be benign, are being, and could be protected against future hostile applications in the development of novel chemical and biological weapons.

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Author : Fred H. Gage
Genre : Medical

Summary : The recent advances in Programming Somatic Cell (PSC) including induced Pluripotent Stem Cells (iPS) and Induced Neuronal phenotypes (iN), has changed our experimental landscape and opened new possibilities. The advances in PSC have provided an important tool for the study of human neuronal function as well as neurodegenerative and neurodevelopmental diseases in live human neurons in a controlled environment. For example, reprogramming cells from patients with neurological diseases allows the study of molecular pathways particular to specific subtypes of neurons such as dopaminergic neurons in Parkinson’s Disease, Motor neurons for Amyolateral Sclerosis or myelin for Multiple Sclerosis. Detecting disease-specific molecular signatures in live human brain cells, opens possibilities for early intervention therapies and new diagnostic tools. Importantly, once the neurological neural phenotype is detected in vitro, the so-called “disease-in-a-dish” approach allows for the screening of drugs that can ameliorate the disease-specific phenotype. New therapeutic drugs could either act on generalized pathways in all patients or be patient-specific and used in a personalized medicine approach. However, there are a number of pressing issues that need to be addressed and resolved before PSC technology can be extensively used for clinically relevant modeling of neurological diseases. Among these issues are the variability in PSC generation methods, variability between individuals, epigenetic/genetic instability and the ability to obtain disease-relevant subtypes of neurons . Current protocols for differentiating PSC into specific subtypes of neurons are under development, but more and better protocols are needed. Understanding the molecular pathways involved in human neural differentiation will facilitate the development of methods and tools to enrich and monitor the generation of specific subtypes of neurons that would be more relevant in modeling different neurological diseases.

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Author : National Academies of Sciences, Engineering, and Medicine
Genre : Medical

Summary : To examine the promise, concerns, and challenges related to neuroscience research using genetically modified nonhuman primates, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop on October 4, 2018, bringing together an international group of experts and stakeholders representing academia, industry, laboratory animal management, disease-focused foundations, and federal agencies. The workshop was designed to explore the current state and future promise of research using genetically modified nonhuman primate models of disease to understand the complex functions of the brain that control behavior, movement, and cognition in both health and disease states. This publication summarizes the presentations and discussions from the workshop.