Gene Therapy In Neurological Disorders

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Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders
  • Author : Mingjie Li,B. Joy Snider
  • Publisher : Academic Press
  • Release Date : 2018-05-25
  • Total pages : 450
  • ISBN : 9780128098219
  • File Size : 18,9 Mb
  • Total Download : 989
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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

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Pedro R. Lowenstein,Maria G. Castro
  • Publisher : CRC Press
  • Release Date : 2006-05-01
  • Total pages : 384
  • ISBN : 0824728475
  • File Size : 33,5 Mb
  • Total Download : 910
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Coedited by internationally recognized leaders in gene therapy research, this guide supplies the most recent advances, studies, and expert opinion on gene therapy for neurological disorders. Spanning conditions such as Parkinsons's and Alzheimer's disease, multiple sclerosis, brain tumors, stroke, epilepsy, and brain and spinal cord trauma, this reference presents the basic science and applications of recombinant gene products, immune and stem cell-mediated therapeutic strategies, and brain tumor gene therapy.

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Frederic P Manfredsson
  • Publisher : Humana Press
  • Release Date : 2015-11-26
  • Total pages : 493
  • ISBN : 1493932705
  • File Size : 31,8 Mb
  • Total Download : 306
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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.

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

Gene Therapy of the Central Nervous System: From Bench to Bedside
  • Author : Matthew During
  • Publisher : Gulf Professional Publishing
  • Release Date : 2006
  • Total pages : 380
  • ISBN : 0123976324
  • File Size : 21,7 Mb
  • Total Download : 392
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Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far 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 of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and 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 of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Advancing Gene-Targeted Therapies for Central Nervous System Disorders
  • Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
  • Publisher : National Academies Press
  • Release Date : 2019-10-31
  • Total pages : 92
  • ISBN : 9780309495875
  • File Size : 26,6 Mb
  • Total Download : 971
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On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Gene Therapy

Gene Therapy
  • Author : David Cooper,Prof Nick Lemoine
  • Publisher : Garland Science
  • Release Date : 2020-09-10
  • Total pages : 431
  • ISBN : 9781000144680
  • File Size : 24,8 Mb
  • Total Download : 801
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Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date.

Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders
  • Author : Mingjie Li,B. Joy Snider
  • Publisher : Academic Press
  • Release Date : 2018-05
  • Total pages : 400
  • ISBN : 0128098139
  • File Size : 24,6 Mb
  • Total Download : 573
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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

Gene Therapy for Neurological Disorders and Brain Tumors

Gene Therapy for Neurological Disorders and Brain Tumors
  • Author : E. Antonio Chiocca,Xandra O. Breakefield
  • Publisher : Humana Press
  • Release Date : 2013-10-03
  • Total pages : 458
  • ISBN : 1475753144
  • File Size : 47,5 Mb
  • Total Download : 371
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Leading gene therapy researchers and clinicians illuminate the field-from basic vector technology to current and future clinical applications in neurology. The authoritative contributors provide cutting-edge reviews of the vectors available for gene transfer to the central nervous system, the strategies against CNS tumors, the potential strategies against neurologic disorder, and the limitations of today's gene therapy approaches. Also discussed are significant applications of gene therapy to brain tumors, Parkinson's disease, ischemia, and Huntington's chorea. Readers will learn the current delivery methods for transgenes, will learn the characteristics of transgene delivery vectors, and come to understand the therapy for both neuro-oncologic and neurologic disorders.

Advances in Cell and Gene Therapy in Treating Neural Diseases

Advances in Cell and Gene Therapy in Treating Neural Diseases
  • Author : Raymond Ching-Bong Wong,Kouichi Hasegawa,Guei-Sheung Liu,Gary S. L. Peh
  • Publisher : Frontiers Media SA
  • Release Date : 2022-01-07
  • Total pages : 252
  • ISBN : 9782889719952
  • File Size : 33,5 Mb
  • Total Download : 123
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PDF book entitled Advances in Cell and Gene Therapy in Treating Neural Diseases written by Raymond Ching-Bong Wong,Kouichi Hasegawa,Guei-Sheung Liu,Gary S. L. Peh and published by Frontiers Media SA which was released on 2022-01-07 with total hardcover pages 252, the book become popular and critical acclaim.

Current Concepts in Movement Disorder Management

Current Concepts in Movement Disorder Management
  • Author : A. Niranjan,L.D. Lunsford,R.M. Richardson
  • Publisher : Karger Medical and Scientific Publishers
  • Release Date : 2018-01-12
  • Total pages : 272
  • ISBN : 9783318062021
  • File Size : 17,6 Mb
  • Total Download : 420
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This book summarizes the current state of movement disorder management and the role of surgical therapies as an alternative to medication. Following a chapter on the history of movement disorder surgery, leaders in their fields describe the pathophysiology, functional neuroanatomy, clinical presentation, and medical management of Parkinson’s disease, dystonia, and essential tremor. This is followed by chapters on the spectrum of movement disorder surgery itself, from the lesioning procedures of radiofrequency ablation, stereotactic radiosurgery, and high-frequency ultrasound to the modulatory procedures of “asleep”, image-guided deep brain stimulation (DBS) and “awake”, microelectrode-guided DBS. The final chapters focus on closed-loop DBS, drug-delivery, gene therapy, and other emerging neurosurgical therapies, highlighting long-standing experimental strategies that are reaching exciting phases of clinical translation. This volume is a valuable tool for accessing the wide spectrum of concepts that currently define this dynamic field.

Gene Therapy Methods

Gene Therapy Methods
  • Author : Anonim
  • Publisher : Elsevier
  • Release Date : 2002-03-06
  • Total pages : 728
  • ISBN : 9780080496948
  • File Size : 44,5 Mb
  • Total Download : 282
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This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Pedro R. Lowenstein,Maria G. Castro
  • Publisher : CRC Press
  • Release Date : 2006-05-01
  • Total pages : 384
  • ISBN : 0824728475
  • File Size : 20,9 Mb
  • Total Download : 350
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Download Gene Therapy for Neurological Disorders in PDF, Epub, and Kindle

Coedited by internationally recognized leaders in gene therapy research, this guide supplies the most recent advances, studies, and expert opinion on gene therapy for neurological disorders. Spanning conditions such as Parkinsons's and Alzheimer's disease, multiple sclerosis, brain tumors, stroke, epilepsy, and brain and spinal cord trauma, this reference presents the basic science and applications of recombinant gene products, immune and stem cell-mediated therapeutic strategies, and brain tumor gene therapy.

Gene Delivery and Therapy for Neurological Disorders

Gene Delivery and Therapy for Neurological Disorders
  • Author : Xuenong Bo,Joost Verhaagen
  • Publisher : Humana Press
  • Release Date : 2015-02-04
  • Total pages : 359
  • ISBN : 1493923056
  • File Size : 49,5 Mb
  • Total Download : 906
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This volume aims to explore the latest developments in adeno-associated viral and lentiviral vectors as well as the gene therapy strategies for the most common neurological disorders, followed by chapters that include step-by-step guides to viral vector-based gene delivery in animal models used in the authors’ laboratories. Although safe gene manipulation in neural cells can be achieved, it may still be years away from efficacious gene-based treatment of neurological disorders such as Parkinson's and Alzheimer’s diseases due to the complexity of the underlying genetic/molecular mechanisms and the difficulty of developing reliable animal models. Gene Delivery and Therapy for Neurological Disorders seeks to aid researchers in this vital work. Written in the popular Neuromethods series format, chapters include the kind of detailed description and expert implementation advice that leads to success in the lab. Meticulous and authoritative, Gene Delivery and Therapy for Neurological Disorders serves as an ideal guide for researchers attempting to explore the potentials of gene therapy for neurological disorders.

Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders
  • Author : Fredric P. Manfredsson
  • Publisher : Humana Press
  • Release Date : 2016-08-23
  • Total pages : 493
  • ISBN : 1493948962
  • File Size : 33,7 Mb
  • Total Download : 359
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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.

Genetics of Common Diseases

Genetics of Common Diseases
  • Author : Ian Day,Prof Steve Humphries
  • Publisher : Garland Science
  • Release Date : 2020-12-17
  • Total pages : 344
  • ISBN : 9781000102277
  • File Size : 12,5 Mb
  • Total Download : 710
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Many common diseases are partly attributable to the genes which an individual inherits. Early steps have now been made in developing ways to determine which genetic variations are important, with some recent successes. This is a collection of papers from the Fifth Annual Molecular Pathology Symposium on the 10 December 1996. They represent the contributions to that meeting made by a set of distinguished scientists and clinicians whose work pertains to the furtherment of our understanding of the genetic components of common diseases and potential future approaches.

Disease and Mortality in Sub-Saharan Africa

Disease and Mortality in Sub-Saharan Africa
  • Author : Dean T. Jamison
  • Publisher : World Bank Publications
  • Release Date : 2006-01-01
  • Total pages : 414
  • ISBN : 9780821363980
  • File Size : 9,5 Mb
  • Total Download : 432
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Current data and trends in morbidity and mortality for the sub-Saharan Region as presented in this new edition reflect the heavy toll that HIV/AIDS has had on health indicators, leading to either a stalling or reversal of the gains made, not just for communicable disorders, but for cancers, as well as mental and neurological disorders.

Oligonucleotide-Based Drugs and Therapeutics

Oligonucleotide-Based Drugs and Therapeutics
  • Author : Nicolay Ferrari,Rosanne Seguin
  • Publisher : John Wiley & Sons
  • Release Date : 2018-06-06
  • Total pages : 576
  • ISBN : 9781119070306
  • File Size : 20,8 Mb
  • Total Download : 542
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A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.

Textbook of Gene Therapy

Textbook of Gene Therapy
  • Author : Kewal K. Jain
  • Publisher : Seattle ; Toronto : Hogrefe & Huber
  • Release Date : 1998
  • Total pages : 434
  • ISBN : UOM:39015045638882
  • File Size : 35,6 Mb
  • Total Download : 794
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During the past eight years following initial gene therapy experiments, more than 200 clinical protocols have been submitted worldwide and more than 2000 patients have been treated. Although a lot remains to be accomplished before gene therapy becomes a standard medical practice, enough information has accumulated to justify a textbook on the subject. Some of the procedures and products are expected to enter the marketplace and medical practice by the year 2000. The public is very much aware of the potential of gene therapy and the medical profession should prepare itself for the new developments by learning about the basics and applications of these techniques. Much of there-search has been conducted in the bio-pharmaceutical industry by gene therapy companies and the commercial opportunities for gene therapy are enormous. A large segment of the society thus has an interest in gene therapy. This book presents a unique critical review of the tremendous progress which has been made in this field. The textbook is an effort to bring a wide range of important developments together in an accessible format. This book is aimed at physicians, gene therapists, molecular biologists, nurse practitioners and students in these fields, as well as other healthcare professionals interested in developments in the field of gene therapy and its impact, both short- and long-term, on the practice of medicine. Industrial executives planning long-term strategies in gene therapy will find this handy textbook to be a comprehensive source of information on the subject and the companies involved in developing it. Prof. K. K. Jain is a neurosurgeon trained in Canada and the United States. He has heldacademic positions in major medical centers around the world. For the last decade, he has been a consultant to a number of major biopharmaceutical companies and now runs his own biotechnology company specializing in gene therapy research and documentation.

Handbook of Innovations in Central Nervous System Regenerative Medicine

Handbook of Innovations in Central Nervous System Regenerative Medicine
  • Author : Antonio Salgado
  • Publisher : Elsevier
  • Release Date : 2020-06-10
  • Total pages : 710
  • ISBN : 9780128180853
  • File Size : 28,8 Mb
  • Total Download : 350
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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

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies

Gene Therapy in the CNS – Progress and Prospects for Novel Therapies
  • Author : Casper René Gøtzsche,David Woldbye,Merab Kokaia,Andreas Toft Sørensen,Marco Ledri
  • Publisher : Frontiers Media SA
  • Release Date : 2021-12-21
  • Total pages : 188
  • ISBN : 9782889718528
  • File Size : 24,8 Mb
  • Total Download : 173
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PDF book entitled Gene Therapy in the CNS – Progress and Prospects for Novel Therapies written by Casper René Gøtzsche,David Woldbye,Merab Kokaia,Andreas Toft Sørensen,Marco Ledri and published by Frontiers Media SA which was released on 2021-12-21 with total hardcover pages 188, the book become popular and critical acclaim.

Genome Editing in Neurosciences

Genome Editing in Neurosciences
  • Author : Rudolf Jaenisch,Feng Zhang,Fred Gage
  • Publisher : Unknown
  • Release Date : 2020-10-08
  • Total pages : 128
  • ISBN : 1013268563
  • File Size : 26,9 Mb
  • Total Download : 698
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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.