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

Publisher: National Academies Press

Published: 2019-10-31

Total Pages: 93

ISBN-13: 0309495873

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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.

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

Publisher: National Academies Press

Published: 2019-12-01

Total Pages: 93

ISBN-13: 0309495849

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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.

Handbook of Innovations in Central Nervous System Regenerative Medicine
Handbook of Innovations in Central Nervous System Regenerative Medicine

Author: António Salgado

Publisher: Elsevier

Published: 2020-06-10

Total Pages: 712

ISBN-13: 0128180854

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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

Improving and Accelerating Therapeutic Development for Nervous System Disorders
Improving and Accelerating Therapeutic Development for Nervous System Disorders

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2014-02-06

Total Pages: 107

ISBN-13: 0309292492

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Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

Central Nervous System Metastases
Central Nervous System Metastases

Author: Manmeet Ahluwalia

Publisher: Springer Nature

Published: 2019-11-05

Total Pages: 411

ISBN-13: 3030234177

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This book provides a comprehensive overview of brain metastases, from the molecular biology aspects to therapeutic management and perspectives. Due to the increasing incidence of these tumors and the urgent need to effectively control brain metastatic diseases in these patients, new therapeutic strategies have emerged in recent years. The volume discusses all these innovative approaches combined with new surgical techniques (fluorescence, functional mapping, integrated navigation), novel radiation therapy techniques (stereotactic radiosurgery) and new systemic treatment approaches such as targeted- and immunotherapy. These combination strategies represent a new therapeutic model in brain metastatic patients in which each medical practitioner (neurosurgeon, neurologist, medical oncologist, radiation oncologist) plays a pivotal role in defining the optimal treatment in a multidisciplinary approach. Written by recognized experts in the field, this book is a valuable tool for neurosurgeons, neuro-oncologists, neuroradiologists, medical oncologists, radiation oncologists, cognitive therapists, basic scientists and students working in the area of brain tumors.

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

Author: Michael G. Kaplitt

Publisher: Gulf Professional Publishing

Published: 2006

Total Pages: 380

ISBN-13: 9780123976321

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Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology 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 in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who 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 which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.

Histiocytic Disorders
Histiocytic Disorders

Author: Oussama Abla

Publisher: Springer

Published: 2017-11-17

Total Pages: 391

ISBN-13: 3319596322

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This volume provides a comprehensive and world-class review of the field of histiocytic neoplasms and hemophagocytic lymphohistiocytosis (HLH). It reviews all the advances in the field of histiocytoses during the last ten years, particularly with regards to the genomic findings in LCH and other histiocytic neoplasms and the new suggested classification of the histiocytic disorders. Additionally, it features a state-of-the art update on the most recent treatment strategies for LCH, including the results of the last LCH-III international trial, salvage therapies such as reduced-intensity conditioning (RIC) stem cell transplant (SCT), and targeted therapies with BRAF and MEK inhibitors, as well as the challenging cases of CNS-neurodegenerative LCH and its therapeutic perspectives. For primary and secondary HLH the book updates the most recent genetic and pathophysiological findings, including macrophage-activation syndrome (MAS), and includes a special chapter on HLH in adults. Treatment chapters encompass therapy for newly diagnosed HLH and refractory disease as well as stem-cell transplantation and novel therapies. The text also highlights the most recent advances in the treatment of the uncommon histiocytic disorders, such as Erdheim-Chester disease (ECD), Juvenile xanthogranuloma (JXG) and JXG-like conditions, Rosai-Dorfman disease (RDD), and the very rare malignant histiocytoses. Written by international experts in the field, Histiocytic Disorders is a valuable resource for clinicians, researchers, fellows and residents who are interested in or manage histiocytic disorders in children and adults.

Muscle Gene Therapy
Muscle Gene Therapy

Author: Dongsheng Duan

Publisher: Springer Science & Business Media

Published: 2009-11-26

Total Pages: 281

ISBN-13: 144191207X

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Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.

Genome Editing in Neurosciences
Genome Editing in Neurosciences

Author: Rudolf Jaenisch

Publisher:

Published: 2020-10-08

Total Pages: 128

ISBN-13: 9781013268564

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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.

Epigenetic Regulation in the Nervous System
Epigenetic Regulation in the Nervous System

Author: J. David Sweatt

Publisher: Academic Press

Published: 2012-12-31

Total Pages: 374

ISBN-13: 0123944058

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Epigenetic Regulation in the Nervous System addresses current understanding of the roles of epigenetic processes at the molecular/cellular level, their impact on neural development and behavior, and the potential roles of these mechanisms in neurological and psychiatric disorders. This award-winning volume spans molecular epigenetics, development, cellular physiology and biochemistry, synaptic and neural plasticity, and behavioral models, and is unique in covering epigenetically based disorders of the central nervous system.Behavioral epigenetics is the study of how environmental factors alter behavior, addressing the fundamental mechanisms that shape development and individual vulnerability/resilience to adverse behavioral outcomes. By understanding the molecular mechanisms involved in epigenetic modulation, researchers may be able to develop targeted therapies for those individuals in whom it malfunctions.Edited by the most highly regarded leaders in the field, this book offers a comprehensive review of behavioral epigenetics and a balanced treatment of the strengths and weaknesses in experimentation in this area. Covering background material as well as topics of current interest, it serves both as a cutting-edge resource and a foundational reference. The book will benefit neuroscience researchers and graduate students with an interest in the links between gene regulation and behavior, as will clinicians dealing with disorders such as addiction, depression, and schizophrenia. - BMA Medical Book Awards 2014 - Highly Commended, Neurology, British Medical Association - BMA Medical Book Awards 2014 - First Prize, Neurology, British Medical Association - 2013 PROSE Award winner for Best in Reference Works and Best Single Volume Reference in Science from the Association of American Publishers - Presents a unified view of epigenetic mechanisms from behavior to genes and everything in between - Discusses clinically relevant disorders in the context of epigenetics research, making the volume appealing to clinicians as well as basic scientists - Provides numerous practical examples for the new investigator to facilitate implementation of research in neuroepigenetics