Gene Therapy in Neurological Disorders

Gene Therapy in Neurological Disorders

1st Edition - May 25, 2018

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  • Editors: Mingjie Li, B. Snider
  • Hardcover ISBN: 9780128098134
  • eBook ISBN: 9780128098219

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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.

Key Features

  • 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


Researchers and clinical practitioners in neurology, translational neuroscience, and gene therapies

Table of Contents

  • Section I  Overview and Methods
    1. Gene therapy methods and their applications in neurological disorders
    2. Targeting transgene and RNA interference-based gene silencing sequences to astrocytes using viral vector-mediated approaches
    3. Gene therapy using genomic DNA: Advances and challenges
    4. Stem cell-based gene therapy in neurological disorders

    Section II  Approaches to specific disorders
    5. Targeting astrocytes with viral gene therapy for Alzheimer's disease
    6. Prophylactic and therapeutic applications of catalytic immunoglobulin gene delivery in a mouse model of Alzheimer's disease
    7. Lentiviral vector delivery of orexin gene to study potential role of orexin and sleep modulation in the pathogenesis of Alzheimer’s disease
    8. Pharmacologically controlled neurotrophic factor gene therapy for Parkinson´s Disease
    9. Gene therapy as a novel target for treatment of symptoms and medication complications in Parkinson’s disease
    10. Inhibition of aggregation of mutant huntingtin by nucleic acid aptamers in vitro and in a yeast model of Huntington’s disease
    11. Intraspinal delivery of recombinant AAV mediated Interleukin-10 modifies disease in ALS mice.
    12. Differentiation of human induced pluripotent stem cells to Purkinje neurons
    13. Expressing full-length dystrophin using adeno-associated virus
    14. Genome editing therapy for Duchenne muscular dystrophy
    15. AAV vector mediated gene delivery to neurons and glial cells in damaged spinal cord
    16. Transplantation of human induced pluripotent stem cell-derived neural precursor cells for treatment of spinal cord injury
    17. Viral transduction of primary Schwann cells using a Cre-lox system to regulate GDNF expression
    18. Application of Herpes simplex virus vectors in treatment of neuropathic pain
    19. Oncolytic virotherapy for gliomas: a preclinical and clinical summary

Product details

  • No. of pages: 450
  • Language: English
  • Copyright: © Academic Press 2018
  • Published: May 25, 2018
  • Imprint: Academic Press
  • Hardcover ISBN: 9780128098134
  • eBook ISBN: 9780128098219

About the Editors

Mingjie Li

Dr. Li is an Associate Professor in the Department of Neurology at Washington University School of Medicine in St. Louis, Missouri. He is also a Senior Scientist in the Viral Vectors Core at the Hope Center for Neurological Disorders. Dr. Li has over 15 years’ experience in viral vector development and gene therapy, and some of the vectors have been used in clinical trials. After joining Department of Neurology at Washington University, Dr. Li has focused on developing vectors for applications in neuroscience and constructed numerous new vectors targeting neurological disorders.

Affiliations and Expertise

Assistant Professor, Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA

B. Snider

Dr. Snider is a Professor in the Department of Neurology at Washington University School of Medicine. She is also the Director of Hope Center Viral vectors Core. Dr. Snider is interested in translational research on neurodegenerative disorders and undertakes clinical studies at the Washington University Alzheimer's Disease Research Center. She also sees patients at Barnes-Jewish Hospital/Washington University in St. Louis, Missouri

Affiliations and Expertise

Associate Professor, Director of Hope Center Viral Vectors Core, Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA

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