Gene Therapy in Neurological Disorders - 1st Edition - ISBN: 9780128098134

Gene Therapy in Neurological Disorders

1st Edition

Editors: Mingjie Li B. Snider
Hardcover ISBN: 9780128098134
Imprint: Academic Press
Published Date: 1st May 2018
Page Count: 400
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Gene Therapy in Neurological Disorders covers viral and non-viral gene deliveries and tools, including emerging technologies, such as CRISPR/Cas9 gene editing. These tools can be applied to a range of disorders, including neurodegeneration, neuroregeneration, muscular dystrophy, behavior disorders and retinal diseases. Through the use of successful examples, scientists, physicians and students will better understand the principles of gene therapy and learn how to use these tools in their research. The book begins with a detailed introduction on gene therapy, including its history, lessons learned, improvements, current status, prospects and applications in neurological disorders.

Key Features

  • Covers experimental details of gene therapy tools for neuroscience research and neurological disorders
  • Written for researchers and clinical practitioners
  • Focuses on translational neuroscience, neurology and gene therapies
  • Discusses varying applications of gene therapy tools, ranging from neurodegeneration, neuroregeneration, muscular dystrophy, behavior disorders and for use in retinal diseases


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

Table of Contents

  1. Opposing effects of viral mediated brain expression of apolipoprotein E2 (apoE2) and apoE4 on apoE lipidation and AB metabolism in apoE4-targeted replacement mice
    2. Potential role of orexin and sleep modulation in the pathogenesis of Alzheimer’s disease
    3. Genetic Correction of Tauopathy Phenotypes in Neurons Derived from Human Induced Pluripotent Stem Cells
    4. Enhancing Astrocytic Lysosome Biogenesis Facilitates a Clearance and Attenuates Amyloid Plaque Pathogenesis
    5. Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    6. Fiber-Modified Adenovirus for Central Nervous System Parkinson’s Disease Gene Therapy
    7. Pharmacologically controlled, discontinuous GDNF gene therapy restores motor function in a rat model of Parkinson's disease
    8. Regulator of G-Protein Signaling-10 Negatively Regulates NF-B in Microglia and Neuroprotects Dopaminergic Neurons in Hemiparkinsonian Rats
    9. Striatal transplantation of human dopaminergic neurons differentiated from induced pluripotent stem cells derived from umbilical cord blood using lentiviral reprogramming
    10. In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense
    11. Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice
    12. Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
    13. Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors
    14. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy
    15. Gene therapy for lysosomal storage diseases
    16. Prohibitin Viral Gene Transfer Protects Hippocampal CA1 Neurons from Ischemia and Ameliorates Postischemic Hippocampal Dysfunction
    17. Adenosine kinase, glutamine synthetase and EAAT2 as gene therapy targets for temporal lobe epilepsy
    18. Dual Transgene Expression in Murine Cerebellar Purkinje Neurons by Viral Transduction In Vivo
    19. Viral Transduction of Primary Schwann Cells Using a Cre-Lox System to Regulate GDNF
    20. Transduction efficiency of neurons and glial cells by AAV-1, -5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury


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© Academic Press 2018
Academic Press
Hardcover ISBN:

About the Editor

Mingjie Li

Dr. Li is an Assistant 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 an Associate 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