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Disease-Modifying Targets in Neurodegenerative Disorders - 1st Edition - ISBN: 9780128051207, 9780128052662

Disease-Modifying Targets in Neurodegenerative Disorders

1st Edition

Paving the Way for Disease-Modifying Therapies

Editors: Veerle Baekelandt Evy Lobbestael
Hardcover ISBN: 9780128051207
eBook ISBN: 9780128052662
Imprint: Academic Press
Published Date: 31st March 2017
Page Count: 320
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Disease-Modifying Targets in Neurodegenerative Disorders: Paving the Way for Disease-Modifying Therapies examines specific neurodegenerative disorders in comprehensive chapters written by experts in the respective fields. Each chapter contains a summary of the disease management field, subsequently elaborating on the molecular mechanisms and promising new targets for disease-modifying therapies.

This overview is ideal for neuroscientists, biomedical researchers, medical doctors, and caregivers, not only providing readers with a summary of the way patients are treated today, but also offering a glance at the future of neurodegenerative disorder treatment.

Key Features

  • Provides a comprehensive overview of how key proteins in neurodegenerative disorders can be used as targets to modify disease progress
  • Summarizes how patients are treated today, providing a glance at future disease management
  • Includes intelligible and informative information that is perfect for non-specialists, medical practitioners, and scientists
  • Written and peer reviewed by outstanding scientists in their respective fields


Neuroscientists, graduate and undergraduate students in biological and biomedical sciences, post-doctoral fellows, researchers, neurologists, clinicians

Table of Contents

Chapter 1: The multitude of therapeutic targets in neurodegenerative proteinopathies

  • Abstract
  • Protein Misfolding and Aggregation
  • Mechanism of Assembly
  • Prion-Like Propagation of Protein Assemblies
  • Propagation Routes of Pathogenic Protein Aggregates
  • Limiting Steps in the Propagation of Pathogenic Protein Assemblies
  • Therapeutic Strategies Targeting the Physiological Levels of Aggregation-Prone Proteins Involved in Neurodegeneration
  • Therapeutic Strategies Targeting the Misfolding and Aggregation of Proteins Involved in Neurodegeneration
  • Therapeutic Strategies Targeting the Accumulation of Misfolded Pathologic Protein Aggregates
  • Therapeutic Strategies Targeting the Cell-to-Cell Propagation of Pathogenic Protein Aggregates
  • Therapeutic Strategies Aimed at Restoring the Damage Pathogenic Protein Aggregates Induce
  • Therapeutic Strategies Targeting Misfolded Pathologic Protein Aggregate–Mediated Neuroinflammation
  • Limitations of the Different Therapeutic Strategies
  • Acknowledgments

Chapter 2: Synuclein misfolding as a therapeutic target

  • Abstract
  • Introduction
  • The Conformational Landscape of α-Synuclein: the Native State
  • The Conformational Landscape of α-Synuclein: Misfolded Variants
  • Misfolding of α-Synuclein as a Therapeutic Target
  • Conclusions

Chapter 3: Neuroinflammation as a therapeutic target in neurodegenerative diseases

  • Abstract
  • Introduction
  • Glial Cells in CNS Development, Homeostasis, and Pathology
  • Chronic Neuroinflammation as a Common Pathophysiological Mediator in Progressive Neurodegenerative Diseases
  • Therapeutic Strategies Targeting Neuroinflammation in Progressive Neurodegenerative Diseases
  • Future Perspectives
  • Conclusions

Chapter 4A: Stem cells in neurodegeneration: mind the gap

  • Abstract
  • Introduction
  • Part I: Stem Cells
  • Part II: Stem Cells as Regenerative Therapy
  • Part III: Stem Cells to Model Neurodegenerative Diseases

Chapter 4B: The potential of stem cells in tackling neurodegenerative diseases

  • Abstract
  • Endogenous Stem Cells as a Therapeutic Target

Chapter 5: Preclinical models of Alzheimer’s disease for identification and preclinical validation of therapeutic targets: from fine-tuning strategies for validated targets to new venues for therapy

  • Abstract
  • Introduction
  • Conclusions

Chapter 6: Parkinson’s disease

  • Abstract
  • Introduction
  • Current Treatment Approaches in Parkinson’s Disease
  • Drugs That are Being Evaluated Clinically for Disease Modification in PD
  • Novel Targets for Disease-Modifying Therapies in PD
  • Conclusions
  • Acknowledgments

Chapter 7: Lewy body dementia

  • Abstract
  • Lewy Body Dementia
  • Management of LBD
  • Concluding Remarks

Chapter 8: Frontotemporal dementia

  • Abstract
  • Overview of Frontotemporal Dementia
  • Disease Management
  • Novel Possibilities in Disease-Modifying Drug Development
  • Discussion: Essentials for the Development of a Disease-Modifying Therapy
  • Conclusions

Chapter 9: From huntingtin gene to Huntington’s disease-altering strategies

  • Abstract
  • Huntington’s Disease
  • Huntingtin Gene and Transcripts
  • Huntingtin Protein
  • HD Pathogenic Mechanisms
  • Molecular Strategies for HD
  • ASO
  • Genome Editing
  • Conclusions and Perspectives
  • Acknowledgments

Chapter 10: Amyotrophic lateral sclerosis: mechanisms and therapeutic strategies

  • Abstract
  • Introduction
  • Excitotoxicity
  • Hyperexcitability
  • Pathogenic Role of Non-Neuronal Cells
  • Shortage of Neurotrophic Factors
  • Mitochondrial Dysfunction
  • Axonal Defects
  • Altered Proteostasis and Autophagy
  • Altered RNA Metabolism and Stress Granule Formation
  • Hexanucleotide Repeats in C9ORF72 and Disturbances in Nucleocytoplasmic Transport
  • Conclusions
  • Acknowledgments


No. of pages:
© Academic Press 2017
31st March 2017
Academic Press
Hardcover ISBN:
eBook ISBN:

About the Editors

Veerle Baekelandt

Dr. Veerle Baekelandt obtained a Master of Romance Languages, Master of Biology, and a PhD in Biology at the Katholieke Universiteit (KU) Leuven. She received a Frank Boas Fulbright scholarship for graduate study at Harvard University and became a research fellow in the Laboratory for Neuroscience Research headed by Dr. Larry Benowitz, Children’s Hospital, Harvard Medical School, Boston (1992-1993). Her post-doctoral fellowship looked at gene therapy for neurodegenerative diseases, which became the cornerstone of her own research group.

She was appointed Assistant Professor at the KU Leuven in 2003 and in 2007, as full-time Research Professor (BOF-ZAP). She is now Head of the Laboratory for Neurobiology and Gene Therapy. Her research focuses on disease modeling and therapy for Parkinson’s disease using viral vectors in cell culture and in vivo. The underlying rationale is that the generation of more relevant models in cells and in pre-clinical model brain will lead to a better insight into the molecular pathogenesis of PD and to the development of new therapeutic strategies and drugs.

Affiliations and Expertise

Katholieke Universiteit (KU) Leuven, Belgium

Evy Lobbestael

Dr. Evy Lobbestael obtained her Master’s degree in biomedical sciences in the Faculty of Medicine at the KU Leuven. She completed her PhD training in the laboratory for Neurobiology and Gene Therapy at the KU Leuven under the supervision of Prof. Baekelandt, after obtaining a PhD scholarship from the Research Foundation Flanders. Her doctoral research focused on the function and dysfunction of the Parkinson’s disease-linked gene LRRK2. Besides the development of multiple tools to study LRRK2 including anti-LRRK2 antibodies, LRRK2-encoding lentiviral vectors, and cell lines, she identified protein phosphatase 1 as a physiological regulator of cellular LRRK2 phosphorylation. Currently, she continues her LRRK2 research as a post-doc with a focus on LRRK2 signaling.

Affiliations and Expertise

Katholieke Universiteit (KU) Leuven, Belgium

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