rAAV-Mediated Gene Therapy Targeting CNS Disorders
Publication date
2025-01-01
Authors
Suarez-Amaran, Lester
Chatzi, Christina
Samulski, Richard Jude
Flier, Inge
Editors
Advisors
Supervisors
Document Type
Part of book
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taverne
Abstract
Central nervous system (CNS) disorders are the leading cause of disability and the second leading cause of death worldwide, with conditions such as Parkinson’s disease, Alzheimer’s disease, epilepsy, and amyotrophic lateral sclerosis (ALS) having profound impacts on patients’ quality of life. These disorders impair motor, cognitive, and sensory functions, imposing significant emotional, social, and financial burdens on patients and caregivers [36, 56, 57]. This chapter will discuss the present set of tools of gene therapy that can be applied to these disorders.
Keywords
Taverne, General Neuroscience, General Medicine
Citation
Suarez-Amaran, L, Chatzi, C, Samulski, R J & Flier, I 2025, rAAV-Mediated Gene Therapy Targeting CNS Disorders. in Translational Neuroscience : Fundamental Approaches for Neurological Disorders. Springer, pp. 9-30. https://doi.org/10.1007/978-3-031-89307-0_2