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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.

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rAAV-Mediated Gene Therapy Targeting CNS Disorders

  • 章节

  • 发表日期：

    2025年

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  • Lester Suarez-Amaran,
  • Christina Chatzi,
  • Inge Flier,
  • Richard Jude Samulski

摘要

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.