**Gene Delivery into the Central Nervous System (CNS) Using AAV Vectors**

Koichi Miyake, Noriko Miyake and Takashi Shimada

Additional information is available at the end of the chapter

http://dx.doi.org/10.5772/61638

#### **Abstract**

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Application of gene therapies is a promising approach to the treatment of various neuro‐ logical disorders, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lysosomal storage disorders, which are not treatable by any other means. However, the blood–brain barrier (BBB) is a key obstacle to gene delivery to the central nervous system (CNS). Adeno-associated virus (AAV) vectors have emerged as a promising tool for gene delivery to the CNS, thanks to their safety and ability to transduce non-dividing neuronal cells. In this chapter, we discuss strategies for delivering genes across the BBB, focusing especially on potential routes of administration of AAV vectors and promising applica‐ tions of AAV vectors to the treatment of CNS disorders.

**Keywords:** Adeno-associated virus vector, central nervous system, routes of administra‐ tion, lysosomal storage disorders
