Gene Delivery into the Central Nervous System (CNS) Using AAV Vectors
( Gene Therapy - Principles and Challenges )
Publication series ： Gene Therapy - Principles and Challenges
Author： Koichi Miyake Noriko Miyake and Takashi Shimada
Publisher： IntechOpen
Publication year： 2015
E-ISBN: INT5982461638
P-ISBN(Paperback): 9789535122210
P-ISBN(Hardback): 9789535122210
Subject： R394 medical genetics
Keyword： 医学遗传学
Language： ENG
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Gene Delivery into the Central Nervous System (CNS) Using AAV Vectors
Description
Application of gene therapies is a promising approach to the treatment of various neurological 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 applications of AAV vectors to the treatment of CNS disorders.
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