Improved Helper-Dependent Adenoviral Vectors for High-Level, Neuron-Restricted Gene Transfer into the CNS

Adenoviral vectors (Ad) possess a number of attributes which favour their use for CNS gene delivery, e.g., their ability to infect post-mitotic tissues. However, the widespread distribution of the cellular receptor for Ad, the Coxsackie/adenovirus receptor (CAR), allows adenoviral vectors to infect a broad range of cells in the host, thus limiting cell-type specificity for gene transfer in the CNS. Also, the safety issue of these vectors is still a concern. Thus, there is a rationale for the development of Ad vectors capable of enhanced gene transfer specificity and efficacy, yet without evoking an immune respon…