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Gene therapy for neurological, and in particular neurodegenerative, disease is now a reality. A number of early phase clinical trials have been completed and several are currently in progress. In view of this, it is critically important to evaluate the immunological risk associated with neurological gene therapy, which has clear implications for trial safety and efficacy. Moreover, it is imperative in particular to identify factors indicating potential high risk. In the light of recent advances in understanding immune regulation in the central nervous system (CNS) and with the continued development of new gene delivery vectors, this review critically assesses the current knowledge of immunobiology within the CNS in terms of likely immunological risk pertaining to viral vectors and gene therapy applications for neurodegenerative disease.

Original publication




Journal article


Gene Ther

Publication Date





448 - 458


Central Nervous System, Gene Silencing, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Immunity, Innate, Models, Biological, Neurodegenerative Diseases