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Progress and prospects: Immunobiology of gene therapy for neurodegenerative disease: prospects and risks

Gene Therapy volume 17, pages 448–458 (2010)Cite this article

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A Corrigendum to this article was published on 13 May 2010

Abstract

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.

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Acknowledgements

We thank members of the Wood laboratory for critical input and in particular Yiqi Seow for help in the preparation of Figure 1. MJAW receives funding from the Medical Research Council UK and the Parkinson's Disease Society.

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    M M McMenamin & M J A Wood

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McMenamin, M., Wood, M. Progress and prospects: Immunobiology of gene therapy for neurodegenerative disease: prospects and risks. Gene Ther 17, 448–458 (2010). https://doi.org/10.1038/gt.2010.2

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