IAP–Based Gene Therapy for Neurodegenerative Disorders
Stephen J. Crocker, Daigen Xu, Charlie S.Thompson, Peter Liston, and George S. Robertson
Neurodegenerative diseases are steadily becoming more prominent as human life expectancy increases. Gene therapy holds tremendous promise for the long term treatment of neurodegenerative disorders. The potential successes of gene therapy as a clinically useful therapeutic will be determined by two key components: the vector(s) used and the payloads they carry. The major constraints at the present time for the use of adenoviral delivery systems include the duration of vector-derived gene expression, targeting of vectors to specific cell or tissue types, and toxicity. At the same time, however, the number of candidate genes for potential delivery is immense and growing daily. Ultimately, the clinical potential provided by a candidate gene for the treatment of a neurodegenerative disorder requires a detailed understanding of the molecular pathway(s) in which the virally-delivered protein product will function.