Successful experimental gene therapy for Alzheimer’s disease using a novel viral vector system that drives a brain-specific gene expression by intravenous administration

The research group of Professor Nobuhisa Iwata (Nagasaki University School of Pharmaceutical Sciences/JST CREST) and others, in cooperation with the research group of Associate Professor Haruhisa Inoue, Professor Shinya Nakayama (Kyoto University CiRA/JST Yamanaka iPS Cell Project) and others, has clarified the pathological condition present in each of several patients with Alzheimer’s disease (AD), and has shown a way to pre-emptive treatment using disease-specific iPS cells.
The research groups established iPS cells from patients of both early-onset and late-onset Alzheimer’s diseases, respectively, and successfully modeled the pathological conditions using neuronal cells differentiated from the iPS cells. They found that in both types there is a subtype in which a protein called amyloid-beta peptide (Aβ) is accumulated in the neuronal cells. Further, they clarified that Aβ accumulated in the cells becomes an aggregate (Aβ oligomer), causing intracellular stresses and increasing cell vulnerability, and this condition could be alleviated by administration of a specific inhibitor for Aβ production (β-secretase inhibitor, BSI) or docosahexaenoic acid (DHA).
In addition to clarification of pathogenic mechanism of the disease using iPS cell technology and its application to drug discovery research, these research results show a way to predicting the pathological condition in advance and providing a appropriate pre-emptive treatment.

In this joint research, Nagasaki University contributed the experimental design, analysis of Aβ metabolism of neural cells differentiated from iPS cells, analysis and evaluation of data, and composition and writing of the paper as specialists in Alzheimer’s disease research.

This research has been published in the online version of the American scientific journal Cell Stem Cell on February 21, 2013 (Eastern Standard Time).

Joint press release with Kyoto University” (PDF: 1MB)

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