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Neural Regen Res. 2013 Nov 25;8(33):3095-106. doi: 10.3969/j.issn.1673-5374.2013.33.003.

Targeting β-secretase with RNAi in neural stem cells for Alzheimer's disease therapy.

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Department of Neurobiology and Anatomy, Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510080, Guangdong Province, China.


There are several major pathological changes in Alzheimer's disease, including apoptosis of cholinergic neurons, overactivity or overexpression of β-site amyloid precursor protein cleaving enzyme 1 (BACE1) and inflammation. In this study, we synthesized a 19-nt oligonucleotide targeting BACE1, the key enzyme in amyloid beta protein (Aβ) production, and introduced it into the pSilenCircle vector to construct a short hairpin (shRNA) expression plasmid against the BACE1 gene. We transfected this vector into C17.2 neural stem cells and primary neural stem cells, resulting in downregulation of the BACE1 gene, which in turn induced a considerable reduction in reducing Aβ protein production. We anticipate that this technique combining cell transplantation and gene therapy will open up novel therapeutic avenues for Alzheimer's disease, particularly because it can be used to simultaneously target several pathogenetic changes in the disease.


Alzheimer's disease; C17.2 neural stem cells; genetic modification; grants-supported paper; neural regeneration; neural stem cells; neurodegenerative disease; neuroregeneration; plasmid; primary neural stem cells; shRNA; β-secretase

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