Katsumi Higaki

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Human artificial chromosome (HAC) has several advantages as a gene therapy vector, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including the regulatory elements. Induced pluripotent stem (iPS) cells have great potential for gene therapy, as such cells can be generated from the(More)
Lipid movement between organelles is a critical component of eukaryotic membrane homeostasis. Niemann Pick type C (NP-C) disease is a fatal neurodegenerative disorder typified by lysosomal accumulation of cholesterol and sphingolipids. Expression of yeast NP-C-related gene 1 (NCR1), the orthologue of the human NP-C gene 1 (NPC1) defective in the disease, in(More)
OBJECTIVE Gaucher disease (GD) is a lysosomal storage disease characterized by a deficiency of glucocerebrosidase. Although enzyme-replacement and substrate-reduction therapies are available, their efficacies in treating the neurological manifestations of GD are negligible. Pharmacological chaperone therapy is hypothesized to offer a new strategy for(More)
BACKGROUND Fucoidan is a high-molecular polysaccharide whose main constituent is sulfated fucose. We specifically focused on the anti-proliferation activity of fucoidan and examined the underlying mechanism in MKN45 gastric cancer cells. METHODS MKN45 cell proliferation was analyzed by BrdU assay and fucoidan cytotoxicity was examined by LDH and(More)
Most Pharmacological chaperones (PC's) described until now are substrate analogues which bind to the active site of the target protein. Consequently , such PC's also inhibit the target protein at higher concentrations thus rendering a narrow therapeutic window and have poor drug-like properties. Through our proprietary technology platform SEE-Tx™, we(More)
G M1-gangliosidosis i s a n a utosomal r ecessive l ysosomal s torage disease, caused by deficiency of lysosomal β-galactosidase (β-gal), encoded by the GLB1 gene. To date, more than 160 human GLB1 gene mutations has been identified i n patients w ith t his disease, an d ab out 7 0% o f t hem ar e m issense mutations. C hemical (or p harmacological) ch(More)
Valienamine (1) i s the unsaturated derivative of carbocyclic analogues of glycosylamines. I ts α-and β-anomeric i somers ar e n ot i nterconvertible each o ther, existing as chemically stable forms (Fig. 1). Compound 1 was first isolated from antibiotic v alidamycins a nd f ound t o pos sess a s trong i nhibitory a ctivity a gainst α-glucosidase. It was(More)
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