摘要:
The present invention relates to a method for preparing a GM1 gangliosidosis human cell model based on induced pluripotent stem cells (iPSCs) and iPSCs originated neural progenitor cells, and a use of the GM1 model above for the development of a GM1 gangliosidosis treating agent. The iPSCs originated from GM1 patient fibroblasts can be differentiated into neural progenitor cells (NPCs) and neurosphere cells that can emulate the characteristics shown in GM1 patient, so that the said cells can be efficiently used for the investigation of intracellular GM1 symptoms such as the GM1 gangliosidosis and lysosome accumulation and the gene expression pattern change. So, the GM1 cell model of the present invention can be efficiently used for the study of GM1 development mechanism and the study for the development of a therapeutic agent for the disease. The present inventors also established the inflammasome inhibitor rhIL1RA or Z-YVAD-FMK by using the above GM1 cell model and further confirmed that it can be efficiently used as a relieving/treating agent of GM1 gangliosidosis. In addition, the molecular symptoms of GM1 patient could be reproduced in the transformed cells having the E186A mutation which is newly identified as the GM1 gangliosidosis causing protein mutation. Therefore, the mutant cells containing the induced E186A mutation can be efficiently used as the GM1 gangliosidosis cell model.
摘要:
The present invention relates to a composition comprising a repressor of mitofusin gene expression, an inhibitor of mitofusin protein activity, or a mixture thereof as an active ingredient for promoting reprogramming a differentiated cell into a pluripotent stem cell, and a use thereof. The composition according to the present invention increases the efficiency of reprogramming as well as reduces the time required for reprogramming to produce pluripotent stem cells. Therefore, the present composition can be beneficially used to develop the production technology of high efficiency pluripotent stem cell and secure a large-scale culture system. Further, the present composition can be beneficially used to maintain pluripotent stem cells and screen the compounds capable of promoting the reprogramming into pluripotent stem cells.
摘要:
A method comprising transforming Schizosaccharomyces pombe with a deletion cassette, constructed by four-round serial PCR, block PCR or total gene synthesis, containing a homologous recombination site is provided for preparing gene-targeted heterozygous deletion Schizosaccharomyces pombe. Also provided are gene-targeted hetero2ygous deletion Schizosaccharomyces pombe mutants prepared by the method, and a library of gene-targeted heterozygous deletion Schizosaccharomyces pombe mutants. Further, the library is useful in constructing a method and a kit for screening a drug's modes of action.
摘要:
The present invention relates to a composition comprising an extract of Tiarella polyphylla, and tiarellic acid isolated therefrom having anti-inflammatory, anti-allergic and anti-asthmatic activity. The extract of Tiarella polyphylla and the tiarellic acid isolated therefrom shows the inhibitory effect on the LTC 4 release in vitro test and the suppressive effect on the IgE level and the cytokine(IL-4, IL-5 and IL- 13) production, airway hyperresponsiveness, and leukocyte infiltration in OVA-induced asthmatic mice. Therefore, it can be used as the therapeutics or functional health food for treating and preventing inflammatory, allergic and asthmatic disease.