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报告题目:
Using pluripotent stem cell to study and treat aging-associated disorders
 报告人:
Guanghui Liu
Professor of National Laboratory of Biomacromolecules, 
Institute of Biophysics, Chinese Academy of Sciences
报告时间:
2014-09-02 10:30
报告地点:
Room B323, Medical School Building
主办单位:
生命科学学院
  简介:
Global population aging has been causing increasing crisis around the world. Hutchinson–Gilford progeria syndrome (HGPS) and Werner syndrome (WS) are two human premature aging disorders with features that closely recapitulate the features of human ageing. Mutations in LMNA and WRN genes lead to aberrant splicing product progerin and protein loss in HGPS and WS, respectively. Study on how genetic and environmental alterations lead to the cellular and organismal phenotypes of premature aging will provide clues to the molecular mechanisms that underlie physiological ageing and increase our understanding of molecular pathways contributing to healthy aging. We have generated induced pluripotent stem cells (iPSCs) from fibroblasts obtained from patients with HGPS. Further, using targeted gene correction technology, we corrected the mutated LMNA gene in HGPS-iPSCs. Additionally, by using targeted gene “knock-in” technique, we created WS-specific human embryonic stem cells (hESCs) with WRN mutation, Parkinson’s disease (PD)-specific hESCs with LRRK2 mutation, and Fanconi Anemia-specific hESCs with FANCA mutation. Upon differentiation of these "diseased" human pluripotent stem cells into different somatic cell types, they demonstrated tissue-specific and aging-associated phenotypic defects. Together, these tools offer an unprecedented platform to study the pathogenesis of human aging-related diseases.
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