利用CRISPR/Cas9构建LRP2敲除人胚胎干细胞系及在眼疾病研究中的应用
Generation of a homozygous LRP2 knockout human embryonic stem cell line (FDCHDPe010-A-56) by CRISPR/Cas9 system
Jie You, Yun Cheng, Xian-Jie Yang, Ling Chen
Journal：Stem Cell Research
IF：2.02
DOI：10.1016/j.scr.2021.102342
PMID：33878707
Published：2021-04-08
research field：分子生物学再生医学微生物学
Abstract
LRP2 is mainly expressed in the cell membrane of epithelia, maintaining normal endocytosis of nutrients from the extracellular microenvironment and mediating growth factor signals. The deficiency of LRP2 can result in abnormal lysosomal and mitochondrial function as well as insufficient resistance to oxidative stress. LRP2-KO animals show enlarged eyes and malfunction of the retinal pigment epithelium (RPE). We were able to generate an LRP2-KO human embryonic stem (ES) cell line using CRISPR/Cas9 gene editing and differentiate the mutant ES cells into RPE cells. Thus, this LRP2-KO human ES line will facilitate studying cellular mechanisms of eye disease due to LRP2 deficiency.
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