基因敲除
生物
信使核糖核酸
分子生物学
RNA剪接
BETA(编程语言)
β地中海贫血
珠蛋白
转基因
地中海贫血
癌症研究
基因
遗传学
核糖核酸
计算机科学
程序设计语言
作者
Shuyang Xie,Wei Li,Zhaorui Ren,Jingzhi Zhang,Xinbin Guo,Shu Wang,Shu‐Zhen Huang,Fanyi Zeng,Yi‐Tao Zeng
标识
DOI:10.1016/s1673-8527(08)60080-6
摘要
Large amounts of aberrantly spliced mRNA from the β654 allele was present in erythroid cells, which might impair the erythropoiesis. A therapeutic strategy for β-thalassemia was explored by knocking down the aberrantly spliced mRNA of β-globin. Lentiviral vector with siRNA fragment targets on the specific portion of β654-globin aberrantly spliced pre-mRNA was constructed. In HeLa β654 cells, the siRNA vector could reduce approximately 60% of aberrantly spliced mRNA, which was assessed by RT-PCR and qRT-PCR. Furthermore, a disease model of β654 thalassemia mice with lentiviral-mediated siRNA was produced by subzonal injection (named Hβi-Hbbth-4/Hbb+ transgenic mice). Our results showed that the hemotological parameters were improved in Hβi-Hbbth-4/Hbb+ transgenic mice. This study provides a potential way for β654-thalassemia therapy by knocking down the aberrantly spliced β-globin mRNA, whilst supporting that the aberrantly spliced β-globin mRNA may aggravate the disease.
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