Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients.
Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients.
复制标题
从杜氏肌营养不良症患者中产生两种诱导多能干细胞系。
DOI：
10.1016/j.scr.2023.103207
复制
发表时间：
2023
影响因子：
1.2
通讯作者：
Wu,JosephC
中科院分区：
文献类型：
--
作者：
Liu,Wenqiang;Zeng,Wenshu;Kong,Xiaohui;Htet,Min;Yu,Rebecca;Wheeler,Matthew;Day,JohnW;Wu,JosephC
Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder that leads to death in early adulthood. Patients with DMD have null mutations leading to loss of functional dystrophin protein. Here we generated two DMD induced pluripotent stem cell (iPSC) lines, one with deletion of exon 51 and the other with a single nucleotide nonsense mutation (c.10171C > T). Both lines expressed high levels of pluripotency markers, had the capability of differentiating into derivatives of the three germ layers, and possessed normal karyotypes. These iPSC lines can serve as powerful tools to model DMD in vitro and as a platform for therapeutic development.
