Article
CRISPR/Cas9-Mediated Gene Correction in Osteopetrosis Patient-Derived iPSCs.
Frontiers in bioscience (Landmark edition) - 30 Jun 2023
Li Dandan, Ou Minglin, Zhang Wei, Luo Qi, Cai Wanxia, Mo Chune, Liang Wenken, Dai Guandong, Yin Lianghong, Zhu Peng, Tang Donge, Dai Yong
Abstract excerpt
BACKGROUND: Osteopetrosis represents a rare genetic disease with a wide range of clinical and genetic heterogeneity, which results from osteoclast failure. Although up to 10 genes have been identified to be related with osteopetrosis, the pathogenesis of osteopetrosis remains foggy. Disease-specific induced pluripotent stem cells (iPSCs) and gene-corrected disease specific iPSCs provide a platform to generate...
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