Article
Generation of glycosylphosphatidylinositol anchor protein-deficient blood cells from human induced pluripotent stem cells.
Stem cells translational medicine - 1 Nov 2013
Yuan Xuan, Braunstein Evan M, Ye Zhaohui, Liu Cyndi F, Chen Guibin, Zou Jizhong, Cheng Linzhao, Brodsky Robert A
Abstract excerpt
PIG-A is an X-linked gene required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIG-A mutant cells have a deficiency or absence of all GPI-anchored proteins (GPI-APs). Acquired mutations in hematopoietic stem cells result in the disease paroxysmal nocturnal hemoglobinuria, and hypomorphic germline PIG-A mutations lead to severe developmental abnormalities, seizures, and early death....
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