Establishment of a cell model of X-linked sideroblastic anemia using genome editing

•An in vitro cell model for X-linked sideroblastic anemia (XLSA) was established.•Ring sideroblasts were observed reproducibly in ALAS2 knockdown (HA2low) cells.•Erythroid differentiation induced ring sideroblast formation in HA2low cells.•Mitochondrial iron deposition in HA2low1 cells was confirmed...

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Bibliographic Details
Published in:Experimental hematology Vol. 65; pp. 57 - 68.e2
Main Authors: Kaneko, Kiriko, Kubota, Yoshiko, Nomura, Kazumi, Hayashimoto, Haruka, Chida, Taisei, Yoshino, Naoto, Wayama, Marina, Ogasawara, Katsutoshi, Nakamura, Yukio, Tooyama, Ikuo, Furuyama, Kazumichi
Format: Journal Article
Language:English
Published: Netherlands Elsevier Inc 01-09-2018
Online Access:Get full text
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