Generation of gene-corrected functional osteoclasts from osteopetrotic induced pluripotent stem cells

Abstract Background Infantile malignant osteopetrosis (IMO) is an autosomal recessive disorder characterized by non-functional osteoclasts and a fatal outcome early in childhood. About 50% of patients have mutations in the TCIRG1 gene. Methods IMO iPSCs were generated from a patient carrying a homoz...

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Bibliographic Details
Main Authors: Xiaojie Xian, Roksana Moraghebi, Henrik Löfvall, Anders Fasth, Kim Henriksen, Johan Richter, Niels-Bjarne Woods, Ilana Moscatelli
Format: Article
Language:English
Published: BMC 2020-05-01
Series:Stem Cell Research & Therapy
Subjects:
Online Access:http://link.springer.com/article/10.1186/s13287-020-01701-y