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...
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2020-05-01
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Series: | Stem Cell Research & Therapy |
Subjects: | |
Online Access: | http://link.springer.com/article/10.1186/s13287-020-01701-y |