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

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 homozygous c...

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發表在:Stem Cell Res Ther
Main Authors: Xian, Xiaojie, Moraghebi, Roksana, Löfvall, Henrik, Fasth, Anders, Henriksen, Kim, Richter, Johan, Woods, Niels-Bjarne, Moscatelli, Ilana
格式: Artigo
語言:Inglês
出版: BioMed Central 2020
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Research
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7227215/
https://ncbi.nlm.nih.gov/pubmed/32414402
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13287-020-01701-y
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