Osteopetrotic induced pluripotent stem cells derived from patients with different disease-associated mutations by non-integrating reprogramming methods

Summary

Generation of iPSC using SeV and episomal DNA vectors have several advantages over other methods like the ease of production, reliability, high efficiency, and safety, which is required for translational research. Furthermore, owing to the pluripotency and self-renewal capacity, patient-specific iPSCs seem to be ideal cell source for the modeling of a rare genetic bone disease like osteopetrosis to identify osteoclast defects, leading to clinical heterogeneity in osteopetrosis patients, especially among those with different mutations in the same gene.

Authors Okur FV, Cevher İ, Özdemir C, Kocaefe Ç, Çetinkaya DU
Journal Stem cell research & therapy
Publication Date 2019 Jul 17;10(1):211
PubMed 31315669
PubMed Central PMC6637500
DOI 10.1186/s13287-019-1316-8

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