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

Artikel i vetenskaplig tidskrift
Författare X. J. Xian
R. Moraghebi
H. Lofvall
Anders Fasth
K. Henriksen
J. Richter
N. B. Woods
I. Moscatelli
Publicerad i Stem Cell Research & Therapy
Volym 11
Nummer/häfte 1
Sidor 9
ISSN 1757-6512
Publiceringsår 2020
Publicerad vid Institutionen för kliniska vetenskaper, Avdelningen för pediatrik
Sidor 9
Språk en
Länkar dx.doi.org/10.1186/s13287-020-01701...
Ämnesord Infantile malignant osteopetrosis, iPSC generation, Gene transfer, Osteoclast differentiation, bone-marrow-transplantation, management, diagnosis, therapy, Cell Biology, Research & Experimental Medicine
Ämneskategorier Cellbiologi

Sammanfattning

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.11279G>A (IVS18+1) mutation in TCIRG1 and transduced with a lentiviral vector expressing human TCIRG1. Embryoid bodies were generated and differentiated into monocytes. Non-adherent cells were harvested and further differentiated into osteoclasts on bovine bone slices. Results Release of the bone resorption biomarker CTX-I into the media of gene-corrected osteoclasts was 5-fold higher than that of the uncorrected osteoclasts and 35% of that of control osteoclasts. Bone resorption potential was confirmed by the presence of pits on the bones cultured with gene-corrected osteoclasts, absent in the uncorrected IMO osteoclasts. Conclusions The disease phenotype was partially corrected in vitro, providing a valuable resource for therapy development for this form of severe osteopetrosis.

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