Generation of an immunodeficient mouse model of tcirg1-deficient autosomal recessive osteopetrosis.
Mouse model
Osteopetrosis
Transplantation
Journal
Bone reports
ISSN: 2352-1872
Titre abrégé: Bone Rep
Pays: United States
ID NLM: 101646176
Informations de publication
Date de publication:
Jun 2020
Jun 2020
Historique:
received:
30
09
2019
revised:
23
12
2019
accepted:
04
01
2020
entrez:
16
1
2020
pubmed:
16
1
2020
medline:
16
1
2020
Statut:
epublish
Résumé
Autosomal recessive osteopetrosis is a rare skeletal disorder with increased bone density due to a failure in osteoclast bone resorption. In most cases, the defect is cell-autonomous, and >50% of patients bear mutations in the We generated a new mouse model, which we named NSG oc/oc, presenting severe autosomal recessive osteopetrosis owing to the We demonstrated that neonatal murine bone marrow transplantation rescued NSG oc/oc mice, in line with previous findings in the oc/oc parental strain and with evidence from clinical practice in humans. Importantly, we also demonstrated human cell chimerism in the bone marrow of NSG oc/oc mice transplanted with human CD34 Our work paves the way to generating an improved xenograft model for
Sections du résumé
BACKGROUND
BACKGROUND
Autosomal recessive osteopetrosis is a rare skeletal disorder with increased bone density due to a failure in osteoclast bone resorption. In most cases, the defect is cell-autonomous, and >50% of patients bear mutations in the
METHODS
METHODS
We generated a new mouse model, which we named NSG oc/oc, presenting severe autosomal recessive osteopetrosis owing to the
RESULTS
RESULTS
We demonstrated that neonatal murine bone marrow transplantation rescued NSG oc/oc mice, in line with previous findings in the oc/oc parental strain and with evidence from clinical practice in humans. Importantly, we also demonstrated human cell chimerism in the bone marrow of NSG oc/oc mice transplanted with human CD34
CONCLUSION
CONCLUSIONS
Our work paves the way to generating an improved xenograft model for
Identifiants
pubmed: 31938717
doi: 10.1016/j.bonr.2020.100242
pii: S2352-1872(20)30001-2
pii: 100242
pmc: PMC6953598
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100242Subventions
Organisme : BLRD VA
ID : I01 BX002490
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR065407
Pays : United States
Informations de copyright
© 2020 Published by Elsevier Inc.
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