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Article

Hematopoietic stem cell transplantation, a curative approach in infantile osteopetrosis.

Bone - 1 Feb 2023

Schulz Ansgar, Moshous Despina

Abstract excerpt

Most patients with osteopetrosis (OPT) can be causally and curatively treated with allogeneic hematopoietic stem cell transplantation (HSCT) because osteoclasts are derived from the HSC. However, HSCT is contraindicated in some forms of OPT, namely OPT with neurodegeneration (in all patients with OSTM1 and about half of patients with CLCN7 mutations) and OPT caused by an osteoblast defect (patients with RANKL...

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Identifiers

PMID: 36470372
DOI: 10.1016/j.bone.2022.116634

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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