Testing a Novel Therapy to Treat NF1-Related Skeletal Defects
Abstract
The goal of this study was to test whether recombinant Osteolectin protein improved osteogenic differentiation of cells from patients with pseudarthrosis as well as cells from mouse models of Nf1-associated skeletal disease. Using single-cell techniques, we identified molecular dysregulation associated with somatic loss of NF1, which underlies fracture pseudathroses in these patients. We confirmed that osteogenic differentiation is blunted in pathologic cells from patient pseudarthroses; however, treatment of cells with Osteolectin did not improve differentiation. Recombinant Osteolectin also failed to improve osteogenic differentiation of cells harvested from mouse models of Nf1-associated skeletal disease. Taken together, our study suggests Osteolectin is not a potential therapy for treatment ofNF1-associated somatic skeletal disease, such as fracture pseudarthroses.
Document Details
- Document Type
- Technical Report
- Publication Date
- Jan 01, 2022
- Accession Number
- AD1191103
Entities
People
- Jonathan J. Rios
Organizations
- Scottish Rite for Children
- University of Texas at Dallas