Journal article
Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta
Nature medicine, v 5(3), pp 309-313
01 Mar 1999
PMID: 10086387
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
In principle, transplantation of mesenchymal progenitor cells would attenuate or possibly correct genetic disorders of bone, cartilage and muscle, but clinical support for this concept is lacking. Here we describe the initial results of allogeneic bone marrow transplantation in three children with osteogenesis imperfecta, a genetic disorder in which osteoblasts produce defective type I collagen, leading to osteopenia, multiple fractures, severe bony deformities and considerably shortened stature. Three months after osteoblast engraftment (1.5–2.0% donor cells), representative specimens of trabecular bone showed histologic changes indicative of new dense bone formation. All patients had increases in total body bone mineral content ranging from 21 to 29 grams (median, 28), compared with predicted values of 0 to 4 grams (median, 0) for healthy children with similar changes in weight. These improvements were associated with increases in growth velocity and reduced frequencies of bone fracture. Thus, allogeneic bone marrow transplantation can lead to engraftment of functional mesenchymal progenitor cells, indicating the feasibility of this strategy in the treatment of osteogenesis imperfecta and perhaps other mesenchymal stem cell disorders as well.
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Details
- Title
- Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta
- Creators
- Edwin M. Horwitz - St. Jude Children's Research HospitalDarwin J. Prockop - Allegheny CollegeLorraine A. Fitzpatrick - Mayo ClinicWinston W. K. Koo - Wayne State UniversityPatricia L. Gordon - St. Jude Children's Research HospitalMichael Neel - St. Jude Children's Research HospitalMichael Sussman - Shriners Hospitals for Children - EriePaul Orchard - University of MinnesotaJeffrey C. Marx - St. Jude Children's Research HospitalReed E. Pyeritz - Allegheny CollegeMalcolm K. Brenner - St. Jude Children's Research Hospital
- Publication Details
- Nature medicine, v 5(3), pp 309-313
- Publisher
- Nature Publishing
- Number of pages
- 5
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- College of Medicine
- Web of Science ID
- WOS:000078851600036
- Scopus ID
- 2-s2.0-0032976690
- Other Identifier
- 991022201457504721
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- Collaboration types
- Domestic collaboration
- Web of Science research areas
- Biochemistry & Molecular Biology
- Cell Biology
- Medicine, Research & Experimental