Modeling skeletal dysplasia in Hurler syndrome using patient-derived bone marrow osteoprogenitor cells
Dysostosis multiplex is a major cause of morbidity in Hurler syndrome (mucopolysaccharidosis type IH [MPS IH], OMIM #607014) because currently available therapies have limited success in its prevention and reversion. Unfortunately, the elucidation of skeletal pathogenesis in MPS IH is limited by dif...
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| Hoofdauteurs: | , , , , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Society for Clinical investigation
2024-03-01
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| Reeks: | JCI Insight |
| Onderwerpen: | |
| Online toegang: | https://doi.org/10.1172/jci.insight.173449 |
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