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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Autors principals:	Samantha Donsante, Alice Pievani, Biagio Palmisano, Melissa Finamore, Grazia Fazio, Alessandro Corsi, Andrea Biondi, Shunji Tomatsu, Rocco Piazza, Marta Serafini, Mara Riminucci
Format:	Artigo
Idioma:	Inglês
Publicat:	American Society for Clinical investigation 2024-03-01
Col·lecció:	JCI Insight
Matèries:	Bone biology Stem cells
Accés en línia:	https://doi.org/10.1172/jci.insight.173449
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