Rescue of ATPa3-deficient Murine Malignant Osteopetrosis by Hematopoietic Stem Cell Transplantation In Utero. (Articolo in rivista)

Type
Label
  • Rescue of ATPa3-deficient Murine Malignant Osteopetrosis by Hematopoietic Stem Cell Transplantation In Utero. (Articolo in rivista) (literal)
Anno
  • 2005-01-01T00:00:00+01:00 (literal)
Alternative label
  • Frattini A, Blair HC, Sacco MG, Cerisoli F, Faggioli F, Mira Catò E, Pangrazio A, Musio A, Rucci F, Sobacchi C, Sharrow AC, Kalla SE, Buzzone MG, Colombo R, Magli MC, Vezzoni P, Villa A. (2005)
    Rescue of ATPa3-deficient Murine Malignant Osteopetrosis by Hematopoietic Stem Cell Transplantation In Utero.
    in Proceedings of the National Academy of Sciences of the United States of America
    (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori
  • Frattini A, Blair HC, Sacco MG, Cerisoli F, Faggioli F, Mira Catò E, Pangrazio A, Musio A, Rucci F, Sobacchi C, Sharrow AC, Kalla SE, Buzzone MG, Colombo R, Magli MC, Vezzoni P, Villa A. (literal)
Pagina inizio
  • 14629 (literal)
Pagina fine
  • 14634 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume
  • 102 (literal)
Rivista
Note
  • ISI Web of Science (WOS) (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni
  • Istituto di Tecnologie Biomediche, CNR (literal)
Titolo
  • Rescue of ATPa3-deficient Murine Malignant Osteopetrosis by Hematopoietic Stem Cell Transplantation In Utero. (literal)
Abstract
  • Autosomal recessive osteopetrosis (ARO) is a paradigm for genetic diseases that cause severe, often irreversible, defects before birth. In ARO, osteoclasts cannot remove mineralized cartilage, bone marrow is severely reduced, and bone cannot be remodeled for growth. More than 50% of the patients show defects in the osteoclastic vacuolar-proton-pump subunit, ATP6a3. We treated ATP6a3-deficient mice by in utero heterologous hematopoietic stem cell (HSC) transplant from outbred GFP transgenic mice. Dramatic phenotype rescue by GFP osteoclasts was obtained with engraftment, which was observed in most cases. Engraftment survived for variable periods. Recipients were not immunosuppressed, and graft-versus-host disease was not observed in all pups born after in utero treatment. Thus, differentiation of unmatched HSC transplanted in utero is sufficient to prevent fatal defects in ARO and may prevent complications of ARO unresponsive to conventional bone marrow transplantation. The presence of defective cells is not a barrier to the rescue of the phenotype by donor HSC. (literal)
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