In utero transplantation of adult bone marrow decreases perinatal lethality and rescues the bone phenotype in the knockin murine model for classical, dominant osteogenesis imperfecta.

doi:10.1182/blood-2008-12-195859

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Article

In utero transplantation of adult bone marrow decreases perinatal lethality and rescues the bone phenotype in the knockin murine model for classical, dominant osteogenesis imperfecta.

Blood - 9 Jul 2009

Panaroni Cristina, Gioia Roberta, Lupi Anna, Besio Roberta, Goldstein Steven A, Kreider Jaclynn, Leikin Sergey, Vera Juan Carlos, Mertz Edward L, Perilli Egon, Baruffaldi Fabio, Villa Isabella, Farina Aurora, Casasco Marco, Cetta Giuseppe, Rossi Antonio, Frattini Annalisa, Marini Joan C, Vezzoni Paolo, Forlino Antonella

Abstract excerpt

Autosomal dominant osteogenesis imperfecta (OI) caused by glycine substitutions in type I collagen is a paradigmatic disorder for stem cell therapy. Bone marrow transplantation in OI children has produced a low engraftment rate, but surprisingly encouraging symptomatic improvements. In utero transplantation (IUT) may hold even more promise. However, systematic studies of both methods have so far been limited to a...

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Identifiers

PMID: 19414862
DOI: 10.1182/blood-2008-12-195859
PMCID: PMC2714216

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