title: Stem cell transplantation before birth - a realistic option for treatment of osteogenesis imperfecta?
abstract: Osteogenesis imperfecta (OI) is characterized by severe bone deformities, growth retardation and bones that break easily, often from little or no apparent cause. OI is a genetic disorder primarily with defective type I collagen with a wide spectrum of clinical expression. In the more severe cases, it can be diagnosed before birth. Transplantation of mesenchymal stem cells (MSC) has the potential to improve the bone structure and stability, growth and fracture healing. Prenatal and postnatal cell transplantation has been investigated in preclinical and clinical studies of OI and suggests that this procedure is safe and has positive effects. Cell transplantation resulted in improved linear growth, mobility and reduced fracture incidence. However, the effect is transient and for this reason re-transplantation may be needed. So far there is limited experience in this area, and proper studies are required to accurately determine if MSC transplantation is of clinical benefit in the treatment of OI. In this review, we summarize what is currently known in this field.
authors.affiliation: Center for Fetal Medicine, Karolinska University Hospital, Stockholm, Sweden.|Division of Obstetrics and Gynaecology, Karolinska Institutet, Stockholm, Sweden.
authors.forename: Magnus
authors.initials: M
authors.lastname: Westgren
authors.affiliation: Division of Obstetrics and Gynaecology, Karolinska Institutet, Stockholm, Sweden.|Center for Haematology and Regenerative Medicine, Karolinska Institutet, Stockholm, Sweden.
authors.forename: Cecilia
authors.initials: C
authors.lastname: Götherström
mesh_terms: D005260:Female; D046128:Fetal Therapies / Q000941:ethics / Q000379:methods*; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation / Q000941:ethics / Q000379:methods*; D010013:Osteogenesis Imperfecta / Q000175:diagnosis / Q000628:therapy*; D011247:Pregnancy; D011296:Prenatal Diagnosis; D016896:Treatment Outcome