In utero stem cell transplantation.

AIM

To investigate feasibility, safety, and efficacy of in utero transplantation of hemopoietic stem cells.

METHODS

A 10-week fetus was found to have b-thalassemia major after prenatal diagnosis by chorionic villus sampling and DNA analysis. The couple asked for prenatal treatment and, after extensive genetic counseling and local Ethical Committee approval, CD34+ hematopoietic progenitor cells purified from paternal bone marrow were injected to the fetus intraperitoneally, under ultrasound guidance.

RESULTS

A healthy 3.5 kg fetus was spontaneously delivered with no clinical or laboratory signs of graft-versus-host-disease. Analysis of the cord blood by high-pressure liquid chromatography revealed the absence of adult HbA, typical of b-thalassemic patients. Analysis of the Hb chain synthesis showed no clear signs of b-chain presence.

CONCLUSIONS

In utero transplantation of hematopoietic progenitor cells was not successful for b-thalassemia. Caution should be taken when considering other applications than immunodeficiency diseases.