In utero stem cell transplantation.
OBJECTIVE
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.
Monni
G
G
Department of Obstetrics and Gynecology, Prenatal and Preimplantation Diagnosis and Fetal Therapy, Ospedale Regionale per le Microcitemie, Via Jenner, Cagliari, 09121, Italy.
Ibba
R M
RM
Zoppi
M A
MA
Floris
M
M
D000328:Adult; D005240:Feasibility Studies; D005260:Female; D005315:Fetal Diseases / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D011247:Pregnancy; D017086:beta-Thalassemia / Q000628:therapy*