In utero hematopoietic stem cell transplantation: A caprine model for prenatal therapy in inherited metabolic diseases

Objectives: We explored the feasibility and efficacy of in utero hematopoie tic stem cell transplantation in the caprine animal model system with the o bjectives of determining procedures for transplantation and establishing me thods for detecting engraftment. Methods: Male fetal liver hematopoietic st em cells were injected into female fetuses during the immunotolerant period , using either hysterotomy or ultrasound-guided injections. Results: The ra te of fetal death was much lower for the ultrasound-guided injections. Dono r cells were observed in the peritoneal fluid of 4 fetuses 3 days after inj ection, but no donor cells were detected in tissues at longer time periods. Conclusions: Ultrasound-guided injection of hematopoietic stem cells into the abdomen of a developing fetus is safe and feasible. The parameters requ ired for successful engraftment have not yet been identified. Copyright (C) 2001 S. Karger AG, Basel.