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In utero  hematopoietic cell transplantation ( IUHCT ) has been performed in Mucopolysaccharidosis Type  VII  ( MPSVII ) mice, but a lifelong engraftment of allogeneic donor cells has not been achieved. In this study, we sought to confirm a lifelong engraftment of allogeneic donor cells immunologically matched to the mother and to achieve partial rescue of phenotypes in the original  MPSVII  strain through  IUHCT  by intravenous injection. We performed  in vitro  fertilization in a  MPSVII  murine model and transferred affected embryos to  ICR /B6‐ GFP  surrogate mothers in cases where fetuses receiving  IUHCT  were all homozygous. Lineage‐depleted cells from  ICR / B6 ‐ GFP  mice were injected intravenously at  E 14.5. Chimerism was confirmed by flow cytometry at 4 weeks after birth, and β‐glucuronidase activity in serum and several phenotypes were assessed at 8 weeks of age or later. Donor cells in chimeric mice from  ICR / B6 ‐ GFP  mothers were detected at death, and were confirmed in several tissues including the brains of sacrificed chimeric mice. Although the serum enzyme activity of chimeric mice was extremely low, the engraftment rate of donor cells correlated with enzyme activity. Furthermore, improvement of bone structure and rescue of reproductive ability were confirmed in our limited preclinical study. We confirmed the lifelong engraftment of donor cells in an original immunocompetent  MPSVII  murine model using intravenous  IUHCT  with cells immunologically matched to the mother without myeloablation, and the improvement of several phenotypes.

Partial rescue of mucopolysaccharidosis type VII mice with a lifelong engraftment of allogeneic stem cells in utero