The Patients' Stories

Successful Case Stories, Published in Scientific Journals

Intrauterine stem cell transplantation in preparation for treatment of genetic diseases

January 24, 2023
Shimon Slavin

Many genetic diseases can be treated by stem cell transplantation from a normal donor. Many genetic disorders can be diagnosed during early pregnancy using a diagnostic amniocentesis or chorionic villus biopsy. Because of this, it was important to investigate if stem cell transplantation could be safely accomplished in utero.

To investigate if pre-natal allogeneic stem cell transplantation was feasible, it was important to investigate whether such treatment can be safely accomplished. The first successful attempt investigating the feasibility of intra-uterine allogeneic stem cell transplantation to treat genetic disease was accomplished during pregnancy. This was done on a mother with a suspected beta thalassemic embryo.

This case documents the first technically successful attempt to treat genetic disease by infusing the donor’s normal hematopoietic stem cells into the baby’s cord, while still connected to the placenta. For this, we used an ultrasound-guided needle. It can also be accomplished through a direct intra-peritoneal injection of normal donor’s stem cells.

The picture shows Professor Slavin after the delivery of the baby. He is pointing to the spot where the needle (carrying the donor’s stem cells) was inserted. The procedure was uneventful for both the mother and the newborn baby.

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