Researchers at Duke University are looking at the possibility of treating children. using cord blood, before they are born. The scientists are exploring the treatment of metabolic diseases including Krabbe disease, metachromatic leukodystrophy (MLD), Pelizaeus-Maerzbacher Disease (PMD), Tay-Sachs disease, or Sandoff disease.
The babies will be treated, while in utero, using stem cells from banked cord blood that have been treated to accelerate and and improve the process of engraftment. The transplant is relatively simple. The stem cells will be injected directly into the baby’s abdomen at 12-14 weeks of pregnancy.
The babies will be tested after birth to determine if donor cells are present and if they are working to repair the malfunctioning genes. If not, the baby may be eligible for a conventional transplant with chemotherapy during the first few weeks of life.
Read about what other diseases are currently treated by cord blood.
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