New hope for treating a rare genetic disease before birth

Summary: Stanford Medicine is starting a new trial for a rare genetic disease called Fanconi anemia. This disease stops a person's body from making healthy blood cells. Usually, kids with this disease need harsh treatments like chemotherapy and a bone marrow transplant after they are born. But doctors have a new idea: treat the baby before birth!

In this new trial, a pregnant mother donates her own healthy stem cells. Doctors give these cells to the baby through the umbilical cord. Because the baby's immune system is still developing, it accepts the mother's cells without needing strong drugs. This could fix the disease before the baby is even born, saving them from severe illness and long hospital stays later in life.

For more details, see rssapp-med-stanford-edu-news at med.stanford.edu/news/insights/2026/03/fanconi-anemia-prenatal-stem-cell-transplant-trial.html (opens in new tab)