In Utero Transplant in First Clinical Trial Successful

Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.

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(From left) Nichelle Obar, Elianna Constantino, and Tippi MacKenzie, the pediatric surgeon who performed the transplantNOAH BERGER/UCSF

Pediatric surgeons at the University of California, San Francisco, have treated a second-trimester fetus with stem cells taken from her mother’s bone marrow. The baby, born in February, was the first patient enrolled in the world’s first clinical trial using stem cells transplanted prior to birth. She is “apparently healthy,” despite living with a deadly genetic disease called alpha thalassemia, according to a statement from the university.

Tippi MacKenzie, a pediatric surgeon at the University of California, San Francisco (UCSF) Benioff Children’s Hospital, led the team that performed the transplant. “It is too early to say how effective the stem cell transplantation will be, but we are encouraged by how well she and her mother have tolerated the treatment,” she says in the statement. “Her ...

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