News & Events: Alpha Thalassemia Major

UCSF News reports on the first patient enrolled in the world's first clinical trial using blood stem cells transplanted prior to birth. The stem cell transplant was used to treat alpha thalassemia major, a blood disorder that is only detected in the last few months of pregnancy and has been almost uniformly fatal in the past. The clinical trial is led by Tippi MacKenzie, MD, an associate professor in the UCSF Division of Pediatric Surgery and co-Director of the UCSF Center for Maternal-Fetal Precision Medicine. The study is funded by a $12.1M grant from the California Institute for [...]

Pediatric Surgeon Tippi MacKenzie, MD was awarded $12.1 million by the California Institute for Regenerative Medicine (CIRM) to treat fetuses in the womb suffering from alpha thalassemia major, a blood disorder that is only detected in the last few months of pregnancy and is almost uniformly fatal.  Dr. MacKenzie, an associate professor in the UCSF Division of Pediatric Surgery and co-Director of the UCSF Center for Maternal-Fetal Precision Medicine, is using hematopoietic stem cells (HSCs), taken from the mother's bone marrow, and transplanting them into the fetus before birth. The fetus's [...]