CIRM Awards Tippi MacKenzie, MD $12.1M to Treat Fetuses in the Womb Alpha Thalassemia Major
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 immune system is able to tolerate the mother’s cells, increasing the chances of a healthy birth, and improving the chances of having effective treatments after birth.
Alpha thalassemia is a recessively inherited hemoglobinopathy (genetic defect in the hemoglobin) caused by mutations in the alpha globin gene. It is one of the most common single-gene disorders worldwide, affecting approximately 5% of the population with prevalence the highest in China, South East Asia, the Middle East, and India.
What is alpha thalassemia? Fetal Treatment Center Website