Tippi C. MacKenzie

Pediatric and fetal surgeon / Director of the Broad Stem Cell Center · University of California, San Francisco · company speakers →

Tippi MacKenzie, MD, is a pediatric and fetal surgeon at the University of California, San Francisco, and Director of the Broad Stem Cell Center. Her lab examines fetal biology and maternal-fetal tolerance with the goal of developing new fetal therapies for patients with genetic diseases or pregnancy complications. She has moved two fetal molecular therapies from the laboratory to the clinic as FDA-approved phase 1 clinical trials: in utero hematopoietic stem cell transplantation to treat fetuses with alpha thalassemia and in utero enzyme replacement therapy to treat fetuses with lysosomal storage disorders. Her ongoing research is focused on developing prenatal somatic cell gene therapies using antisense oligonucleotides, gene therapy and genome editing.

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