MacKenzie Lab - Homepage
Tippi MacKenzie, MD
Professor of Surgery
Benioff UCSF Professor of Children’s Health
Director, Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF
Co-Director, Center for Maternal-Fetal Precision Medicine
John G. Bowes Distinguished Professor of Stem Cell and Tissue Biology
MacKenzie Lab
Mission:
Understanding the fundamental mechanisms of maternal-fetal biology in order to develop innovative fetal therapies for genetic diseases and pregnancy complications.
To create a collaborative interdisciplinary research environment to train the next generation of scientists/physicians.
Our Vision:
To translate discoveries in biology and technology into prenatal therapies that will transform medical care for expectant families.
Our Values:
The MacKenzie Lab is a diverse group of scientists from various backgrounds and from all over the world. As such, we approach each other with cultural humility, and we believe...
Videos
- Treating Rare Genetic Diseases — In Utero
- UCSF Prenatal Enzyme Replacement for Lysosomal Storage Diseases—PEARL Trial
- Prenatal Enzyme Replacement for Lysosomal Storage Diseases (PEARL) Phase 1 Clinical Trial Presentation
- ATM — Part 1: Tippi and Elianna’s Story: Stopping a Fatal Blood Disease Before Birth
- ATM — Part 2: Tippi, Elliott and Elianna’s Story: Little Girl Thrives After Fetal Stem Cell Transplant
- MacKenzie Mentor Award Nomination
Featured Publications
- In Utero Hematopoietic Stem Cell Transplantation for Fanconi Anemia.
2024 | PubMed - Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells.
2024 | PubMed - Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus.
2024 | PubMed - Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
2024 | PubMed - Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.
2023 | PubMed