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...
A gift to the MacKenzie Lab will help us to develop better ways to treat genetic diseases before birth.
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
- Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.
2025 |
PubMed
- In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
2025 |PubMed
- Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases.
2025 |PubMed
- Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
2025 |PubMed
- In utero hematopoietic stem cell transplantation for Fanconi anemia.
2024 |PubMed