Biography
Videos
Education
| Institution | Degree | Dept or School | End Date |
|---|---|---|---|
| Children's Hospital of Philadelphia | Fellow | Pediatric Surgery | 2007 |
| Brigham and Women's Hospital | Chief Resident | Surgery | 2005 |
| Brigham and Women's Hospital | Residency | Surgery | 2004 |
| Children's Hospital of Philadelphia | Postdoctoral Studies | Fetal Therapy | 2002 |
| Stanford University | M.D. | Medicine | 1997 |
Board Certifications
- American Board of Surgery, 2006
Clinical Expertise
Advanced Laparoscopy
Birth Defects
Endocrine and Biliary Surgery
Fetal Surgery
Pediatric Surgery
Program Affiliations
- Fetal Treatment Center
- Biomedical Sciences Program
- Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research
- Center for Maternal-Fetal Precision Medicine
In the News
Grants and Funding
- Prenatal AAV9 gene replacement therapy for patients with severe infantile GM1 gangliosidosis | NIH | 2026-04-01 - 2029-03-31 | Role: Principal Investigator
- Phase 1 Study of In Utero Enzyme Replacement Therapy for the Treatment of Lysosomal Storage Diseases | NIH/NICHHD | 2022-09-20 - 2027-04-30 | Role: Principal Investigator
- Phase 1 Study of In Utero Enzyme Replacement Therapy for the Treatment of Lysosomal Storage Diseases | NIH | 2022-09-20 - 2027-04-30 | Role: Principal Investigator
- Developing gene therapy strategies to treat alpha thalassemia | NIH | 2022-01-01 - 2025-12-31 | Role: Principal Investigator
- Developing gene therapy strategies to treat alpha thalassemia | NIH/NHLBI | 2022-01-01 - 2025-12-31 | Role: Principal Investigator
- Autoimmune Regulator gene (Aire)-mediated tolerance to pregnancy-associated self-antigens | NIH | 2019-08-21 - 2025-07-31 | Role: Principal Investigator
- In utero hematopoietic stem cell transplantation for the treatment of fetuses with alpha thalassemia major (phase I clinical trial) | California Institute for Regenerative Medicine (CIRM) | 2017-08-01 - 2022-07-31 | Role: Principal Investigator
- A precision medicine approach to detect dysbiosis and immune activation in preterm labor | Burroughs Wellcome Fund Preterm Labor Initiative | 2017-07-01 - 2021-06-30 | Role: Principal Investigator
- T cell activation and the breakdown of maternal-fetal tolerance in preterm labor | NIH | 2015-06-15 - 2021-05-31 | Role: Principal Investigator
Research Narrative
Dr. MacKenzie has an active laboratory and is a member of the Biomedical Sciences Program and the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research. Her research focus is on mechanisms of tolerance induction following in utero stem cell transplantation and the pathophysiology of prenatally diagnosed diseases, such as congenital diaphragmatic hernia and gastroschisis, to identify biomarkers that predict prognosis and molecular pathways that may be targets for prenatal intervention.
Research Interests
In-Utero Stem Cell Transplantation
Research Pathways
Publications
- In Utero Hematopoietic Cell Transplantation in Fetuses with Alpha Thalassemia Major: A Phase 1 Clinical Trial.| |
PubMed
- Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.| |
PubMed
- Single-cell spatiotemporal dissection of the human maternal-fetal interface.| |
PubMed
- Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community.| |
PubMed
- Single nuclei and spatial profiling of sacrococcygeal teratomas reveals cellular composition and X inactivation heterogeneity.| |
PubMed
- Evidence for Maternal Autoantibodies in the Pathogenesis of Preterm Birth.| |
PubMed
- Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.| |
PubMed
- GFP-on mouse model for interrogation of in vivo gene editing.| |
PubMed
- Integrated single-nuclei and spatial transcriptomic profiling of human sacrococcygeal teratomas reveals heterogeneity in cellular composition and X-chromosome inactivation.| |
PubMed
- In utero genetic therapy: Treatment of early onset neurological disorders before they start.| |
PubMed