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
Clinical Trials
- Related Conditions: Mucopolysaccharidosis, Mucopolysaccharidosis Type I, Hunter Syndrome, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type VII, Pompe Disease, Gaucher Disease, Wolman Disease| Start Date: | End Date:
- Related Conditions: Alpha Thalassemia, Alpha Thalassemia Major, Beta Thalassemia| Start Date: | End Date:
- Related Conditions: Mucopolysaccharidosis, Mucopolysaccharidosis Type I, Hunter Syndrome, Mucopolysaccharidosis Type Iva, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type VII, Gaucher Disease, Pompe Disease, Wolman Disease| Start Date: | End Date:
- Related Conditions: Alpha Thalassemia, Alpha Thalassemia Major, Hemoglobinopathy, Anemia, Hydrops Fetalis, Bone Marrow Transplant, Hematopoietic Cell Transplantation, Stem Cell Transplant, Beta Thalassemia| Start Date: | End Date:
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
- 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
- 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 - 2024-07-31 | 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
- Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases.| | PubMed
- Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.| | PubMed
- In utero hematopoietic stem cell transplantation for Fanconi anemia.| | PubMed
- The Conundrum of Mechanics Versus Genetics in Congenital Hydrocephalus and Its Implications for Fetal Therapy Approaches: A Scoping Review.| | PubMed
- Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells.| | PubMed
- Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus.| | PubMed
- Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.| | PubMed
- Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.| | PubMed
- Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies.| | PubMed
- Are we prepared to deliver gene-targeted therapies for rare diseases?| | PubMed