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: 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:
- Related Conditions: Alpha Thalassemia, Alpha Thalassemia Major, 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
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 a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.| | View in PubMed
- Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.| | View in PubMed
- Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies.| | View in PubMed
- Are we prepared to deliver gene-targeted therapies for rare diseases?| | View in PubMed
- The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.| | View in PubMed
- In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.| | View in PubMed
- Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.| | View in PubMed
- Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).| | View in PubMed
- Thymic and extrathymic Aire-expressing cells in maternal-fetal tolerance.| | View in PubMed
- Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.| | View in PubMed
- Consensus statement for the perinatal management of patients with α thalassemia major.| | View in PubMed
- Advances in the management of α-thalassemia major: reasons to be optimistic.| | View in PubMed
- Prenatal Gene Therapy.| | View in PubMed
- Maternal and Infant Immune Repertoire Sequencing Analysis Identifies Distinct Ig and TCR Development in Term and Preterm Infants.| | View in PubMed
- Exome sequencing of fetuses with congenital diaphragmatic hernia supports a causal role for NR2F2, PTPN11, and WT1 variants.| | View in PubMed
- Extrathymic Aire-expressing cells support maternal-fetal tolerance.| | View in PubMed
- Forever Connected: The Lifelong Biological Consequences of Fetomaternal and Maternofetal Microchimerism.| | View in PubMed
- Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement.| | View in PubMed
- Exome Sequencing for Prenatal Diagnosis in Nonimmune Hydrops Fetalis.| | View in PubMed
- Detection of microbial cell-free DNA in maternal and umbilical cord plasma in patients with chorioamnionitis using next generation sequencing.| | View in PubMed
- Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.| | View in PubMed
- CD161 contributes to prenatal immune suppression of IFNγ-producing PLZF+ T cells.| | View in PubMed
- In Utero Gene Therapy Consensus Statement from the IFeTIS.| | View in PubMed
- Evaluation of Clinical Outcomes of Sutureless vs Sutured Closure Techniques in Gastroschisis Repair.| | View in PubMed
- Depletion of murine fetal hematopoietic stem cells with c-Kit receptor and CD47 blockade improves neonatal engraftment.| | View in PubMed
- Large Differences in Small RNA Composition Between Human Biofluids.| | View in PubMed
- Future AAVenues for In Utero Gene Therapy.| | View in PubMed
- Meta-Analysis of Maternal and Fetal Transcriptomic Data Elucidates the Role of Adaptive and Innate Immunity in Preterm Birth.| | View in PubMed
- Alloreactive fetal T cells promote uterine contractility in preterm labor via IFN-γ and TNF-α.| | View in PubMed
- Systemic multilineage engraftment in mice after in utero transplantation with human hematopoietic stem cells.| | View in PubMed
- Esophagoesophagopexy technique for assisted fistulization of esophageal atresia.| | View in PubMed
- Non-Immune Hydrops Fetalis: Do Placentomegaly and Polyhydramnios Matter?| | View in PubMed
- Sacrococcygeal teratoma: late recurrence warrants long-term surveillance.| | View in PubMed
- Fetal stem cell and gene therapy.| | View in PubMed
- Epidemiology of Live Born Infants with Nonimmune Hydrops Fetalis-Insights from a Population-Based Dataset.| | View in PubMed
- Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature.| | View in PubMed
- A Transient Developmental Hematopoietic Stem Cell Gives Rise to Innate-like B and T Cells.| | View in PubMed
- Magnetic resonance imaging of Müllerian duct anomalies in children.| | View in PubMed
- Heightened Immune Activation in Fetuses with Gastroschisis May Be Blocked by Targeting IL-5.| | View in PubMed
- Mass Effect Alone May Not Explain Pulmonary Vascular Pathology in Severe Congenital Diaphragmatic Hernia.| | View in PubMed
- Fetal Surgical conditions and the unraveling of maternal-fetal tolerance.| | View in PubMed
- A booster shot to cure hemoglobinopathies.| | View in PubMed
- Maternal factors associated with the occurrence of gastroschisis.| | View in PubMed
- Increased maternal T cell microchimerism in the allogeneic fetus during LPS-induced preterm labor in mice.| | View in PubMed
- Consensus statement from the first international conference for in utero stem cell transplantation and gene therapy.| | View in PubMed
- In utero hematopoietic cell transplantation for hemoglobinopathies.| | View in PubMed
- Placental drug delivery for treatment of congenital hematopoietic disorders.| | View in PubMed
- The many faces of hydrops.| | View in PubMed
- Novel non-surgical prenatal approaches to treating congenital diaphragmatic hernia.| | View in PubMed
- Favorable outcomes in high-risk congenital pulmonary airway malformations treated with multiple courses of maternal betamethasone.| | View in PubMed
- Maternal-fetal cellular trafficking: clinical implications and consequences.| | View in PubMed
- In utero depletion of fetal hematopoietic stem cells improves engraftment after neonatal transplantation in mice.| | View in PubMed
- Memory regulatory T cells reside in human skin.| | View in PubMed
- Outcomes of fetal intervention for primary hydrothorax.| | View in PubMed
- Fetal intervention increases maternal T cell awareness of the foreign conceptus and can lead to immune-mediated fetal demise.| | View in PubMed
- Anal duplication in a one-year-old girl.| | View in PubMed
- Fetal production of growth factors and inflammatory mediators predicts pulmonary hypertension in congenital diaphragmatic hernia.| | View in PubMed
- Direct and indirect antigen presentation lead to deletion of donor-specific T cells after in utero hematopoietic cell transplantation in mice.| | View in PubMed
- Clinical implications of maternal-fetal cellular trafficking.| | View in PubMed
- Hepatic portocholecystostomy for biliary atresia: a 25-year follow-up and review.| | View in PubMed
- Type IV intestinal atresia, congenital bilateral perisylvian syndrome, and chronic pulmonary hypertension secondary to multiple vascular disruption syndrome in a monochorionic twin.| | View in PubMed
- The diagnosis and management of intradiaphragmatic extralobar pulmonary sequestrations: a report of 4 cases.| | View in PubMed
- Increased maternal microchimerism after open fetal surgery.| | View in PubMed
- Alterations in maternal-fetal cellular trafficking after fetal surgery.| | View in PubMed
- Long-term outcomes after fetal therapy for congenital high airway obstructive syndrome.| | View in PubMed
- In utero hematopoietic cell transplantation for the treatment of congenital anomalies.| | View in PubMed
- Maternal microchimerism in patients with biliary atresia: Implications for allograft tolerance.| | View in PubMed
- Decreased risk of graft failure with maternal liver transplantation in patients with biliary atresia.| | View in PubMed
- Pancreatic mesenchyme regulates epithelial organogenesis throughout development.| | View in PubMed
- The maternal immune response inhibits the success of in utero hematopoietic cell transplantation.| | View in PubMed
- A mouse model of in utero transplantation.| | View in PubMed
- Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice.| | View in PubMed
- Image of the month--quiz case. Gastric duplication cyst.| | View in PubMed
- Long-term surgical outcomes in congenital diaphragmatic hernia: observations from a single institution.| | View in PubMed
- Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice.| | View in PubMed
- Impact of continuous intraoperative monitoring on outcomes in open fetal surgery.| | View in PubMed
- Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors.| | View in PubMed
- Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice.| | View in PubMed
- Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes.| | View in PubMed
- The ex-utero intrapartum treatment.| | View in PubMed
- Engraftment of bone marrow and fetal liver cells after in utero transplantation in MDX mice.| | View in PubMed
- The natural history of prenatally diagnosed conjoined twins.| | View in PubMed
- Human mesenchymal stem cells: insights from a surrogate in vivo assay system.| | View in PubMed
- Risk factors for intermediate-term survival after coronary artery bypass grafting.| | View in PubMed
- Heterogeneity of type I diabetes: analysis of monozygotic twins in Great Britain and the United States.| | View in PubMed
- The impact of the diabetes control and complications trial and humalog insulin on glycohemoglobin levels and severe hypoglycemia in type 1 diabetes.| | View in PubMed
- Human mesenchymal stem cells engraft and demonstrate site-specific differentiation after in utero transplantation in sheep.| | View in PubMed
- Response to treatment as a predictor of longterm outcome in patients with lupus nephritis.| | View in PubMed
- The time-dependence of long-term prediction in lupus nephritis.| | View in PubMed
- Can experienced clinicians predict the outcome of lupus nephritis?| | View in PubMed
- Cerebrovascular deaths before and after the appearance of oral contraceptives.| | View in PubMed
- Systemic and renal hemodynamic consequences of manipulation of serum calcium and/or parathyroid hormone in the intact conscious mongrel dog.| | View in PubMed
- Hemoglobin A1 in renal transplant recipients.| | View in PubMed
- Effect of clonidine therapy on renal hemodynamics in renal transplant hypertension.| | View in PubMed