
Director
Broad Stem Cell Center
+1 415 476-2538
UCSF Broad Stem Cell Center Director, Tippi MacKenzie, MD, is focused on developing safe and effective stem cell transplantation and other therapies for fetuses with congenital anomalies. In utero stem cell transplantation could treat diseases in which stem cells are missing or mutated, such as immunodeficiencies, inborn errors of metabolism, or muscular dystrophy.
Publications
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.
Science translational medicine
Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases.
American journal of medical genetics. Part C, Seminars in medical genetics
The Conundrum of Mechanics Versus Genetics in Congenital Hydrocephalus and Its Implications for Fetal Therapy Approaches: A Scoping Review.
Prenatal diagnosis
Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells.
bioRxiv : the preprint server for biology
Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus.
Molecular therapy. Methods & clinical development
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Molecular therapy : the journal of the American Society of Gene Therapy
Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.
Prenatal diagnosis
Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies.
American journal of human genetics
Are we prepared to deliver gene-targeted therapies for rare diseases?
American journal of medical genetics. Part C, Seminars in medical genetics
The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.
Blood advances
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.
The New England journal of medicine
Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.
Prenatal diagnosis
Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).
Journal of clinical pharmacology
Thymic and extrathymic Aire-expressing cells in maternal-fetal tolerance.
Immunological reviews
Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.
Orphanet journal of rare diseases
Consensus statement for the perinatal management of patients with α thalassemia major.
Blood advances
Advances in the management of α-thalassemia major: reasons to be optimistic.
Hematology. American Society of Hematology. Education Program
Prenatal Gene Therapy.
Clinical obstetrics and gynecology
Maternal and Infant Immune Repertoire Sequencing Analysis Identifies Distinct Ig and TCR Development in Term and Preterm Infants.
Journal of immunology (Baltimore, Md. : 1950)
Exome sequencing of fetuses with congenital diaphragmatic hernia supports a causal role for NR2F2, PTPN11, and WT1 variants.
American journal of surgery
Extrathymic Aire-expressing cells support maternal-fetal tolerance.
Science immunology
Forever Connected: The Lifelong Biological Consequences of Fetomaternal and Maternofetal Microchimerism.
Clinical chemistry
Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement.
Molecular therapy : the journal of the American Society of Gene Therapy
Exome Sequencing for Prenatal Diagnosis in Nonimmune Hydrops Fetalis.
The New England journal of medicine
Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.
Science translational medicine
CD161 contributes to prenatal immune suppression of IFN?-producing PLZF+ T cells.
The Journal of clinical investigation
In Utero Gene Therapy Consensus Statement from the IFeTIS.
Molecular therapy : the journal of the American Society of Gene Therapy
Evaluation of Clinical Outcomes of Sutureless vs Sutured Closure Techniques in Gastroschisis Repair.
JAMA surgery
Depletion of murine fetal hematopoietic stem cells with c-Kit receptor and CD47 blockade improves neonatal engraftment.
Blood advances
Future AAVenues for In Utero Gene Therapy.
Cell stem cell
Meta-Analysis of Maternal and Fetal Transcriptomic Data Elucidates the Role of Adaptive and Innate Immunity in Preterm Birth.
Frontiers in immunology
Alloreactive fetal T cells promote uterine contractility in preterm labor via IFN-γ and TNF-α.
Science translational medicine
Systemic multilineage engraftment in mice after in utero transplantation with human hematopoietic stem cells.
Blood advances
Esophagoesophagopexy technique for assisted fistulization of esophageal atresia.
Pediatric surgery international
Non-Immune Hydrops Fetalis: Do Placentomegaly and Polyhydramnios Matter?
Journal of ultrasound in medicine : official journal of the American Institute of Ultrasound in Medicine
Sacrococcygeal teratoma: late recurrence warrants long-term surveillance.
Pediatric surgery international
Fetal stem cell and gene therapy.
Seminars in fetal & neonatal medicine
Epidemiology of Live Born Infants with Nonimmune Hydrops Fetalis-Insights from a Population-Based Dataset.
The Journal of pediatrics
Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature.
Prenatal diagnosis
A Transient Developmental Hematopoietic Stem Cell Gives Rise to Innate-like B and T Cells.
Cell stem cell
Magnetic resonance imaging of Müllerian duct anomalies in children.
Pediatric radiology
Heightened Immune Activation in Fetuses with Gastroschisis May Be Blocked by Targeting IL-5.
Journal of immunology (Baltimore, Md. : 1950)
Mass Effect Alone May Not Explain Pulmonary Vascular Pathology in Severe Congenital Diaphragmatic Hernia.
Fetal diagnosis and therapy
Fetal Surgical conditions and the unraveling of maternal-fetal tolerance.
Journal of pediatric surgery
Maternal factors associated with the occurrence of gastroschisis.
American journal of medical genetics. Part A
Consensus statement from the first international conference for in utero stem cell transplantation and gene therapy.
Frontiers in pharmacology
In utero hematopoietic cell transplantation for hemoglobinopathies.
Frontiers in pharmacology
Placental drug delivery for treatment of congenital hematopoietic disorders.
Journal of pediatric surgery
The many faces of hydrops.
Journal of pediatric surgery
Novel non-surgical prenatal approaches to treating congenital diaphragmatic hernia.
Seminars in fetal & neonatal medicine
Favorable outcomes in high-risk congenital pulmonary airway malformations treated with multiple courses of maternal betamethasone.
Journal of pediatric surgery
Maternal-fetal cellular trafficking: clinical implications and consequences.
Current opinion in pediatrics
Memory regulatory T cells reside in human skin.
The Journal of clinical investigation
Outcomes of fetal intervention for primary hydrothorax.
Journal of pediatric surgery
Fetal intervention increases maternal T cell awareness of the foreign conceptus and can lead to immune-mediated fetal demise.
Journal of immunology (Baltimore, Md. : 1950)
Anal duplication in a one-year-old girl.
Journal of pediatric surgery case reports
Fetal production of growth factors and inflammatory mediators predicts pulmonary hypertension in congenital diaphragmatic hernia.
Pediatric research
Clinical implications of maternal-fetal cellular trafficking.
Seminars in pediatric surgery
Hepatic portocholecystostomy for biliary atresia: a 25-year follow-up and review.
Journal of pediatric surgery
Type IV intestinal atresia, congenital bilateral perisylvian syndrome, and chronic pulmonary hypertension secondary to multiple vascular disruption syndrome in a monochorionic twin.
Journal of pediatric surgery
The diagnosis and management of intradiaphragmatic extralobar pulmonary sequestrations: a report of 4 cases.
Journal of pediatric surgery
Alterations in maternal-fetal cellular trafficking after fetal surgery.
Journal of pediatric surgery
Long-term outcomes after fetal therapy for congenital high airway obstructive syndrome.
Journal of pediatric surgery
In utero hematopoietic cell transplantation for the treatment of congenital anomalies.
Clinics in perinatology
Maternal microchimerism in patients with biliary atresia: Implications for allograft tolerance.
Chimerism
Decreased risk of graft failure with maternal liver transplantation in patients with biliary atresia.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons
The maternal immune response inhibits the success of in utero hematopoietic cell transplantation.
Chimerism
A mouse model of in utero transplantation.
Journal of visualized experiments : JoVE
Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice.
The Journal of clinical investigation
Image of the month--quiz case. Gastric duplication cyst.
Archives of surgery (Chicago, Ill. : 1960)
Long-term surgical outcomes in congenital diaphragmatic hernia: observations from a single institution.
Journal of pediatric surgery
Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice.
Molecular therapy : the journal of the American Society of Gene Therapy
Impact of continuous intraoperative monitoring on outcomes in open fetal surgery.
Fetal diagnosis and therapy
Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors.
The journal of gene medicine
Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice.
The journal of gene medicine
Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes.
Molecular therapy : the journal of the American Society of Gene Therapy
The ex-utero intrapartum treatment.
Current opinion in pediatrics
Engraftment of bone marrow and fetal liver cells after in utero transplantation in MDX mice.
Journal of pediatric surgery
The natural history of prenatally diagnosed conjoined twins.
Journal of pediatric surgery
Human mesenchymal stem cells: insights from a surrogate in vivo assay system.
Cells, tissues, organs
Risk factors for intermediate-term survival after coronary artery bypass grafting.
The Annals of thoracic surgery
Large-volume paracentesis in the management of ascites in children.
Journal of pediatric gastroenterology and nutrition
Survival after first esophageal variceal hemorrhage in patients with biliary atresia.
The Journal of pediatrics
The management of prenatally diagnosed choledochal cysts.
Journal of pediatric surgery
A fetal lung lesion consisting of bronchogenic cyst, bronchopulmonary sequestration, and congenital cystic adenomatoid malformation: the missing link?
Fetal diagnosis and therapy
Human mesenchymal stem cells persist, demonstrate site-specific multipotential differentiation, and are present in sites of wound healing and tissue regeneration after transplantation into fetal sheep.
Blood cells, molecules & diseases
Heterogeneity of type I diabetes: analysis of monozygotic twins in Great Britain and the United States.
Diabetologia
Human mesenchymal stem cells engraft and demonstrate site-specific differentiation after in utero transplantation in sheep.
Nature medicine
Response to treatment as a predictor of longterm outcome in patients with lupus nephritis.
The Journal of rheumatology
The time-dependence of long-term prediction in lupus nephritis.
Arthritis and rheumatism
Cerebrovascular deaths before and after the appearance of oral contraceptives.
Acta neurologica Scandinavica
Systemic and renal hemodynamic consequences of manipulation of serum calcium and/or parathyroid hormone in the intact conscious mongrel dog.
Journal of the American College of Nutrition
Hemoglobin A1 in renal transplant recipients.
Archives of internal medicine
Effect of clonidine therapy on renal hemodynamics in renal transplant hypertension.
Archives of internal medicine