Tippi MacKenzie, MD

Professor of Surgery, Co-director, UCSF Center for Maternal-Fetal Precision Medicine
Program Lead: Women's Precision Medicine
Committee Members

Tippi MacKenzie is a pediatric and fetal surgeon who is focused on developing better ways to diagnose and treat genetic diseases before birth. She leads a translational research lab examining the unique biology between the mother and her fetus, with the idea that pregnancy complications such as preterm labor arise from a breakdown in maternal-fetal tolerance. At present, the MacKenzie group is focused on preterm labor and fetal therapies including stem cell transplantation, enzyme replacement therapy, and gene therapy.


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

Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namduri S, Borges B, Kirn G, Valencia J, Lopez ME, Lu JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC

Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.

Prenatal diagnosis

Herzeg A, Borges B, Lianoglou BR, Gonzalez-Velez J, Canepa E, Munar D, Young SP, Bali D, Gelb MH, Chakraborty P, Kishnani PS, Harmatz P, Cohen JL, MacKenzie TC

Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies.

American journal of human genetics

Lowther C, Valkanas E, Giordano JL, Wang HZ, Currall BB, O'Keefe K, Pierce-Hoffman E, Kurtas NE, Whelan CW, Hao SP, Weisburd B, Jalili V, Fu J, Wong I, Collins RL, Zhao X, Austin-Tse CA, Evangelista E, Lemire G, Aggarwal VS, Lucente D, Gauthier LD, Tolonen C, Sahakian N, Stevens C, An JY, Dong S, Norton ME, MacKenzie TC, Devlin B, Gilmore K, Powell BC, Brandt A, Vetrini F, DiVito M, Sanders SJ, MacArthur DG, Hodge JC, O'Donnell-Luria A, Rehm HL, Vora NL, Levy B, Brand H, Wapner RJ, Talkowski ME

Are we prepared to deliver gene-targeted therapies for rare diseases?

American journal of medical genetics. Part C, Seminars in medical genetics

Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, Gold NB, Kennedy A, Kishnani PS, Might M, Brooks PJ, Morris JA, Parisi MA, Urv TK

The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.

Blood advances

MacKenzie TC, Schwab ME, Lianoglou BR, Gano D, Gonzalez J, Arvon RL, Baschat AA, Bianchi DW, Bitanga M, Bourguignon A, Brown RN, Chen B, Chien M, Davis-Nelson S, de Laat MWM, Ekwattanakit S, Gollin Y, Hirata G, Jelin A, Jolley J, Meyer P, Miller J, Norton ME, Ogasawara KK, Panchalee T, Schindewolf E, Shaw SW, Stumbaugh TL, Thompson AA, Towner D, Tsai PS, Viprakasit V, Volanakis E, Vichinsky E, Allen IE, Zhang L

In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.

The New England journal of medicine

Cohen JL, Chakraborty P, Fung-Kee-Fung K, Schwab ME, Bali D, Young SP, Gelb MH, Khaledi H, DiBattista A, Smallshaw S, Moretti F, Wong D, Lacroix C, El Demellawy D, Strickland KC, Lougheed J, Moon-Grady A, Lianoglou BR, Harmatz P, Kishnani PS, MacKenzie TC

Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.

Prenatal diagnosis

Schwab ME, Shao S, Zhang L, Lianoglou B, Belter L, Jarecki J, Schroth M, Sumner CJ, MacKenzie T

Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).

Journal of clinical pharmacology

Herzeg A, Almeida-Porada G, Charo RA, David AL, Gonzalez-Velez J, Gupta N, Lapteva L, Lianoglou B, Peranteau W, Porada C, Sanders SJ, Sparks TN, Stitelman DH, Struble E, Sumner CJ, MacKenzie TC

Thymic and extrathymic Aire-expressing cells in maternal-fetal tolerance.

Immunological reviews

Sun IH, Gillis-Buck E, Mackenzie TC, Gardner JM

Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.

Orphanet journal of rare diseases

Schwab ME, Brown JEH, Lianoglou B, Jin C, Conroy PC, Gallagher RC, Harmatz P, MacKenzie TC

Consensus statement for the perinatal management of patients with alpha thalassemia major.

Blood advances

MacKenzie TC, Amid A, Angastiniotis M, Butler C, Gilbert S, Gonzalez J, Keller RL, Kharbanda S, Kirby-Allen MA, Koenig BA, Kyono W, Lal A, Lianoglou BR, Norton ME, Ogasawara KK, Panchalee T, Rosner M, Schwab ME, Thompson AA, Waye JS, Vichinsky E

Advances in the management of α-thalassemia major: reasons to be optimistic.

Hematology. American Society of Hematology. Education Program

Horvei P, MacKenzie T, Kharbanda S

Prenatal Gene Therapy.

Clinical obstetrics and gynecology

Schwab ME, MacKenzie TC

Maternal and Infant Immune Repertoire Sequencing Analysis Identifies Distinct Ig and TCR Development in Term and Preterm Infants.

Journal of immunology (Baltimore, Md. : 1950)

Le BL, Sper R, Nielsen SCA, Pineda S, Nguyen QH, Lee JY, Boyd SD, MacKenzie TC, Sirota M

Exome sequencing of fetuses with congenital diaphragmatic hernia supports a causal role for NR2F2, PTPN11, and WT1 variants.

American journal of surgery

Schwab ME, Dong S, Lianoglou BR, Aguilar Lucero AF, Schwartz GB, Norton ME, MacKenzie TC, Sanders SJ

Extrathymic Aire-expressing cells support maternal-fetal tolerance.

Science immunology

Gillis-Buck E, Miller H, Sirota M, Sanders SJ, Ntranos V, Anderson MS, Gardner JM, MacKenzie TC

Forever Connected: The Lifelong Biological Consequences of Fetomaternal and Maternofetal Microchimerism.

Clinical chemistry

Bianchi DW, Khosrotehrani K, Way SS, MacKenzie TC, Bajema I, O'Donoghue K

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

Sagar R, Almeida-Porada G, Blakemore K, Chan JKY, Choolani M, Götherström C, Jaulent A, MacKenzie TC, Mattar C, Porada CD, Peranteau WH, Schneider H, Shaw SW, Waddington SN, Westgren M, David AL

Exome Sequencing for Prenatal Diagnosis in Nonimmune Hydrops Fetalis.

The New England journal of medicine

Sparks TN, Lianoglou BR, Adami RR, Pluym ID, Holliman K, Duffy J, Downum SL, Patel S, Faubel A, Boe NM, Field NT, Murphy A, Laurent LC, Jolley J, Uy C, Slavotinek AM, Devine P, Hodoglugil U, Van Ziffle J, Sanders SJ, MacKenzie TC, Norton ME

Detection of microbial cell-free DNA in maternal and umbilical cord plasma in patients with chorioamnionitis using next generation sequencing.

PloS one

Witt RG, Blair L, Frascoli M, Rosen MJ, Nguyen QH, Bercovici S, Zompi S, Romero R, Mackenzie TC

Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.

Science translational medicine

Nguyen QH, Witt RG, Wang B, Eikani C, Shea J, Smith LK, Boyle G, Cadaoas J, Sper R, MacKenzie JD, Villeda S, MacKenzie TC

CD161 contributes to prenatal immune suppression of IFN?-producing PLZF+ T cells.

The Journal of clinical investigation

Halkias J, Rackaityte E, Hillman SL, Aran D, Mendoza VF, Marshall LR, MacKenzie TC, Burt TD

In Utero Gene Therapy Consensus Statement from the IFeTIS.

Molecular therapy : the journal of the American Society of Gene Therapy

Almeida-Porada G, Waddington SN, Chan JKY, Peranteau WH, MacKenzie T, Porada CD

Evaluation of Clinical Outcomes of Sutureless vs Sutured Closure Techniques in Gastroschisis Repair.

JAMA surgery

Witt RG, Zobel M, Padilla B, Lee H, MacKenzie TC, Vu L

Large Differences in Small RNA Composition Between Human Biofluids.

Cell reports

Godoy PM, Bhakta NR, Barczak AJ, Cakmak H, Fisher S, MacKenzie TC, Patel T, Price RW, Smith JF, Woodruff PG, Erle DJ

Future AAVenues for In Utero Gene Therapy.

Cell stem cell

MacKenzie TC

Meta-Analysis of Maternal and Fetal Transcriptomic Data Elucidates the Role of Adaptive and Innate Immunity in Preterm Birth.

Frontiers in immunology

Vora B, Wang A, Kosti I, Huang H, Paranjpe I, Woodruff TJ, MacKenzie T, Sirota M

Alloreactive fetal T cells promote uterine contractility in preterm labor via IFN-? and TNF-a.

Science translational medicine

Frascoli M, Coniglio L, Witt R, Jeanty C, Fleck-Derderian S, Myers DE, Lee TH, Keating S, Busch MP, Norris PJ, Tang Q, Cruz G, Barcellos LF, Gomez-Lopez N, Romero R, MacKenzie TC

Systemic multilineage engraftment in mice after in utero transplantation with human hematopoietic stem cells.

Blood advances

Witt RG, Kreger EM, Buckman LB, Moradi PW, Ho PT, Derderian SC, Tsai P, Baker C, Schramm N, Cleary R, Garcia JV, MacKenzie TC

Esophagoesophagopexy technique for assisted fistulization of esophageal atresia.

Pediatric surgery international

Chumfong I, Lee H, Padilla BE, MacKenzie TC, Vu LT

Non-Immune Hydrops Fetalis: Do Placentomegaly and Polyhydramnios Matter?

Journal of ultrasound in medicine : official journal of the American Institute of Ultrasound in Medicine

Berger VK, Sparks TN, Jelin AC, Derderian C, Jeanty C, Gosnell K, Mackenzie T, Gonzalez JM

Sacrococcygeal teratoma: late recurrence warrants long-term surveillance.

Pediatric surgery international

Padilla BE, Vu L, Lee H, MacKenzie T, Bratton B, O'Day M, Derderian S

Fetal stem cell and gene therapy.

Seminars in fetal & neonatal medicine

Witt R, MacKenzie TC, Peranteau WH

Epidemiology of Live Born Infants with Nonimmune Hydrops Fetalis-Insights from a Population-Based Dataset.

The Journal of pediatrics

Steurer MA, Peyvandi S, Baer RJ, MacKenzie T, Li BC, Norton ME, Jelliffe-Pawlowski LL, Moon-Grady AJ

Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature.

Prenatal diagnosis

Kreger EM, Singer ST, Witt RG, Sweeters N, Lianoglou B, Lal A, Mackenzie TC, Vichinsky E

A Transient Developmental Hematopoietic Stem Cell Gives Rise to Innate-like B and T Cells.

Cell stem cell

Beaudin AE, Boyer SW, Perez-Cunningham J, Hernandez GE, Derderian SC, Jujjavarapu C, Aaserude E, MacKenzie T, Forsberg EC

Magnetic resonance imaging of Müllerian duct anomalies in children.

Pediatric radiology

Li Y, Phelps A, Zapala MA, MacKenzie JD, MacKenzie TC, Courtier J

Heightened Immune Activation in Fetuses with Gastroschisis May Be Blocked by Targeting IL-5.

Journal of immunology (Baltimore, Md. : 1950)

Frascoli M, Jeanty C, Fleck S, Moradi PW, Keating S, Mattis AN, Tang Q, MacKenzie TC

Mass Effect Alone May Not Explain Pulmonary Vascular Pathology in Severe Congenital Diaphragmatic Hernia.

Fetal diagnosis and therapy

Derderian SC, Jayme CM, Cheng LS, Keller RL, Moon-Grady AJ, MacKenzie TC

A booster shot to cure hemoglobinopathies.


Walters MC, MacKenzie TC

Maternal factors associated with the occurrence of gastroschisis.

American journal of medical genetics. Part A

Baer RJ, Chambers CD, Jones KL, Shew SB, MacKenzie TC, Shaw GM, Jelliffe-Pawlowski LL

Consensus statement from the first international conference for in utero stem cell transplantation and gene therapy.

Frontiers in pharmacology

MacKenzie TC, David AL, Flake AW, Almeida-Porada G

In utero hematopoietic cell transplantation for hemoglobinopathies.

Frontiers in pharmacology

Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC

Placental drug delivery for treatment of congenital hematopoietic disorders.

Journal of pediatric surgery

Derderian SC, Moradi PW, MacKenzie TC

The many faces of hydrops.

Journal of pediatric surgery

Derderian SC, Jeanty C, Fleck SR, Cheng LS, Peyvandi S, Moon-Grady AJ, Farrell J, Hirose S, Gonzalez J, Keller RL, MacKenzie TC

Novel non-surgical prenatal approaches to treating congenital diaphragmatic hernia.

Seminars in fetal & neonatal medicine

Jeanty C, Kunisaki SM, MacKenzie TC

Favorable outcomes in high-risk congenital pulmonary airway malformations treated with multiple courses of maternal betamethasone.

Journal of pediatric surgery

Derderian SC, Coleman AM, Jeanty C, Lim FY, Shaaban AM, Farrell JA, Hirose S, MacKenzie TC, Lee H

Maternal-fetal cellular trafficking: clinical implications and consequences.

Current opinion in pediatrics

Jeanty C, Derderian SC, Mackenzie TC

In utero depletion of fetal hematopoietic stem cells improves engraftment after neonatal transplantation in mice.


Derderian SC, Togarrati PP, King C, Moradi PW, Reynaud D, Czechowicz A, Weissman IL, MacKenzie TC

Memory regulatory T cells reside in human skin.

The Journal of clinical investigation

Sanchez Rodriguez R, Pauli ML, Neuhaus IM, Yu SS, Arron ST, Harris HW, Yang SH, Anthony BA, Sverdrup FM, Krow-Lucal E, MacKenzie TC, Johnson DS, Meyer EH, Löhr A, Hsu A, Koo J, Liao W, Gupta R, Debbaneh MG, Butler D, Huynh M, Levin EC, Leon A, Hoffman WY, McGrath MH, Alvarado MD, Ludwig CH, Truong HA, Maurano MM, Gratz IK, Abbas AK, Rosenblum MD

Outcomes of fetal intervention for primary hydrothorax.

Journal of pediatric surgery

Derderian SC, Trivedi S, Farrell J, Keller RL, Rand L, Goldstein R, Feldstein VA, Hirose S, MacKenzie TC

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)

Wegorzewska M, Nijagal A, Wong CM, Le T, Lescano N, Tang Q, MacKenzie TC

Anal duplication in a one-year-old girl.

Journal of pediatric surgery case reports

Cheng LS, Courtier J, MacKenzie TC

Fetal production of growth factors and inflammatory mediators predicts pulmonary hypertension in congenital diaphragmatic hernia.

Pediatric research

Fleck S, Bautista G, Keating SM, Lee TH, Keller RL, Moon-Grady AJ, Gonzales K, Norris PJ, Busch MP, Kim CJ, Romero R, Lee H, Miniati D, MacKenzie TC

Clinical implications of maternal-fetal cellular trafficking.

Seminars in pediatric surgery

Nijagal A, MacKenzie TC

Hepatic portocholecystostomy for biliary atresia: a 25-year follow-up and review.

Journal of pediatric surgery

Schecter SC, Courtier J, Cho SJ, Saadai P, Hirose S, Mackenzie TC, Miniati D

The diagnosis and management of intradiaphragmatic extralobar pulmonary sequestrations: a report of 4 cases.

Journal of pediatric surgery

Nijagal A, Jelin E, Feldstein VA, Courtier J, Urisman A, Jones KD, Lee H, Hirose S, MacKenzie TC

Alterations in maternal-fetal cellular trafficking after fetal surgery.

Journal of pediatric surgery

Saadai P, Lee TH, Bautista G, Gonzales KD, Nijagal A, Busch MP, Kim CJ, Romero R, Lee H, Hirose S, Rand L, Miniati D, Farmer DL, MacKenzie TC

Long-term outcomes after fetal therapy for congenital high airway obstructive syndrome.

Journal of pediatric surgery

Saadai P, Jelin EB, Nijagal A, Schecter SC, Hirose S, MacKenzie TC, Rand L, Goldstein R, Farrell J, Harrison M, Lee H

In utero hematopoietic cell transplantation for the treatment of congenital anomalies.

Clinics in perinatology

Nijagal A, Flake AW, MacKenzie TC

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

Nijagal A, Fleck S, Hills NK, Feng S, Tang Q, Kang SM, Rosenthal P, MacKenzie TC

Pancreatic mesenchyme regulates epithelial organogenesis throughout development.

PLoS biology

Landsman L, Nijagal A, Whitchurch TJ, Vanderlaan RL, Zimmer WE, Mackenzie TC, Hebrok M

A mouse model of in utero transplantation.

Journal of visualized experiments : JoVE

Nijagal A, Le T, Wegorzewska M, Mackenzie TC

Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice.

The Journal of clinical investigation

Nijagal A, Wegorzewska M, Jarvis E, Le T, Tang Q, MacKenzie TC

Image of the month--quiz case. Gastric duplication cyst.

Archives of surgery (Chicago, Ill. : 1960)

Zaid HB, Rhee SJ, Hirose S, Mackenzie TC

Long-term surgical outcomes in congenital diaphragmatic hernia: observations from a single institution.

Journal of pediatric surgery

Jancelewicz T, Vu LT, Keller RL, Bratton B, Lee H, Farmer D, Harrison M, Miniati D, Mackenzie T, Hirose S, Nobuhara K

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

Sabatino DE, Mackenzie TC, Peranteau W, Edmonson S, Campagnoli C, Liu YL, Flake AW, High KA

Impact of continuous intraoperative monitoring on outcomes in open fetal surgery.

Fetal diagnosis and therapy

Keswani SG, Crombleholme TM, Rychik J, Tian Z, Mackenzie TC, Johnson MP, Wilson RD, Flake AW, Hedrick HL, Howell LJ, Adzick NS

Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors.

The journal of gene medicine

MacKenzie TC, Kobinger GP, Louboutin JP, Radu A, Javazon EH, Sena-Esteves M, Wilson JM, Flake AW

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

Bouchard S, MacKenzie TC, Radu AP, Hayashi S, Peranteau WH, Chirmule N, Flake AW

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

MacKenzie TC, Kobinger GP, Kootstra NA, Radu A, Sena-Esteves M, Bouchard S, Wilson JM, Verma IM, Flake AW

The ex-utero intrapartum treatment.

Current opinion in pediatrics

MacKenzie TC, Crombleholme TM, Flake AW

Engraftment of bone marrow and fetal liver cells after in utero transplantation in MDX mice.

Journal of pediatric surgery

Mackenzie TC, Shaaban AF, Radu A, Flake AW

The natural history of prenatally diagnosed conjoined twins.

Journal of pediatric surgery

Mackenzie TC, Crombleholme TM, Johnson MP, Schnaufer L, Flake AW, Hedrick HL, Howell LJ, Adzick NS

Risk factors for intermediate-term survival after coronary artery bypass grafting.

The Annals of thoracic surgery

Gardner SC, Grunwald GK, Rumsfeld JS, Mackenzie T, Gao D, Perlin JB, McDonald G, Shroyer AL

Heterogeneity of type I diabetes: analysis of monozygotic twins in Great Britain and the United States.


Redondo MJ, Yu L, Hawa M, Mackenzie T, Pyke DA, Eisenbarth GS, Leslie RD

Human mesenchymal stem cells engraft and demonstrate site-specific differentiation after in utero transplantation in sheep.

Nature medicine

Liechty KW, MacKenzie TC, Shaaban AF, Radu A, Moseley AM, Deans R, Marshak DR, Flake AW

Response to treatment as a predictor of longterm outcome in patients with lupus nephritis.

The Journal of rheumatology

Fraenkel L, MacKenzie T, Joseph L, Kashgarian M, Hayslett JP, Esdaile JM

The time-dependence of long-term prediction in lupus nephritis.

Arthritis and rheumatism

Esdaile JM, Abrahamowicz M, MacKenzie T, Hayslett JP, Kashgarian M

Can experienced clinicians predict the outcome of lupus nephritis?


Esdaile JM, Mackenzie T, Barr? P, Danoff D, Osterland CK, Somerville P, Quintal H, Kashgarian M, Suissa S

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

Zawada ET, Johnson M, McClung D, TerWee J, MacKenzie T

Hemoglobin A1 in renal transplant recipients.

Archives of internal medicine

Zawada ET, Johnson M, Mackenzie T, Sica DA, Muakkassa W, Green SJ, Goldman M

Effect of clonidine therapy on renal hemodynamics in renal transplant hypertension.

Archives of internal medicine

Green S, Zawada ET, Muakkassa W, Johnson M, Mackenzie T, McClanahan M, Graybill A, Goldman M