Tippi MacKenzie, M.D.
- Professor of Surgery
- Division of Pediatric Surgery
- Benioff UCSF Professor in Children’s Health
- Director, Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF
- Co-Director, Center for Maternal-Fetal Precision Medicine
- John G. Bowes Distinguished Professor in Stem Cell and Tissue Biology
Contact Information
Education
- 1987-91, Harvard University, B.A., Biochemistry
- 1992-97, Stanford University School of Medicine, M.D., Medicine
Residencies
- 1998-99, Brigham and Women's Hospital, Resident, Surgery
- 2002-04, Brigham and Women's Hospital, Resident, Surgery
- 2004-05, Brigham and Women's Hospital, Chief Resident, Surgery
Fellowships
- 1999-02, Children's Hospital of Philadelphia, Fellow, Fetal Therapy
- 2005-07, Children's Hospital of Philadelphia, Fellow, Pediatric Surgery
Biography
Dr. Tippi MacKenzie is Professor of Surgery at the UCSF Division of Pediatric Surgery and the Fetal Treatment Center. Dr. MacKenzie obtained her undergraduate degree in Biochemistry at Harvard, then came to the Bay Area for medical school at Stanford. She did her surgical residency at Brigham and Women's Hospital in Boston. During this time, she took three years to do research on fetal surgery and in utero stem cell transplantation at the Children's Hospital of Philadelphia. Following residency, she returned to CHOP for her clinical pediatric surgery fellowship.
Dr. MacKenzie's clinical interests include fetal surgery, advanced laparoscopy, and endocrine and biliary surgery.
Research Overview
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 & Funding
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- Phase 1 Study of In Utero Enzyme Replacement Therapy for the Treatment of Lysosomal Storage DiseasesSponsor: NIH/NICHHDSponsor ID: 1R01HD110270Funding Period:Sep 2022-Apr 2027Principal Investigator
- Developing gene therapy strategies to treat alpha thalassemiaSponsor: NIH/NHLBISponsor ID: 1R01HL161291-01Funding Period:Jan 2022-Dec 2025Principal Investigator
- Autoimmune Regulator gene (Aire)-mediated tolerance to pregnancy-associated self-antigensSponsor: NIH/NIAIDSponsor ID: R01AI145858Funding Period:Aug 2019-Jul 2024Principal Investigator
- In utero hematopoietic stem cell transplantation for the treatment of fetuses with alpha thalassemia major (phase I clinical trial)Sponsor: California Institute for Regenerative Medicine (CIRM)Sponsor ID: CLIN2-09183Funding Period:Aug 2017-Jul 2022Principal Investigator
- A precision medicine approach to detect dysbiosis and immune activation in preterm laborSponsor: Burroughs Wellcome Fund Preterm Labor InitiativeSponsor ID:Funding Period:Jul 2017-Jun 2021Principal Investigator
- T cell activation and the breakdown of maternal-fetal tolerance in preterm laborSponsor: NIH/NIAIDSponsor ID: R01AI116880Funding Period:Jun 2015-May 2020Principal Investigator
- Maternal and fetal immune responses to in utero HSC transplantationSponsor: NIH/NIAIDSponsor ID: K08AI085042Funding Period:Jul 2010-May 2015Principal Investigator
- IN UTERO MESENCHYMAL STEM CELL TRANSPLANTATIONSponsor: NIH/NHLBISponsor ID: F32HL010229Funding Period:Jan 2000-Jan 0001Principal Investigator
Clinical Trials
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- Study ID: NCT05619900Start Date: May 2022Estimated Completion Date: May 2050Condition(s): Mucopolysaccharidosis, Mucopolysaccharidosis Type I, Hunter Syndrome, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type VII, Pompe Disease, Gaucher Disease, Wolman Disease
- Study ID: NCT04532047Start Date: Dec 2020Estimated Completion Date: Nov 2034Condition(s): Mucopolysaccharidosis, Mucopolysaccharidosis Type I, Hunter Syndrome, Mucopolysaccharidosis Type IVA, Mps VII, Gaucher Disease, Pompe Disease, Wolman Disease
- Study ID: NCT02986698Start Date: Oct 2017Estimated Completion Date: Feb 2024Condition(s): Alpha Thalassemia, Alpha Thalassemia Major, Hemoglobinopathy, Anemia, Hydrops Fetalis, Hematopoietic Cell Transplantation
- Study ID: NCT04872179Start Date: Jan 2017Estimated Completion Date: Jan 2037Condition(s): Alpha Thalassemia, Alpha Thalassemia Major
Publications
MOST RECENT PUBLICATIONS FROM A TOTAL OF 101
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- Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namuduri SR, Borges B, Kirn G, Valencia J, Lopez ME, Lui JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC. Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice. Mol Ther. 2024 Apr 03; 32(4):935-951. View in PubMed
- 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. Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects. Prenat Diagn. 2023 12; 43(13):1638-1649. View in PubMed
- 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. Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies. Am J Hum Genet. 2023 09 07; 110(9):1454-1469. View in PubMed
- 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. Are we prepared to deliver gene-targeted therapies for rare diseases? Am J Med Genet C Semin Med Genet. 2023 03; 193(1):7-12. View in PubMed
- 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. In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease. N Engl J Med. 2022 12 08; 387(23):2150-2158. View in PubMed
- View All Publications
Videos
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- Part 1: Tippi and Elianna’s Story: Stopping a Fatal Blood Disease Before Birth
- Part 2: Tippi, Elliott and Elianna’s Story: Little Girl Thrives After Fetal Stem Cell Transplant
- First Clinical Trial of In Utero Stem Cell Transplant for Alpha Thalassemia Major
- Treating Disease Before Birth with Tippi MacKenzie, MD
In the News
- UCSF Campus News - - November 09, 2022Fetal Therapy Offers Hope for Patients with Rare Genetic Disorders Toddler Thrives with Same Disease That Ended Siblings' Lives When Zahid and Sobia Bashir discovered their fetus had the same genetic disease that took the lives of two of their young children, they didn't hesitate to sign up for a promising but experimental new therapy. "I found out (the therapy) was a possibility and I was kind of in shock; I couldn't believe it," said Zahid Basir. "I only [...]
- UCSF Campus News - - October 17, 2022Three UC San Francisco faculty members are among the 100 new national members elected this year to the National Academy of Medicine (NAM), one of the highest honors in the fields of health and medicine. Membership in the NAM recognizes individuals who have demonstrated outstanding professional achievements and commitment to service in the medical sciences, health care and public health. "This extraordinary class of new members is comprised of exceptional scholars and leaders who have been [...]
- UCSF General Surgery Residency Program - - May 08, 2020Eva Gillis-Buck, UCSF School of Medicine Class of 2020 and incoming general surgery resident, was awarded the UCSF 2020 Long-Term Dean's Prize in Research. The Long-Term Dean's Prize is available to all UCSF graduating medical students. Finalists were selected based on the submission of a one-page abstract, letter of nomination, and 5-minute presentation to the faculty review committee. Five prizes for long-term research were awarded and all winners presented a virtual pre-recorded video at [...]
- UCSF Pediatric Surgery - - March 02, 2020"This group of vulnerable patients has been relatively ignored in the fetal surgery world," said UCSF's Tippi MacKenzie, MD, senior author of the new study and member of the Eli and Edythe Broad Center for Regeneration Medicine and Stem Cell Research, who has worked for a decade developing novel therapies for heritable diseases that can be treated before birth. "We know these patients could potentially benefit from a number of medical therapies. So this is our first foray into treating one of [...]
- UCSF Pediatric Surgery - - June 25, 2018UCSF News reports on the first patient enrolled in the world's first clinical trial using blood stem cells transplanted prior to birth. The stem cell transplant was used to treat alpha thalassemia major, a blood disorder that is only detected in the last few months of pregnancy and has been almost uniformly fatal in the past. The clinical trial is led by Tippi MacKenzie, MD, an associate professor in the UCSF Division of Pediatric Surgery and co-Director of the UCSF Center for Maternal-Fetal [...]
- UCSF Surgical Innovations - - May 22, 2018On May 17, the Second Annual UCSF Engineering for Children's Health Symposium was held at Mission Bay Conference Center. Over one hundred clinicians, engineers, scientists, administrators, industry professionals, and government partners gathered to discuss groundbreaking research and developing technologies in pediatric medicine. The event commenced with opening remarks by Hanmin Lee, MD, Surgeon-in-Chief of the UCSF Benioff Children's Hospitals and Professor and Chief of the UCSF Division [...]
- UCSF Pediatric Surgery - - July 10, 2017Pediatric Surgeon Tippi MacKenzie, MD was awarded $12.1 million by the California Institute for Regenerative Medicine (CIRM) to treat fetuses in the womb suffering from alpha thalassemia major, a blood disorder that is only detected in the last few months of pregnancy and is almost uniformly fatal. Dr. MacKenzie, an associate professor in the UCSF Division of Pediatric Surgery and co-Director of the UCSF Center for Maternal-Fetal Precision Medicine, is using hematopoietic stem cells (HSCs), [...]
- Department of Surgery, Surgical Innovations Program - - March 02, 2017On January 12, clinicians, engineers, scientists, and administrators from UCSF, UC Berkeley. UCSF Benioff Children's Hospitals, and Children's Hospital Oakland Research Institute (CHORI) convened at the Mission Bay campus for the inaugural Engineering for Children's Health Symposium. The event, reported in UCSF News, marked the launch of a collaborative effort dedicated to developing innovative technological solutions to improve patient safety, eliminate health disparities, cure disease, and [...]
- UCSF Pediatric Surgery - - May 24, 2016UCSF News reports on the work of a UCSF research team investigating the causes and potential treatments for gastroschisis, a rare disorder in which developing intestines protrude outside of the body through a hole alongside the belly button. Led by pediatric surgeon Tippi MacKenzie, MD, Associate Professor of Surgery in the UCSF Division of Pediatric Surgery and co-Director of the UCSF Center for Maternal-Fetal Precision Medicine, the team published findings elucidating the role of the immune [...]
- UCSF Pediatric Surgery - January 24, 2014UCSF News report on how the immune system drives pregnancy complications after fetal surgery in mice. As a fetal surgeon at UC San Francisco, Tippi MacKenzie, MD, has long known that conducting surgery on a fetus to correct a problems such as spina bifida often results in preterm labor and premature birth. Now, MacKenzie and her UCSF colleagues have shown that, in mice at least, pregnancy complications after fetal surgery are triggered by activation of the mother's T cells – the same T [...]
- UCSF Pediatric Surgery - November 16, 2012UCSF News reports that on a study that suggests maternal liver grafts more tolerable for children with biliary atresia, a rare, life-threatening disease, results that may have important implications for counseling parents on organ donation Children with a rare, life-threatening disease that is the most common cause of neonatal liver failure – biliary atresia – better tolerate liver transplants from their mothers than from their fathers, according to a UCSF-led study. In the study, [...]
- UCSF Pediatric Surgery - January 18, 2011UCSF News reports on fetal stem cell transplantation, taking healthy cells from the bone marrow of a donor, and transplanting them into the fetus through ultrasound-guided injections with the goal of having the implanted cells, or graft, replenish the patient's supply of healthy blood-forming cells. UCSF researchers have tackled a decade-long scientific conundrum, and their discovery is expected to lead to significant advances in using stem cells to treat genetic diseases before birth. [...]
- UCSF Pediatric Surgery - November 02, 2010UCSF announces the formation of the Institute for Fetal and Neonatal Health symposium brings together clinicians and basic scientists involved in different aspects of development and fetal intervention.
- UCSF Pediatric Surgery - June 15, 2009As a gifted pianist in high school, Assistant Professor of Surgery Tippi MacKenzie, MD, spent every Saturday studying music at Juilliard. This musical training was good preparation for her current work as a researcher and surgeon. "When you're playing the piano, you work out the details in a dif cult passage until it's perfect," she said. "It's the same with science. If there's an issue with your experiment, you can't just sweep it under the rug. you tackle that problem." With a [...]