UCSF Center for Maternal-Fetal Precision Medicine

05/26/2026

UCSF Center for Maternal-Fetal Precision Medicine Co-Director Dr. Tippi MacKenzie will lead a first-in-human clinical trial of prenatal gene therapy for GM1 gangliosidosis — a rare and fatal genetic disorder in which irreversible damage occurs before birth. The intention is to treat prenatally to improve outcomes and establish a model for other prenatal gene therapies.

Read more: https://www.the-scientist.com/fetal-gene-therapy-makes-strides-toward-the-clinic-74497

04/23/2026

SEMINAR: Please join us on 5/20, 5pm PDT, when Dr. Tippi MacKenzie from UCSF “In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Major”
REGISTER: https://tinyurl.com/ATM-May2026

REGISTER and join us on May 20, 2026, 5:00-6:00 pm (PDT) for "In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major "

01/08/2026

SEMINAR: Please join us on 1/29, 8am PST, when Prof. Khaled Musallam from Burjeel Cancer Institute, Burjeel Medical City, will present “Current Understanding of Alpha Burden: Evidence and Gaps”
REGISTER: https://tinyurl.com/ATM-Jan2026

REGISTER and join us on January 29, 2026, 8:00-9:00 am (PST) for "Current Understanding of Alpha Thalassemia Burden: Evidence and Gaps"

06/12/2025

An example of the value of federally funded medical research: This prenatal diagnosis research, supported by NICHD funding, showed that chromosomal microarray analysis examined chromosomes in finer detail and picked up additional genetic defects compared with traditional karyotyping testing, leading to changes in prenatal diagnostic testing recommendations.



Read SMFM Consult Series #41: https://publications.smfm.org/publications/584-society-for-maternal-fetal-medicine-consult-series-41/

05/27/2025

A clinical study led by highlights the utility of exome sequencing for different types of fetal effusions and discerns patterns in how genetic diseases present in utero. We continue to offer genome sequencing for pregnancies with one or more fetal effusions through ongoing research ([email protected]). Recent findings are published in AJOG: https://bit.ly/4mulOm2

05/21/2025

SEMINAR: Please join us on 6/12, 8am PDT, when Dr. Evangelia Vlachodimitropoulou from King’s College Hospital, will present “In Utero Transfusions (IUT) Beyond 35 Weeks Gestation in Alpha Major”
REGISTER: https://tinyurl.com/ATM-Jun2025

05/20/2025

Researchers UCSF & Johns Hopkins Medicine have made a breakthrough in treating spinal muscular atrophy (SMA) before birth. Their innovative approach delivers a genetic therapy into the amniotic fluid of pregnant mice, improving motor function in pups with SMA. This promising study opens the door to potentially treating SMA earlier and more effectively in humans. Learn more about their work in this STAT article by Megan Molteni: https://bit.ly/STAT-SMA

Researchers have taken preliminary steps toward treating spinal muscular atrophy in the womb

05/15/2025

Today, May 15, we proudly observe and stand in solidarity with the community. We thank our patients who participated in the prenatal ERT . Their courage and their contributions are humbling. Meet one of these inspiring families: https://bit.ly/3OgxCZD 💜

Some parents-to-be have to face the terrifying news that their unborn child has been diagnosed with a life-threatening genetic disorder.

05/14/2025

Intra-amniotic antisense oligonucleotide (A*O) treatment has been shown to improve phenotypes in preclinical models of Spinal Muscular Atrophy (SMA). This innovative approach suggests the potential for a minimally invasive prenatal therapy for SMA and potentially other early-onset neurological disorders. Discover more about this pioneering research by Beltrán Borges and colleagues from the lab of Dr. Tippi MacKenzie at UCSF, in collaboration with UC Davis and Johns Hopkins Medicine, published in Science Translational Medicine: https://bit.ly/4jV38tL

Antisense oligonucleotides delivered in utero can treat mouse models of SMA and can feasibly be administered to fetal lambs at midgestation.

04/15/2025

April 15 is , and we stand in solidarity with the global community and raise awareness of .

We’re grateful for the support and collaboration of this community, which has made it possible to enroll three IOPD patients in our .

We’re excited about the potential of prenatal enzyme replacement therapy to change the course of this condition — and proud to share the story of the first IOPD family receiving this protocol.

In a first-of-its-kind fetal therapy, Dr. Tippi MacKenzie, a pediatric surgeon at UCSF, began treating Ayla Bashir’s Pompe disease before she was even born. Garvin Thomas reports.