UCSF Center for Maternal-Fetal Precision Medicine

Prenatal gene therapy takes a major step toward the clinic.

Co-Director Dr. @MackenzieTippi shared plans for a first-in-human trial targeting GM1 gangliosidosis prenatally. bit.ly/FetalGeneThera…

#GeneTherapy #Gangliosidosis #GM1 #PrecisionMedicine

Register at tinyurl.com/ATM-May2026.
Dr. Tippi MacKenzie from UCSF will present results of a phase 1 clinical trial of in utero hematopoietic stem cell transplantation in fetuses with alpha #thalassemia major, including safety, feasibility, and limitations of IUHCT for ATM.

Researchers have made a breakthrough in treating spinal muscular atrophy (SMA) before birth. Their innovative approach delivers a genetic therapy into amniotic fluid, & potentially opens a door to earlier, more effective treatments: @statnews @MeganMolteni bit.ly/STAT-SMA

On May 15 #MPSAwarenessDay, we stand with the #MPS community. We are deeply grateful to the families participating in the prenatal ERT #PEARLTrial @UCSF. Their courage and their contributions are humbling. Meet one of these inspiring families: bit.ly/3OgxCZD #ShineForMPS

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 #Thalassemia Major”
REGISTER: tinyurl.com/ATM-Jun2025