UCSF Center for Maternal-Fetal Precision Medicine

We discover genes that drive fetal anomalies & develop fetal molecular therapies to treat serious genetic diseases before birth.
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[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

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. https://t.co/NH8iv9ZQhZ

#GeneTherapy #Gangliosidosis #GM1 #PrecisionMedicine

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

TOMORROW!

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

Register at https://t.co/8xdxUR44h5.
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.

[University of California @UofCalifornia]

We're bursting with pride at the University of California after last week's record-breaking Nobel Prize announcements, where FIVE UC scientists were honored in three categories. But this is about more than bragging rights. It's about the future of revolutionary, life changing discoveries that are only possible because of federal funding for science. UC President James B. Milliken explains what's at stake 👇

If you care about our nation's health, security and prosperity, please stand with UC and #SpeakUp4Science

[Dr. Alison Bateman-House @ABatemanHouse]

Join us 9/24/25, 11am-1pm ET, 3rd Annual Early Career Scholars Meeting, presented by NYU Grossman School of Medicine Division of Medical Ethics Working Group on Pediatric Gene Therapy & Medical Ethics (PGTME). Junior scholars at all levels of academia welcome; registration FREE!

[UCSF Bakar Computational Health Sciences Institute @UCSF_BCHSI]

Join us for a symposium & reception: A Decade of Impact: Celebrating @atulbutte & BCHSI@10, Tuesday, August 19th 1-5pm: https://t.co/0vn0vNzQ0p @UCSF

[UCSF Surgery @UCSFSurgery]

‼️Join us Thurs, 5/29 for Getting to Patients as Dr. Michael Matthay presents groundbreaking research on bone marrow-derived stromal cells for ARDS—bridging pre-clinical science, clinical trials, and real patient experience. Moderated by Dr. Julia Brown 👇 https://t.co/Ad4b1VAJyH

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

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 https://t.co/NbpfEh3qjO

[STAT @statnews]

Researchers have taken preliminary steps toward treating spinal muscular atrophy in the womb https://t.co/xpyXl8wCmv

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

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: https://t.co/kRCYtkCE5p #ShineForMPS

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

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: https://t.co/Y2dDYB33BL

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

Intra-amniotic ASO treatment improves phenotypes in preclinical SMA models, suggesting a promising, minimally invasive prenatal approach for SMA and other early-onset neurological disorders. @MacKenzieTippi lab, @UCSF, @UCDavis, @HopkinsMedicine https://t.co/DksIrVyPjr

[UCSF Broad Stem Cell @UCSFstemcell]

Hear how gene therapies can address serious genetic diseases before birth. @MacKenzieTippi will deliver a George Stamatoyannopoulos Memorial Lecture at the @ASGCT Annual Meeting on May 15.  #ASGCT2025 #GeneTherapy #PrenatalResearch #raredisease

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

10 years and counting. These are a few of the many remarkable people who contribute to the ambitious vision of our Center co-directors, Mary Norton and Tippi MacKenzie:  A world where all children are born healthy.

[Elizabeth Crouch, MD/PhD @ElizabetsCrouch]

#Crouchlab and friends and family at the @UCSFcmfpm 10 year anniversary celebration 🍾 🎉thrilled to be a part of this #visionary group now w/#clinicaltrials for #inutero therapies. Let’s go for it, for children’s future health 👶🏻👧🏾🧒🏼@UCSFPediatrics @NeoTECaN @UCSFstemcell

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

April 15 is #InternationalPompeDay. We stand with the #Pompe community and are grateful for enrolling 3 #IOPD patients in our prenatal ERT #PEARLTrial. We're excited by the positive impact, as seen in the story of the first family receiving the protocol: https://t.co/YTyex8LGLy

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

Please RT! Applications are due June 1 for this special research fellowship in #FetalDiagnosis and #FetalTherapy. Experience our vibrant training program, mentorship, multidisciplinary collaboration, and cutting-edge fetal diagnostics and therapies. https://t.co/CTpjjsvk4V

[ACMG @TheACMG]

Happening now at #ACMGMtg25: Poster presentations (authors present at odd-numbered posters), 10:30 AM – 12:00 PM. LACC, Exhibit Hall DE. Go see some amazing #medicalgenetics research!

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

Attending #ACMGMtg25? Learn about the latest advances and clinical findings at our poster presentations and Learning Lounge on Thursday, March 20. We hope to connect with you in LA! #PEARLTrial @billielianolgou

[National Organization for Rare Disorders (NORD) @RareDiseases]

Register now for "Empowering Patients 2025: A Cell and Gene Therapies Summit" on March 12–13, hosted by @ASGCTherapy! This free, virtual event in two half-day sessions offers educational discussions for foundation leaders, patients, and caregivers to better understand #GeneTherapy and #CellTherapy. Register here: https://t.co/toYHz1VWHx

[UCSF Center for Maternal-Fetal Precision Medicine @UCSFcmfpm]

Honoring #RareDiseaseDay and the strength of the #rare community. Your voices drive progress, and together, we’re working toward better treatments. #RareDiseaseAwareness #PEARLTrial https://t.co/VlMTowg7er