Joined February 2019
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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/FetalGeneTherapy
#GeneTherapy #Gangliosidosis #GM1 #PrecisionMedicine
ALT A woman with short brown hair and glasses smiles at the camera; the headline reads "Fetal Gene Therapy Makes Strides Toward the Clinic" on The Scientist website.
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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.
ALT International Seminar Series on Alpha Thalassemia Major, May 20, 2026, 5-6pm PDT. Tippi MacKenzie, MD, from the University of California, San Francisco, Department of Surgery, will present: In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major. Register at https://tinyurl.com/ATM-May2026
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
17 Oct 2025
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
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
7 Aug 2025
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!
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
Join us for a symposium & reception: A Decade of Impact: Celebrating @atulbutte & BCHSI@10, Tuesday, August 19th 1-5pm: calendar.ucsf.edu/event/a-de… @UCSF
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
27 May 2025
‼️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 👇 tiny.ucsf.edu/StromalCellsAR…
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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
ALT Microscopic cross-section of a mouse head stained with fluorescent markers, showing ASO 449323–Cy3, yellow, and nuclei stained blue in the brain; STAT news logo in corner and "In The Lab" in lower left
Researchers have taken preliminary steps toward treating spinal muscular atrophy in the womb trib.al/uGyaYoC
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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
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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
ALT Seminar Announcement: Title: In Utero Transfusions (IUT) Beyond 35 Weeks Gestation in Alpha #Thalassemia Major ' Speaker: Dr. Evangelia Vlachodimitropoulou from King’s College Hospital Date/Time: June 12, 2025, 8am PDT / 4pm BST Link to register: https://tinyurl.com/ATM-Jun2025
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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 bit.ly/4jV38tL
ALT Microscopic cross-section of a mouse head stained with fluorescent markers, showing ASO 449323–Cy3, yellow, and nuclei stained blue in the brain; Science Translational Medicine logo in the corner.
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
14 May 2025
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
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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: Â A world where all children are born healthy.
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
30 Apr 2025
#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
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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: bit.ly/3Mb9uqg
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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. tiny.ucsf.edu/15AQrW
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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!
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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
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UCSF Center for Maternal-Fetal Precision Medicine retweeted
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: bit.ly/4g4CVXv
ALT ASGCT's virtual event on March 12–13, 2025, offers cell and gene therapy education, insights, and advocacy discussions.
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Honoring #RareDiseaseDay and the strength of the #rare community. Your voices drive progress, and together, we’re working toward better treatments. #RareDiseaseAwareness #PEARLTrial pearltrial.ucsf.edu
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