In Utero Stem Cell Transplantation


Therapeutic Interventions

Clinical Trials

Currently Recruiting 

Boost Brittle Bones Before Birth

Phase I/II clinical trial to test the safety and efficacy of pre- and/or postnatal MSC transplantation in the severest forms of OI (type III, severe type IV). Transplantation before birth at the onset of disease should lead to greater efficacy and engraftment with less rejection than transplantation after birth. 

Alpha-Thalassemia Major

Phase I clinical trial to demonstrate the safety, feasibility and efficacy of performing in utero stem cell transplantation on fetuses affected with ATM.  Performing stem cell transplantation at the same time as IUT minimizes any additional procedural risk to the fetus. 

Fetal Research Intervention for Vein of Galen Malformation

While many vein of Galen patients do very well with conventional treatment after birth, there remains a significant fraction who will develop severe cardiopulmonary symptoms soon after delivery, and this subgroup has a high risk of morbidity and mortality, even with expert care. 

An IRB and FDA-approved clinical research trial of fetal brain intervention/embolization for vein of Galen malformations at Boston Children’s Hospital is now open for recruitment.


Join IFeTIS at 

The 25th International Conference on Prenatal Diagnosis and Therapy


6-8 June 2021


Organized by:
International Society for Prenatal Diagnosis 

Advances in Alpha Thalassemia Major: No Longer a Fatal Disease

 8-9 January 


Organized by:
UCSF Center for Maternal-Fetal 
Precision Medicine
This two-part, virtual event for healthcare providers, patients, and families will focus on prenatal and postnatal treatment for ATM, and strategies to expand global access to treatment.

Click below to download the following papers: