Fetal Therapy: Current Status and Future Directions
A Stanford Symposium

This multidisciplinary symposium highlighted the most up-to-date basic, translational and clinical research on fetal medicine and therapy. Attendees will learn about cutting-edge investigations around perinatal origins of disease to potential interventions involving gene editing and stem cell therapies. The goal of the symposium is to promote creative collaborations across the Stanford community interested in making an impact on the lives of expectant mothers, children and families — locally and globally.

Listen to a cadre of 10 multidisciplinary speakers who shared the latest in cutting-edge basic, translational and clinical investigations in fetal therapies. Welcoming remarks were delivered by the Dunlevie Center director Dr. Yasser El-Sayed and the symposium was started by Dr. Yair Blumenfeld, Director of Fetal Therapy at LPCH, who spotlighted the multidisciplinary team at LPCH offering variety of therapies for complex fetal conditions. In the first session of the symposium, the attendees heard from Drs. David Cornfield, Karl Sylvester and Anca Pasca about their inventive research on fetal organ development and the opportunities for future therapies in developing lung, gut and brain. This was followed by fascinating stem cell and gene therapy presentations by world know expert in gene therapy Dr. Maria Gracia Roncarolo and her colleagues Drs. Agnieszka Czechowicz and Kyle Cromer – all of whom agreed that the future of stem cell and gene therapy will be to deliver these treatments in utero. The Fetal Therapy symposium was ended by Dr. John Day’s presentation about his impactful work on finding therapies for smooth muscle atrophy and by Dr. Paul Wender, Professor of Chemistry, presenting how research on chemistry may be applied to future fetal therapies. 

Welcome and Introduction to Fetal Therapy

Yasser El-Sayed, MD


Dr. El-Sayed is the Dunlevie MFM Center Director and Chief of Maternal-Fetal Medicine and Obstetrics at Stanford Ob/Gyn. His interests include maternal medical and surgical complications of pregnancy and prenatal diagnosis.

Yair Blumenfeld, MD


Dr. Blumenfeld is the director of Fetal Therapy at Lucile Packard Children’s Hospital Stanford, and has been involved in multidisciplinary clinical and translational research for over 10 years. Dr. Blumenfeld’s research focus is new technologies in prenatal diagnosis and fetal therapy, as well as clinical obstetrics and prematurity.

Session 1: Organ Development

David Cornfield, MD


Over the past 20 years, Dr. Cornfield’s research program has focused upon basic, translational and clinical research, with a primary focus on lung biology. As an active clinician-scientist, delivering care to acutely and chronically ill infants, Dr. Cornfield is highly experienced on the evolution of chronic lung disease of infancy in terms of disease manifestation, management and epidemiology.

Anca Pasca, MD


The research focus of Dr. Pasca’s lab is to understand molecular mechanisms underlying neurodevelopmental disorders associated with premature birth and neonatal brain injury with the long-term goal of translating the lab’s findings into therapeutics.

Karl Sylvester, MD


Dr. Sylvester’s investigative interest is primarily focused on problems of human development and pregnancy, and includes congenital anomalies and acquired disease. Current studies include population health risk stratification methodology development, epidemiologic disease association studies; molecular and cellular metabolic development mechanistic studies and biomarkers for gut development and adaptation.

Session 2: Stem Cell and Gene Editing Therapies

Maria Grazia Roncarolo, MD


Dr. Roncarolo is a pioneer in cell and gene therapy for genetic diseases and a world-renowned expert in immune regulation. As Director of the Center for Definitive and Curative Medicine and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine, she leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.

Agnieszka Czechowicz, MD, PhD


Dr. Czechowicz is a physician-scientist and entrepreneur with 20 years of experience in stem cell biology and the development of new innovative therapies. Her primary clinical interests are in bone marrow failure (including Fanconi anemia and aplastic anemia) and other diseases which could benefit from hematopoietic stem cell transplantation.

Kyle Cromer, PhD


Dr. Cromer is an instructor in the Matthew Porteus Lab in the Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine. The Porteus Lab’s primary interest is to develop homologous recombination as a method of gene therapy for genetic diseases. Dr. Cromer’s core research interest is using genome editing to engineer erythropoiesis for clinical applications.

Carsten Charlesworth


Charlesworth is a PhD student in Nakauchi lab in the Stem Cell and Regenerative Medicine institute. The Nakauchi lab works on the biology of HSCs, immunology, and embryology. His primary interests are in addressing immunological challenges to the delivery of gene editing enzymes in vivo and developing in vivo HSC gene therapies.

Session 3: Medical Therapies

John Day, MD, PhD


As Professor of neurology and pediatrics and the director of the neuromuscular program, Dr. Day leads clinics for patients with neuromuscular disease in the pediatric and adult hospitals. These patients may have muscular dystrophy, ALS, or similar disorders that affect the nerves and muscles in the arms and legs.

Paul Wender, PhD


Dr. Wender is the Bergstrom Professor of Chemistry and courtesy Professor of Chemical and Systems Biology. The Wender Group addresses unsolved problems in chemistry, synthesis, biology, medicine, and materials science with an emphasis on drug discovery, development and delivery and on translational science. Resulting technologies have been licensed by or led to the founding of several new companies.