Fetal Therapy: Current Status and Future Directions — A Stanford Symposium

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.