We believe that unique therapeutic opportunities occur during fetal development. These opportunities create a compelling rationale for the prenatal treatment of many fetal, pediatric and even adult disorders, with potentially less morbidity and mortality than that associated with currently available postnatal treatments. The investigative interests of our group are broad and include not only disorders directly affecting the fetus, but also many disorders that are first manifest in the child or adult.
New Hope for Premature Babies
The combination of advances in maternal screening for fetal disease, fetal imaging, progress with molecular diagnosis of genetic abnormalities, gene chip technology, and the mapping and deciphering of the human genome make it highly likely that within the next decade, nearly all human anatomic and genetic disease will be diagnosed early in gestation.
The early gestational diagnosis of a disease will expand the management options available to families. If significant advantages for fetal treatment over postnatal treatment exist, many families will be candidates for consideration of prenatal therapy.
Advantages of fetal treatment
A variety of biological opportunities are present in the fetus that favor fetal over adult treatment.
The fetus has considerably more plasticity at both the tissue and cellular levels than the adult. This may allow restoration of organ development and function after correction of an anatomic anomaly, or the restoration or genetic correction of a stem cell compartment that may not be possible later in life.
The early gestational fetus is immunologically immature and can be "tolerized" to cells, organs, or proteins that are immunologically foreign, avoiding the potential for rejection and the need for immunosuppression.
Stem cell biology
Stem cells exist at higher frequency, are more proliferative, and have higher accessibility in the early gestational fetus than in postnatal life. In addition, it is the only time in life that stem cell compartments are being formed to develop into tissues.
The small size of the fetus, and the small cell number allow relatively large doses of donor cells or vector to be administered.
Our research interests
Currently, our investigative efforts focus on the following areas of interest:
- In utero stem cell transplantation
- In utero gene therapy (see a selection of recent publications)
- Prenatal and perinatal treatment of congenital diaphragmatic hernia
- Fetal and adult wound healing and regeneration
- Pathogenesis and treatment of myelomeningocele
- Pathogenesis and treatment of gastroschisis
- Alan W. Flake, MD, FACS, FAAP (Director)
- N. Scott Adzick, MD, MMM, FAAP, FACS
- Marcus Davey, PhD
- Holly L. Hedrick, MD, FAAP, FACS
- William H. Peranteau, MD
Visit the Fetal Therapy Research Affinity Group website for more about our research.
Fetal therapy research resources
North American Fetal Therapy Network (NAFTNet)
NAFTNet is a voluntary association of institutions in the U.S. and Canada with expertise in fetal surgery and therapy. It fosters collaborative research to advance the field of fetal medicine. Members can use the site to access current research protocols and participate in research studies.