Prenatal Treatments for Genetic Disorders
A new research program to focus on prenatal treatment approaches for genetic disorders has been organized at Wake Forest Institute for Regenerative Medicine (WFIRM).
The Fetal Research and Therapy Program is led by Professor Graca Almeida-Porada, M.D., Ph.D., and is made up by a multidisciplinary team of faculty at WFIRM. The mission of the program is to pursue basic and translational research to develop optimal prenatal treatment approaches for genetic disorders and other life-threatening conditions.
WFIRM is the only center in North Carolina to currently house such a research program. “As a result of this research program, our scientists will be able to focus on developing novel therapeutic platforms and diagnostic tools for fetal treatment,” said Anthony Atala, M.D., director of WFIRM.
Recent advances in imaging and high-throughput molecular testing have made it possible to diagnose most genetic disorders and other life-threatening conditions relatively early in gestation with minimal risk to the fetus.
“These advances in screening are allowing additional options for prenatal therapies,” said Almeida-Porada, who’s expertise and research is in the area of regenerative capabilities of stem cells and development of cellular and gene delivery approaches to treat genetic and immune-mediated diseases. “Beyond the psychological and financial benefits of curing a disease in utero, there are multiple medical advantages as well.”
The fetus has considerably more regenerative capabilities at both the tissue and cellular levels than the adult, said Almeida-Porada, which may allow for repair of anatomic anomalies, re-establishment of organ function, or correction of genetic disorders in a way that may not be possible after birth.
The fetus also has a more tolerant immune system, she said, which is receptive to accepting proteins, biomaterials, cells or even organs without the need for immunosuppression.
One example of a genetic disorder this program is focused on is hemophilia A, a common inheritable disorder of blood coagulation that has no cure. About 1 in 5,000 children are born each year with the disease. WFIRM researchers are currently exploring the possibility of treating, and possibly curing HA, with a combined cell and gene therapy approach that would happen in utero.