Treat Genetic Disease in Utero with Mom's Own Cells
UCSF researchers have tackled a decade-long scientific conundrum, and their discovery is expected to lead to significant advances in using stem cells to treat genetic diseases before birth.
Through a series of mouse model experiments, the research team determined that a mother's immune response prevents a fetus from accepting transplanted blood stem cells, and yet this response can be overcome simply by transplanting cells harvested from the mother herself. Amazing!
"This research is really exciting because it offers us a straightforward, elegant solution that makes fetal stem cell transplantation a reachable goal," said senior author Tippi MacKenzie, MD, an assistant professor of pediatric surgery at UCSF and fetal surgeon at UCSF Benioff Children's Hospital. "We now, for the first time, have a viable strategy for treating congenital stem cell disorders before birth."Scientists have long viewed in utero blood stem cell transplantation as a promising treatment strategy for many genetic diseases diagnosed as early as the first trimester of pregnancy, including sickle cell disease and certain immune disorders. Fetal stem cell transplantation involves taking healthy cells from the bone marrow of a donor and transplanting them into the fetus through ultrasound-guided injections. When successful, the implanted cells, or graft, replenish the patient's supply of healthy blood-forming cells.
In theory, the developing fetus with an immature immune system should be a prime target for successful transplantation, since the risk of graft rejection is low and the need for long-term immuno-suppressive therapy may be avoided.
However, most previous attempts to transplant blood stem cells into a human fetus have been unsuccessful, prompting some researchers to lose interest in this promising field, according to MacKenzie, who also is an investigator with the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research.
Findings from the study can be found online, in the Journal of Clinical Investigation. They also will be published in the journal's February 2011 issue.
"The fact that fetal stem cell transplantation has not been very successful has been puzzling, especially given the widely accepted dogma that the immature fetal immune system can adapt to tolerate foreign substances," said co-senior author Qizhi Tang, PhD, an assistant professor of transplant surgery and director of the UCSF Transplantation Research Lab. "The surprising finding in our study is that the mother's immune system is to blame."
"Now that we know a fetus can become tolerant to a foreign stem cell source, we can really think big and consider looking at how other types of stem cells might be used to treat everything from neurological disorders to muscular disorders before birth," MacKenzie added.
2011 Christian Nature





























