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Cleft Palate in Fetal Mice Prevented by Treating

Mice engineered to have cleft palates can be rescued in utero by injecting the mothers with a small molecule to correct the defect//, say scientists at the Stanford University School of Medicine and Lucile Packard Children's Hospital. In addition to shedding light on the biology of cleft palate, the research raises hopes that it may one day be possible to prevent many types of human birth defects by using a similar vaccination-type technique in pregnant women likely to have affected fetuses.

"This is a really important baby step that opens the door to the development of fetal therapies," said pediatric craniofacial surgeon Michael Longaker, MD. "Our hope and expectation is that patients at Packard Children's and other institutions will benefit as this basic advance is translated into something that will eventually make a significant difference for this and other birth defects."

Longaker is the senior author of the study, which will be published in the Feb. 11 advance online edition of Nature. The research is the first demonstration that a technique known as chemical genetics - in which small molecules are used to modify gene expression or protein activity - can reach a fetus when administered to a pregnant animal.

"It's such a cool concept," said Longaker, professor of medicine and a leader in Stanford's Institute for Stem Cell Biology and Regenerative Medicine. "We injected a small molecule into mom, and it goes into the embryo and works. There are tremendous implications to the idea of preventing conditions in unborn patients rather than trying to treat them after birth." Cleft defects are the second-most common birth defect worldwide and affect about one in 2,000 births.

Longaker, who spent several years in fetal surgery working to design surgical approaches for life-threatening defects prior to coming to Stanford, said the concept of noninvasively preventing these defects by treating the mother is something that wa
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