Chemical rescue of cleft palate and midline defects in conditional GSK-3β mice

Karen J. Liu (), Joseph R. Arron, Kryn Stankunas, Gerald R. Crabtree and Michael T. Longaker ()
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Karen J. Liu: The Department of Pathology,
Joseph R. Arron: The Department of Pathology,
Kryn Stankunas: Department of Developmental Biology,
Gerald R. Crabtree: The Department of Pathology,
Michael T. Longaker: The Stanford Institute for Stem Cell Biology and Regenerative Medicine,

Nature, 2007, vol. 446, issue 7131, 79-82

Abstract: Cleft palate repair The development of a novel chemical genetics strategy to regulate an important developmental protein (GSK-3β) in a mouse fetus in a reversible time-controlled fashion could have important implications. Remarkably, this control can be achieved within the mother's uterus. GSK-3β (glycogen synthase kinase-β) is involved in many different biological processes, and mice engineered to lack it have cleft palates: this birth defect is reversed by the in utero rescue. Several key advances are required before this research can have clinical relevance, but the demonstration that small-molecule based therapy can prevent a birth defect is an important proof of principle.

Date: 2007
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DOI: 10.1038/nature05557

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