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AssigneeThe Regents of the University of California
InventorsEdward Hsiao, Kelly Wentworth
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Compositions and methods are provided for screening for an agent that inhibits activity of a hyperactive stimulatory guanine nucleotide binding protein alpha subunit (Gsa) for use in treating disorders associated with GNAS hyperactivation. In particular, induced pluripotent stem cells (iPSCs) are provided for therapeutic screening that comprise a genetically modified GNAS gene comprising an activating mutation, wherein the genetically modified GNAS gene comprises a coding sequence encoding a hyperactive stimulatory guanine nucleotide binding protein alpha subunit (Gsa). The GNAS activating mutation may be linked to fibrous dysplasia, McCune-Albright syndrome, cancer, benign tumors, or other GNAS-associated disorders.
A method of screening for an agent that inhibits activity of a hyperactive stimulatory guanine nucleotide binding protein alpha subunit (Gsα) for use in treating a guanine nucleotide-binding protein alpha, stimulatory (GNAS)-associated disorder, the method comprising:
A composition comprising:
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