A Defective Response to Hedgehog Signaling in Disorders of Cholesterol Biosynthesis

This invention provides a method to stimulate stem or progenitor cells with a signaling molecule in vitro and produce a uniform population of the desired differentiated cell type. For example, with this approach it is possible to stimulate neural stem or progenitor cells with a signaling protein such as the Sonic hedgehog protein and cause the entire population of cells to differentiate as a particular cell type such as motor neurons, or dopaminergic neurons. Current methods of producing differentiated neurons from stem cells result in production of a variety of distinct neuronal cell types. Producing a uniform population of differentiated cell types thus represents a significant improvement which will faciliate cell-based therapies in motor neuron diseases, Parkinson?s disease, or more generally in other diseases in which deficits in a particular cell type occur and cell therapies are aimed at replacing that specific cell type. The method is particularly applicable to in vitro cell differentiation that involve the Hedgehog and TGF-?signaling pathways. Description (Set) Proposed Use (Set) This method could be used for production of differentiated cells in vitro that are to be used for cell therapy. Such therapies may be applicable in diseases such as Parkinson?s, ALS, and other degenerative neurological diseases, or in diabetes or any other disease relating to deficiency of a particular cell type.

Inventor(s): Beachy, Philip A.

Type of Offer: Licensing

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