Promoting axonal regeneration in models of ALS via activation of the preconditioning response in iPSC-derived human neurons

2013 Pilot Project Read More

Investigators

Principal Investigator: Aaron DiAntonio (WUSTL Developmental Biology)
Co-Investigators:  Jeffrey Milbrandt (WUSTL Genetics), Timothy Miller (WUSTL Neurology)

Description

Our objective is to find new treatments for neuronal injury and disease by promoting regeneration of axons through the “preconditioning” response. Preconditioning is the phenomenon where a prior neuronal injury stimulates an improved regenerative response to a subsequent injury. Our long-term goal is to identify candidate drugs and/or genes that can activate the pro-regenerative preconditioned state without the need for a prior injury. To accomplish this goal, we will study the preconditioning response of human neurons derived from induced pluripotent stem cells (iPSCs). These stem cells are themselves derived from human skin and so are readily accessible and can be derived from patients with neurological disease. These patient-derived iPSCs will allow us to explore cellular models of neurological disease, such as ALS, in which enhanced regeneration would be helpful. If successful, these studies will allow us to perform large-scale screens for factors that promote regeneration of human neurons.

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Support

This pilot project is made possible by the Danforth Foundation Challenge.

Danforth Challenge