Pluripotent Stem Cells
In Vitro Model Of Spinal Muscular Atrophy
WARF: P09194US02
Inventors: Clive Svendsen, Allison Ebert
The Invention
A population of iPS cells derived from somatic cells from a spinal muscular atrophy patient is disclosed. In one embodiment of the invention, the cells have been cultured to produce neural cells. In another embodiment, the invention is a method of testing compounds for their ability to modify cellular SMN levels comprising the steps of obtaining a population of iPS cells derived from a spinal muscular atrophy patient or cells derived from the iPS cells, and examining the effect of a test compound on SMN levels.
Tech Fields
For current licensing status, please contact Andy DeTienne at [javascript protected email address] or 608-960-9857