Good news: Facebook voting for the Rare Disease Challenge is open! Vote for us here! update: you can vote every 24 hours. please do! If we win we’ll be awarded $10,000 in seed money to get started on our research goals. This early money will be really helpful for us in getting preliminary results that will help us compete for grants in the near future.
Our proposal, submitted late last year with a research team led by George Carlson, is to convert a sample of Sonia’s skin cells to induced pluripotent stem cells and use ‘molecular scissors’ called zinc finger nucleases to create a batch of them that is genetically identical except for the single DNA base mutation that causes fatal familial insomnia. With this tight experimental control in hand, we can study the appearance and gene expression of the cells in great detail to see what is going wrong in the FFI cells, and eventually be able to screen for drugs that make the FFI cells act more like the healthy ‘corrected’ cells.
Surprisingly, according to the list of winners we weren’t the only fatal familial insomnia project in the competition! Christina Chao, we Googled you and couldn’t find you – if you see this drop us a line to let us know who you are – we should combine forces!