Tuesday, September 28, 2010

Huge Step in PWS Research!!!

This is huge!!! There was a PWS Research Workshop held back in November, where a group of scientists involved in PWS research strategized about the top issues and directions for future study. Francis Collins, Ph.D. M.D., the current Director of the National Institutes of Health, was one of the participants in this workshop. One of the top priorities that emerged from the Workshop was the need to create iPS (induced pluripotent stem) cells from a person with PWS.

It happened. :) Why is this important? Theresa Strong, Ph.D., Associate Professor of Medicine at University of Alabama at Birmingham, and Chair of FPWR's Scientific Advisory Board, says this about the iPS cells:
"These cells (which were made by transferring a special set of genes into skin cells from a person with PWS) have the ability to change into a variety of cell types in a dish - muscle cells, pancreatic islet cells, and importantly, neurons. Thus, we now have an ongoing supply of PWS neurons - we can study how their gene expression differs from normal, whether their metabolism is altered, whether neuronal migration and/or interaction is altered, and how their neurochemistry differs from normal. Once they figure out how PWS neurons are different, they can throw some small molecules, existing drugs, new drugs, etc onto them and see if they can get them to behave more like typical neurons. This is an "in vitro" (in the dish) system, so there are some limitations (just b/c a drug works on neurons in a dish doesn't mean it will be able to get into the brain and work the same way), but it gives us so much more to work with than we've had before." 

Thank God (literally!) for super-smart people who care about PWS and who figure out how to do these things!! And thank God for the funding that allowed this to happen. We're expecting good things to come of this!!

Abstract for the study found here: http://www.pnas.org/content/early/2010/09/22/1004487107.short

1 comment:

Katie said...

Wonderful news about the research!!!! We pray God gives them wisdom in what they already have to work with and increase it to benefit the PWS cause. Also, congratulations on your 3rd boy to be born in Feb. Go for it!!! I am praying for you and Alice each day. Love you all bunches!!! Grandma