Miller’s Miracle: In Motion
Phase One: Researching Miller
This December, we traveled to NextGen at the University of Missouri to kick off a functional study that represents phase one of finding Miller her miracle. The study is using a sample of Miller’s skin to allow her cells to be studied in vitro. This will include modeling her disease, or identifying exactly what is going on in her cells as a result of her genetic mutation.
In tandem, we are also working with RareSHIFT at Unravel Biosciences to perform a drug repurposing screen using samples of Miller’s RNA. These results will then be able to be tested/validated with the NextGen team to further understand which therapeutics will have the highest probability of helping correct what is going wrong. Once we know these results, we can move onto phase two: developing the therapeutic.
Phase one is rounded out by the creation of a mouse model in partnership with The Jackson Laboratory. Miller’s mouse will have her exact genetic mutation, allowing us to further study and test the safety and efficacy of various therapeutics.
Phase Two: Making A Miracle
Once we have the learnings of the functional study, we can begin to develop the therapeutic. Whether that’s a personalized gene therapy, RNA-based therapy, gene editing, or funding a trial for drug repurposing, we’re looking at a multi-million dollar endeavor for this next phase.
With phase one successfully fully funded thanks to our generous donors, we are focusing on funding phase two now. Join us is helping make Miller’s Miracle possible.