About The Pax7-Foxo1 Mouse- an international collaboration (crowdfunded)
Many years ago Fred, Janet and Charles competed to define the function of the Pax3-Foxo1 mutation in alveolar rhabdomyosarcoma, a hard-to-treat muscle cancer. With time, we know so much more and potential treatments are emerging. And of course, competitors became friends.
Yet for children with rhabdomyosarcoma, a small but important number of alveolar rhabdomyosarcoma patients have a variation in the mutation, being Pax7-Foxo1 (that is, Pax7 instead of Pax3). The general thought was that Pax7-Foxo1 patients do better than Pax3-Foxo1 patients, but this might not always be true. Certainly, saddening outcomes do happen for these patients, all too frequently.
To create a silver lining, we need more research. But research needs tools!
(no tools = no research, but powerful tools = potential breakthroughs!)
Up to now, resources for Pax7-Foxo1 rhabdomyosarcoma are very, very limited. Only two patient-derived cell lines exist for Pax7-Foxo1, whereas dozens exist for Pax3-Foxo1. And only one patient-derived xenograft model exists for Pax7-Foxo1.
Most importantly, no transgenic (genetically-engineered) mice exist ... which is perhaps the greatest barrier to discovery. GEM mice can be studies in so many ways to really get to the bottom of a disease.
ALRIGHT - HERE'S WHERE WE JUMPSTART DISCOVERY!
Fred, Janet and Charles are going to make the Pax7-Foxo1 GEM mouse together, study the GEM at the same time and share results regularly. And we're going to invite others to work together to jumpstart discovery, too - including Dr. Tadashi Kondo, who studies Pax7-Foxo1 ARMS at the Japan National Cancer Center Research Institute!
Thank you for participating in this crowdfunding campaign (goal=$53,000 total) to make this special Pax7-Foxo1 GEM mouse! Together, we will discover ways to give back childhood to these kids.
Charles Keller MD, www.cc-tdi.org, USA
Janet Shipley PhD, www.icr.ac.uk, GREAT BRITAIN
Fred Relaix PhD, https://www.relaixlab.com, FRANCE