FSHD Canada and Friends of FSH Research Team Up to Fund Three Innovative Research Projects.

Budget

FSHD Canada funded half of each project. Dr. Parker: $50,000. Dr. Wagner/Natoya: $50,000. Dr. Aihara/Dr. Kyba: $33397.

Lay Abstract

FSHD Canada has partnered with Friends of FSH Research to solicit, review, and fund three exciting projects that will accelerate our progress towards a treatment or cure for FSH muscular dystrophy.

This cycle, we received more applications than ever for projects and choosing the final three was quite difficult. The first project that was accepted by the Friends’ Scientific Advisory Board was from Dr. Eric Wagner at the University of Texas. Dr. Wagner and his graduate student, Natoya Peart, have done some amazing work understanding how RNA molecules get packaged. By specifically focusing on sequences within DUX4, the candidate target gene for FSHD, they believe they can develop novel DNA therapeutics. DNA therapeutics are not entirely a pipe-dream. There are a number that are being developed for clinical trials in other diseases. According to Dr. Wagner, these therapeutics are gaining a “resurgence in clinical trials” and some have been given approval by the FDA.

The second project being co-funded is with Drs. Hideki Aihara and Michael Kyba. Their project focuses on two elements of the structure of DUX4. First, it will identify the optimal binding characteristics of DNA bound by DUX4, and second, will seek to identify the structure of the DUX4 protein bound to that DNA. Understanding the structure of DNA and how it interacts with it’s target DNA is critical for the development and fine tuning of compounds designed to interfere with DUX4 function.

Last, but certainly not least, based on a successful review or her 1 year project that was co-funded last year, FSHD Canada and Friends of FSH Research have continued funding Dr. Maura Parker, as she continues to develop the methodologies for isolating and classifying a new class of muscle precursor cells that will function to repopulate the skeletal muscle of mice, as well as to improve cellular models of FSHD.

Friends of FSH Research is a Seattle based non-profit that raises money, solicits, reviews, and funds research towards a treatment or cure for FSH Muscular Dystrophy.

Links of Interest: Friends of FSH Research