Posted by Friends of FSH Research on Dec 3, 2018
This award provides support for a dedicated professional highly skilled in FSHD research who performs high-impact studies of FSHD and provides scientific and technical oversight for investigators, graduate students, and postdoctoral fellows seeking to pursue projects in FSHD. In the last six months, we have:
- Completed the collection and processing of samples from year two of our multi-center Seattle Wellstone Center FSHD clinical study and published the assessment of year one (see Wang et al. 2018. Hum Mol Genet. doi: 10.1093/hmg/ddy364), with the goal of identifying biomarkers for disease activity and progression that might be used in future clinical trials.
- Generated a transgenic mouse that has the potential to enhance the utility of mouse models of FHSD, with the goal of improving preclinical studies of FSHD therapeutics.
- Initiated a new collaboration to determine how compounds of interest block the activity of DUX4, with the goal of developing novel therapeutics for FSHD.
- Maintained current collaborations to investigate DUX4-induced changes in the proteome, with the goal of understanding whether they contribute to cell toxicity and disease.
- Provided daily training and oversight to one postdoctoral fellow, three graduate students, three research technicians, and one bioinformatician studying FSHD.
- Facilitated collaborations with other labs to promote FSHD research, including sharing protocols, trouble-shooting experiments, and distributing reagents.
See grant Regulation and activity of the DUX4 transcription factor
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