Exploiting Genome Editing Technology To Modify And Regulate The Fshd Disease Locus

Recent discoveries of DNA-binding factors have opened up tremendous new possibilities in genome editing. Through the grant, this study will take advantage of and leverage an existing research program in genome editing of FSHD iPS cells, and will provide the field with valuable new tools to study the pathogenesis of FSHD, and to develop cell therapies based on corrected, isogenic, iPS cells.

Budget: $125,000 in total, supported in partnership with the FSH Society (50:50)

Michael Kyba, Ph.D. – Lillehei Heart Institute, University of Minnesota (Minneapolis, MN)