Exploiting Genome Editing Technology 2015

FSHD Canada and the FSH Society are jointly funding a project called “Exploiting Genome Editing Technologies,” led by Michael Kyba, PhD, of the University of Minnesota. The two FSHD organizations joined forces last year to support the first year of the US$125,000 project, with each putting in US$62,500. Based on the success of the first year, they agreed to fund the second year of the project for the same amount.

“The past several years have seen tremendous advances in our ability to specifically modify DNA sequences in the human genome,” said Kyba. “FSHD is caused by the combination of two factors: the contraction of D4Z4 repeat number combined with the presence of a pathogenic sequence downstream of the D4Z4 repeats. Both of these DNA elements are accessible to modification using newly developed genome editing technology. The Kyba lab has developed methods of gene targeting that address both elements and will use these to derive and study genetically corrected FSHD cell lines.”

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