PHASE I, SBIR CONTRACT, N43TR-14-002, ?AN ASSAY TO MEASURE SURROGATE BIOMARKERS FOR DMD?.

Ariadne Diagnostics, Llc, Rockville MD

Investigators

Abstract

We will employ the mdx mouse model of DMD. The mdx mouse has a premature stop codon in exon 23 of the dystrophin gene and as a result lacks this key protein, similarly to the lack of dystrpohin in DMD affected individuals. This model has been used extensively in DMD research and specifically in the development of HaloBr. The model is from the background of the common laboratory strain, C57BL10/ScSn, and as such this strain serves as a control for the mdx model. As with any animal model of disease, some variability has been seen in results coming from the mdx mouse, however, recently extensive work has been done to unify protocols for the use of this model system and have been disseminated by the TREAT-NMD network. We will follow these protocols especially for the effective measure of functional outcomes.