Sensitive dystrophin protein quantification in porcine animal model of Duchenne muscular dystrophy after exon skipping

Maria Schmuck, Germany

Medical Technical Assistant - LMU München

Preclinical studies are essential to establish and verify novel causative treatment approaches for Duchenne muscular dystrophy (DMD). To facilitate preclinical testing of promising compounds, we established a novel animal pig model of DMD carrying an exon 52 deletion in the DMD gene and closely mimicking the human phenotype. Accurate dystrophin quantification in pig muscle is a prerequisite to assess the efficacy of molecular therapy. We examined the optimal conditions for protein extraction, total protein determination, antibody detection upon Western blotting and relative dystrophin quantification techniques. In summary, the STSM in Annemieke Aartsma-Rus’ lab greatly facilitated to improve our dystrophin protein quantification techniques in porcine tissue.

April 2016