Surrogate Biomarkers for Duchenne Muscular Dystrophy using SILAC Mouse.

摘要

1. SILAC mouse strategy enabled unambiguous detection and identification of serum biomarkers for the dystrophin deficient mouse model 2. Close examination of the serum proteome revealed 14 highly elevated proteins (10 to 100 fold) and 7 decreased proteins (10 to 100 fold) in the serum of mdx versus wild type mouse. 3. Several of the increased proteins in the serum of mdx mice were previously reported to be elevated in the serum of DMD patients which include creatine kinase M (a diagnostic hallmark biomarker for DMD), aldolase and pyruvate kinase. One decreased protein in the serum of mdx mice, superoxide dismutase was also reported to be decreased in the serum of DMD patients. 4. Novel surrogate biomarkers were confidently identified and quantified. These are currently being validated in serum of DMD patients and healthy controls using a combination of western blot assays and /or targeted mass spectrometry analyses