Identification of Gene‐Therapy Responsive Blood Biomarkers in mdx Mouse Model
Camilla Johansson,
Jessica F. Boehler,
Kristy J. Brown
et al.
Abstract:IntroductionIdentifying serum biomarkers that reflect the restoration of dystrophin in skeletal muscle is important for evaluating the effect of dystrophin‐restoring therapies in preclinical and clinical trials. Many potential blood biomarkers have been identified in Duchenne muscular dystrophy (DMD) patients, which change with disease progression or respond to pharmacological treatment. In this study, it was suggested that a panel of such blood biomarker candidates could be used to monitor dystrophin rescue i… Show more
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