Western blotting has recently been utilized as the primary endpoint for the detection of the dystrophin protein recovery in gene therapy clinical studies. The method for the detection of dystrophin protein levels in the clinical muscle tissue extracts using this method has been developed through extensive in-house method development and validation processes. Biopsy banks provide normal controls, and disease controls (DMD, BMD). Assays are tailored to the sponsor’s need and validated prior to testing clinical samples.
Western Blotting
