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“…DMD muscles lack the dystrophin protein, and dystrophin gene replacement is expected to halt ongoing myofiber turnover. To drive the transcription of various genes in gene therapy protocols, several strong viral enhancer/promoters were used in previous studies, including cytomegalovirus (CMV) [11], [12],[13], Raus sarcoma virus (RSV) [14], [15], [16], cytomegalovirus enhancer, chicken beta-actin (CAG) promoter [16], [17], [18], [19], murine stem cell virus (MSCV) [16], [17], [20], [21], mouse phosphoglycerate-kinase 1 (pGK) [12], [22], [23], and elongation factor 1 (EF1) [12], [24], which are ubiquitous promoters derived from house keeping gene regulation systems in mammalian cells. As the MSCV promoter drives gene expression as strongly as the CMV promoter and achieves stable gene expression in different type of cells, especially muscle fibers, we previously used the MSCV promoter to regulate the expression of a short version of the dystrophin gene in a lentiviral vector [6].…”
Section: Introductionmentioning
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“…DMD muscles lack the dystrophin protein, and dystrophin gene replacement is expected to halt ongoing myofiber turnover. To drive the transcription of various genes in gene therapy protocols, several strong viral enhancer/promoters were used in previous studies, including cytomegalovirus (CMV) [11], [12],[13], Raus sarcoma virus (RSV) [14], [15], [16], cytomegalovirus enhancer, chicken beta-actin (CAG) promoter [16], [17], [18], [19], murine stem cell virus (MSCV) [16], [17], [20], [21], mouse phosphoglycerate-kinase 1 (pGK) [12], [22], [23], and elongation factor 1 (EF1) [12], [24], which are ubiquitous promoters derived from house keeping gene regulation systems in mammalian cells. As the MSCV promoter drives gene expression as strongly as the CMV promoter and achieves stable gene expression in different type of cells, especially muscle fibers, we previously used the MSCV promoter to regulate the expression of a short version of the dystrophin gene in a lentiviral vector [6].…”
Section: Introductionmentioning