Affiliation:
1. Seoul National University
2. LART Bio Inc
Abstract
Abstract
As the understandings about the biotechnology and the pathophysiology of diseases getting advanced, the genetic materials or genetically engineered cells have brought hopes on patients with inherited diseases. Among many congenital diseases, the muscular dystrophy has been globally one of the major subjects of genetic therap. To apply genetic therapy selectively in muscular tissue, the promoters which express genes specifically in muscle have been necessitated by researchers. In the current study, the promoter region of MSTN gene was postulated as candidate muscle-specific promoter for gene therapy, from the biological significance and muscle-specific distribution of the myostatin. Accordingly, we aimed to isolate a novel promoter for gene therapy from the MSTN gene promoter and trim it more suitable for the therapeutic applications. During the experiments, it was revealed that the MSTN promoter region have functionally distinguishable parts: the highly conserved core region and the region that react to myogenic differentiation. The core region of bovine MSTN gene promoter showed ubiquitous expression of marker gene in differentiated cell lines or cells with stemness, originated from human, mouse, and cattle. In conclusion, we suggest the proximal region of bovine MSTN gene promoter as novel ubiquitous promoter.
Publisher
Research Square Platform LLC