Minigenes enhance heterologous expression and prevent aberrant splicing of mouse Spink1.

ABSTRACT: The digestive enzyme trypsin is an important driver of pancreatitis development and progression. Inhibition of intrapancreatic trypsin activity is an attractive yet so far unsuccessful approach for the treatment and prevention of pancreatitis. Gene therapy using adeno-associated viral vector containing the human serine protease inhibitor Kazal type 1 (SPINK1) gene has shown efficacy against murine pancreatitis in preclinical experiments. To advance this method to clinical testing, viral vectors with improved inhibitor expression are needed. In this study, we investigated the effect of minigenes with single introns on the expression of the mouse Spink1 gene. We found that minigenes markedly stimulated the expression of mouse Spink1 mRNA and SPINK1 protein in HEK 293T cells transfected with

SUBMITTER: Berke G 

PROVIDER: S-EPMC12318039 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature