STAR family function and interaction with RBM20 for intervening alternative splicing (A07)

在这个项目中，我们的目标是详细描述新的心脏剪接因子SLM2。这包括人类心脏剪接靶标的精细定位，辅助因子和调节元件的破译，以及例如条件性基因敲除小鼠的体外和体内功能特征。由于我们提出了SLM2和RBM20的部分拮抗作用，因此我们将研究转基因IPSC和小鼠的联合模型。总之，所获得的结果将为新型心脏剪接调节因子SLM2和治疗操作的概念验证提供机械方面的见解。

In this project, we aim to in detail characterise the novel cardiac splice factor SLM2. This includes the fine mapping of the splice targets in the human heart, the deciphering of co-factors and regulator elements and the in-vitro and in-vivo functional characterisation e.g. in conditional knock-out mice. Since we propose an partially antagonistic role of SLM2 and RBM20, we will study combined transgenic iPSC and mouse models. Together, the obtained results will provide mechanistic insights into the novel cardiac splice regulator SLM2 and proof-of-concept of therapeutic manipulations.