内网

检测到您当前使用浏览器版本过于老旧,会导致无法正常浏览网站;请您使用电脑里的其他浏览器如:360、QQ、搜狗浏览器的极速模式浏览,或者使用谷歌、火狐等浏览器。

下载Firefox

Gene editing strategies to cure human diseases postnatally

日期: 2018-10-11

主讲人:Prof. DOUGLAS R. CAVENER

Verne M. Willaman Dean, Eberly College of Science, Penn State University



Abstract: CRISPR-Cas9 gene editing has provided a powerful method to create targeted mutations. However, repairing preexisting mutations that cause human disease is challenging and inefficient. We are developing new strategies to improve the efficacy and efficiency of CRISPR-based homology directed repair (HDR) and homolog independent targeted integration (HITI). These new strategies are being applied to mutations in the insulin and PERK genes that cause severe monogenic diabetes. In this seminar I will present our progress in developing these tools in cell-culture and mouse models and discuss future applications to cure human genetic diseases.

欢迎各位老师同学积极参加!