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Gene editing strategies to cure human diseases postnatally

日期: 2018-10-11

主講人: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.


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