Targeted Integration in Stem Cells

We have harnessed homologous recombination to modify the genome of human stem cells with high frequencies (>40%). By designing the recombination donor in different ways, we can achieve a wide range of targeted integration outcomes including creating single nucleotide changes, inserting one gene into another gene, simultaneously inserting two genes into the genome, or even replacing one gene with another. In this talk, I will discuss some of these applications to engineering human stem cells to treat patients.

Learning Objectives:

1. Discuss homologous recombination is a powerful and efficient mechanism to engineer cells using the principles of synthetic biology.

2. Review donor design for the recombination process is an essential part of harnessing the broad potential of homologous recombination based genome editing.