Gene editing has opened vast new areas of research and will soon be applied clinically in rare genetic diseases, cancer immunotherapies, and many other applications. While the early phase of gene editing approaches using targetable nucleases such as CRISPR/Cas9 has centered on research and therapeutic applications of gene knockouts, a much broader potential exists when extending the technology to gene knockins. Integrating large new DNA sequences at defined genomic sites will underpin the next generation of gene and cell therapies. This process is dependent on both the delivery method of a new DNA sequence (in addition to a targetable nuclease) into the cell, as well as the nature of the new DNA template.

In this webinar, Theo Roth will talk about the delivery of DNA through electroporation of ex vivo primary human T cells, and discuss different types of homology-directed repair (HDR) templates and their various applications. Special attention will be devoted to varying effects on editing efficiency, cell viability, ease of production, and therapeutic applicability.

About the presenter

Theo Roth

MD/PhD student at UCSF

Theo Roth is an MD/PhD student at the University of California, San Francisco, working with Dr. Alexander Marson. He studied at Stanford and researched at the National Institutes of Health before coming to UCSF to further develop gene editing tools in human T cells for cancer and autoimmune cell therapy applications. He has developed a novel nonviral gene targeting method that greatly simplifies the process of knocking in new DNA at defined genomic sites in T cells.