Medical Research

University of California, Berkeley

Jennifer Doudna, Alex Marson, Peidong Yang
Berkeley, CA
$1,000,000
December 2015

CRISPR-Cas9 is a powerful technology for engineering genomic sequences.  Efforts are underway to use CRISPR-Cas9 directly in human tissues, but delivery efficiency has been limited, especially in human T cells where genome engineering would be a crucial tool to dissect genetic mechanisms controlling immune homeostasis.  A team of University of California, Berkeley and University of California, San Francisco investigators proposes a comprehensive approach to delivering Cas9 protein-RNA complexes into primary human T cells that will be of broad biomedical utility.  The team plans to develop a delivery system that is: 1) efficient and simple to use; 2) independent of T cell stimulation or other ex vivo manipulations of cells; and, 3) tolerated by T cells with minimal toxicity.  Nanowires will serve as ‘nano-syringes,’ transiently puncturing the cell membrane to deliver the genome editing machinery without inflicting permanent cell damage.  This interdisciplinary effort aims to adapt nanowire technology for high throughput genetic engineering of human T cells for experimental and therapeutic purposes.  Genome engineering T cells will offer insights into the genetic basis of immune-mediated diseases, providing a tool to test the cellular consequences of coding and non coding DNA variation.  This project also lays the foundation for a new generation of T cell based immunotherapies for infections, autoimmunity and cancer.

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