The goal of the "CRISPR/Cas9 Genome-Wide sgRNA Library Screening Platform" grant is to develop a validated set of reagents that can be used for specific targeted disruption of all human and mouse genes. This resource will provide a powerful tool for researchers to identify, in a single experiment, the genes that are important for cell growth, drug effectiveness, or other biological responses and has wide implications in the discovery of novel drug targets and identifying drug mechanisms.
CRISPR technology can be used to permanently disrupt ("knock out") genes in mammalian cells. Targeting a gene for knockout using CRISPR requires a short strand of RNA (an "sgRNA") that contains a region matching part of the gene sequence. Leveraging its ability to generate complex libraries expressing thousands of sgRNA sequences at once, Cellecta plans to test multiple sgRNA designs against a wide range of genes and identify the optimal sgRNA design for gene knockout. The data obtained from testing these libraries will be used to design sgRNAs for the whole human genome.
"With this project, we expect to eliminate a lot of the current guesswork required when using CRISPR to knock out genes," says Cellecta's principal investigator Dr. Donato Tedesco. "Our project will give researchers reliable and robust reagents for genetic analysis and identification of new drug targets. We're very excited to have this opportunity to increase the utility of this new powerful gene editing technology."
Upon completion of the project next year, Cellecta plans to build a library that targets 6,500 key disease-related genes in major signal transduction pathways. The company will make the CRISPR library available to academic and non-profit groups through the DECIPHER Project website as it did previously with