Horizon eBook - Drug Discovery - 16

CRISPR-Based Cellular and In Vivo Applications for Drug Discovery * CRISPR-Fine Screen of Human Genome Identifies Essential Genes

This work, which was led by Whitehead Member David
Sabatini and Broad Institute Director Eric Lander, used the
CRISPR-Cas9 approach to investigate cell lines derived from
two cancers, chronic myelogenous leukemia (CML) and Burkitt's
lymphoma. The novel method not only identified the essentiality
of the known genes-in the case of CML, it hit on the BCR and
ABL1 genes, whose translocation is the target of the successful
drug Gleevec-but also highlighted additional genes that may be
therapeutic targets in these cancers.
"The ability to zero in on the essential genes in the highly
complex human system will give us new insight into how diseases,
such as cancer, continue to resist efforts to defeat them," said
Dr. Lander.
"This is really the first time we can reliably, accurately,
and systematically study genetics in mammalian cells," added
Dr. Sabatini. "It's remarkable how well it's working." n

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Horizon eBook - Drug Discovery

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