New technique scours the genome for genes that combat disease

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Using a modified version of the CRISPR genome-editing system, MIT researchers have developed a new way to screen for genes that protect against specific diseases.

CRISPR is normally used to edit or delete genes from living cells. However, the MIT team adapted it to randomly turn on or off distinct gene sets across large populations of cells, allowing the researchers to identify genes that protect cells from a protein associated with Parkinson’s disease.

The new technology, described in the journal Molecular Cell, offers a new way to seek drug targets for many diseases, not just Parkinson’s, says Timothy Lu, an MIT associate professor of electrical engineering and computer science and of biological engineering.

“The state of the art right now is targeting two or three genes simultaneously and then looking at the effects, but we think that perhaps the gene sets that need to be modulated to address some of these diseases are actually broader than that,” says Lu, who is the senior author of the study.

The paper’s lead authors are postdoc Ying-Chou Chen and graduate student Fahim Farzadfard.

Turning genes on or off

The CRISPR genome-editing system consists of a DNA-cutting enzyme called Cas9 and short RNA guide strands that target specific sequences of the genome, telling Cas9 where to make its cuts. Using this process, scientists can make targeted mutations in the genomes of living animals, either deleting genes or inserting new ones.

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