Researchers have harnessed a bacterial immune defense system, known as CRISPR, to efficiently and precisely control the process of RNA splicing. The technology opens the door to new applications, including systematically interrogating the functions of parts of genes and correcting splicing deficiencies that underlie numerous diseases and disorders.
Researchers at the University of Toronto have harnessed a bacterial immune defense system, known as CRISPR, to efficiently and precisely control the process of RNA splicing.
Exons can be alternatively spliced, such that the regulation and function of the approximately 20,000 human genes that encode proteins are greatly diversified, allowing the development and functional specialization of different types of cells. "Our new effector protein activated alternative splicing of around 90 percent of tested target exons," said Li."Importantly, it is capable of simultaneously activating and repressing different exons to examine their combined functions."
"We have developed a versatile engineered splicing factor that outperforms other available tools in the targeted control of alternative exons," said Taipale, also principal investigator on the study, Canada Research Chair in Functional Proteomics and Proteostasis, Anne and Max Tanenbaum Chair in Molecular Medicine and associate professor of molecular genetics at the Donnelly Centre and Temerty Medicine.
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