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Tool developed to silence genes in specific tissues using RNAi

Researchers at The University of Texas M. D. Anderson Cancer Center say they have jumped a significant hurdle in the use of RNA interference (RNAi), believed by many to be the ultimate tool to both decode the function of individual genes in the human genome and to treat disease.

Reporting in the journal Genes and Development, investigators have developed a simple way to use the RNAi approach to silence a selected gene in a specific tissue in a mouse to determine the function of that targeted gene.

Previously reported approaches to achieve this were either technically cumbersome, not generally applicable, or only achieved transient knockdown of the target gene.

"Having a tool that will allow us to knockdown the expression of any given gene in any specific tissue or cell type represents a significant advance in the field," says the study's lead investigator, Miles Wilkinson, Ph.D., a professor in the Department of Immunology.

For example, this method could potentially be used in humans "to knockdown the expression of mutant or overexpressed genes that cause human diseases, including cancer," Wilkinson says. "Scientists and clinicians can use it to reduce the expression of the target gene in a single or limited numbers of cell types or tissues, thereby reducing side effects."

Equally, the technique can help researchers determine the function of a single gene in a single tissue, which "is potentially a powerful investigative tool," Wilkinson says. "By silencing a particular gene in a specific tissue, you can learn what the function of that gene is in that particular tissue without blocking its essential functions in other tissues."

In their study, Wilkinson and his research team demonstrated how well their tool worked by silencing the WT1 tumor suppressor gene in the testes of mice. They found this gene is important in the production of healthy sperm by encoding a regulatory protein called a transcription factor that contr
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Source:University of Texas M. D. Anderson Cancer Center


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