Tool Could Modify Genes to Treat Cancer, Autism

A new technique could allow scientists to edit and repair the human genome, and perhaps one day treat diseases or conditions linked to our genes.

Researchers have figured out how to use the gene-editing tool known as CRISPR-Cas9, on the messenger RNA that carries genetic instructions from the cell’s nucleus to make new proteins.

The experiment, by scientists at the University of California, San Diego, could lead to new treatments for diseases caused by malfunctions in the messenger RNA, such as fragile X syndrome, some types of cancer, autism and neurodegenerative diseases.

“Now we have the ability to modify RNA in live cells,” said Gene Yeo, associate professor of molecular medicine at UCSD and a senior author on the study published today in the journal Cell.

Yeo’s team figured out a new way of targeting and tracking RNA as it moves through human cells.

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