CasRx (magenta) targeting RNA in the nucleus of human cells (gray). Credit: Salk Institute
Most people have heard of the CRISPR/Cas9 gene-editing technology, which acts as targeted molecular scissors to cut and replace disease-causing genes with healthy ones. But DNA is only part of the story; many genetic diseases are caused by problems with RNA, a working copy of DNA that is translated into proteins. Now, Salk Institute scientists have created a new tool that targets not DNA, but RNA, and used it to correct a protein imbalance in cells from a dementia patient, restoring them to healthy levels...
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