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Small-Molecule RNA-Targeting Technology Kills Cancer Cells

05:35 EDT 30 May 2018 | Genetic Engineering News

Scientists at The Scripps Research Institute have developed a small molecule-based RNA editing tool that can selectively delete RNA targets associated with diseases such as cancer. Offering a potential alternative to CRISPR-Cas9 -based tools that edit DNA, the new technology, developed by Matthew D. Disney, Ph.D., attaches an RNA-targeting drug-like molecule to an RNA-degrading enzyme.Because the technology uses small molecules rather than proteins, it could also feasibly be packaged into a convenient, oral pill. Initial tests demonstrated that a ribonuclease-targeting chimera (RIBOTAC) designed against a breast cancer microRNA oncogene caused the death of malignant cells, without harming normal cells. "I believe this is just the tip of the iceberg of how this approach will ultimately be applied," says Disney. “Since it is now known that RNA is a key driver in nearly every disease, optimization of this approach that turns a cell's natural defenses toward destroying disease-causing RNAs ...

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