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RNA could be used for safer and more effective gene therapies, Chinese study finds

  • Technology has ‘significant advantages in terms of safety and deliverability’, according to study author

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The Chinese team used RNA, or ribonucleic acid, to precisely gene-edit mammalian cells. Photo: Getty Images/Science Photo Library RF
Victoria Bela

Chinese scientists have successfully used RNA to precisely gene-edit mammalian cells – something that could be used to develop safer, more effective and lower-cost gene therapies.

RNA, or ribonucleic acid, is one of three macromolecules along with DNA and proteins that are key to many processes in the body.

“We realised all-RNA-based, targeted, full-length functional gene integration in mammalian cells by developing new gene integration tools from naturally occurring [genetic elements known as] retrotransposons,” the researchers from the Chinese Academy of Sciences wrote in a paper published in the peer-reviewed journal Cell on July 8.

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Gene integration is a targeted gene-editing method in which an entire gene of interest is inserted into a target genome, allowing cells to generate a desired protein. It can be used to treat diseases in which production of a protein is impaired.

The development of CRISPR-based gene-editing systems has pushed forward technology but finding a way to efficiently integrate large DNA fragments on a gene scale into genomes “remains a challenge”, study author Li Wei told state-run Science and Technology Daily.

Most gene-editing systems rely on DNA donors – such as plasmids or viral vectors – to carry a gene of interest into a cell so that it can be integrated into the host genome.

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