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posted by cmn32480 on Friday November 10 2017, @07:03AM   Printer-friendly
from the mutations-you-can-grow-out-of dept.

Submitted via IRC for takyon

The Broad Institute and MIT scientists who first harnessed CRISPR for mammalian genome editing have engineered a new molecular system for efficiently editing RNA in human cells. RNA editing, which can alter gene products without making changes to the genome, has profound potential as a tool for both research and disease treatment.

In a paper published today [October, 25, 2017] in Science, senior author Feng Zhang and his team describe the new CRISPR-based system, called RNA Editing for Programmable A to I Replacement, or "REPAIR." The system can change single RNA nucleotides in mammalian cells in a programmable and precise fashion. REPAIR has the ability to reverse disease-causing mutations at the RNA level, as well as other potential therapeutic and basic science applications.

"The ability to correct disease-causing mutations is one of the primary goals of genome editing," says Zhang, a core institute member of the Broad Institute, an investigator at the McGovern Institute, and the James and Patricia Poitras '63 Professor in Neuroscience and associate professor in the departments of Brain and Cognitive Sciences and Biological Engineering at MIT. "So far, we've gotten very good at inactivating genes, but actually recovering lost protein function is much more challenging. This new ability to edit RNA opens up more potential opportunities to recover that function and treat many diseases, in almost any kind of cell."

REPAIR has the ability to target individual RNA letters, or nucleosides, switching adenosines to inosines (read as guanosines by the cell). These letters are involved in single-base changes known to regularly cause disease in humans. In human disease, a mutation from G to A is extremely common; these alterations have been implicated in, for example, cases of focal epilepsy, Duchenne muscular dystrophy, and Parkinson's disease. REPAIR has the ability to reverse the impact of any pathogenic G-to-A mutation regardless of its surrounding nucleotide sequence, with the potential to operate in any cell type.

Unlike the permanent changes to the genome required for DNA editing, RNA editing offers a safer, more flexible way to make corrections in the cell. "REPAIR can fix mutations without tampering with the genome, and because RNA naturally degrades, it's a potentially reversible fix," explains co-first author David Cox, a graduate student in Zhang's lab.

Source: http://news.mit.edu/2017/researchers-engineer-crispr-edit-single-rna-letters-human-cells-1015


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  • (Score: 1, Offtopic) by c0lo on Friday November 10 2017, @08:47AM

    by c0lo (156) Subscriber Badge on Friday November 10 2017, @08:47AM (#595066) Journal

    Until base-pair editing becomes something a low-income "technician" can do for you

    What I am suggesting: there's no incentive for them to reach a point in which a low-income "technician" can do it until the patent expires

    Unless, of course, the Chinese get their own research going and F/O the US patent. It's highly possible/probable because, you see, Feng Zhang sounds as such a traditional American name.

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