That new method is CRISPR, uses RNA-guided enzymes to snip out or add segments of DNA to a cell. In the first time it has been applied to Huntingtons disease, says neuroscientist Nicole Déglon of the University of Lausanne in Switzerland, who led the mouse study, results of which she and her co-researcher Nicolas Merienne shared yesterday at the Society for Neuroscience Conference in Chicago, Illinois. As neurological diseases go, Huntingtons is an ideal candidate for CRISPR therapy, because the disease is determined by a single gene, Déglon notes. A mutation in the gene, which codes for a normally helpful brain protein called huntingtin, consists of different numbers of repeats,” repeating segments of DNA that cause the protein “tandem to fold into a shape that is toxic to the brain. Déglon and her team wondered whether CRISPR could halt production of this dangerous molecule. Using a virus as a delivery vehicle, the researchers infected two separate groups of healthy adult mice with a mutant huntingtin gene, but only one group received the therapy: a CRISPR which includes “cassette,” DNA for the gene-editing enzyme Cas9 and the RNA to target the huntingtin gene. CRISPR-Cas9 works by the part of the “silencing” huntingtin gene that signals protein production. Researchers hypothesized that by cutting into these so-called sites,” they “start would be able to permanently pause synthesis of the huntingtin protein. If this were true, the mice treated with the CRISPR cassette would have little to no buildup of mutated proteins, and that’s exactly what Déglon and her team saw. After only 3 weeks, the two groups of mice showed a striking contrast: Those without the CRISPR treatment had large areas of protein aggregation, and those with the treatment had almost editing had prevented nearly 90% of the rogue proteins. reached about 90% [blockage of production] changes the story “Having [of Huntingtons therapy] completely,” she says. opens new treatment “It strategies that are based in DNA, and so would have a permanent benefit for the rest of someone’s life.” Abdellatif Benraiss, a translational neuroscientist at the University of Rochester in New York who is not involved with the research, cautions that the current technique is not poised for that kind of longevity. Because CRISPR cannot yet discriminate between mutant and healthy
That new method is CRISPR, uses RNA-guided enzymes to snip out or add segments of DNA to a cell. In the first time it has been applied to Huntingtons disease, says neuroscientist Nicole Déglon of the University of Lausanne in Switzerland, who led the mouse study, results of which she and her co-researcher Nicolas Merienne shared yesterday at the Society for Neuroscience Conference in Chicago, Illinois. As neurological diseases go, Huntingtons is an ideal candidate for CRISPR therapy, because the disease is determined by a single gene, Déglon notes. A mutation in the gene, which codes for a normally helpful brain protein called huntingtin, consists of different numbers of “tandem repeats,” repeating segments of DNA that cause the protein to fold into a shape that is toxic to the brain. Déglon and her team wondered whether CRISPR could halt production of this dangerous molecule. Using a virus as a delivery vehicle, the researchers infected two separate groups of healthy adult mice with a mutant huntingtin gene, but only one group received the therapy: a CRISPR “cassette,” which includes DNA for the gene-editing enzyme Cas9 and the RNA to target the huntingtin gene. CRISPR-Cas9 works by “silencing” the part of the huntingtin gene that signals protein production. Researchers hypothesized that by cutting into these so-called “start sites,” they would be able to permanently pause synthesis of the huntingtin protein. If this were true, the mice treated with the CRISPR cassette would have little to no buildup of mutated proteins, and that’s exactly what Déglon and her team saw. After only 3 weeks, the two groups of mice showed a striking contrast: Those without the CRISPR treatment had large areas of protein aggregation, and those with the treatment had almost editing had prevented nearly 90% of the rogue proteins. “Having reached about 90% [blockage of production] changes the story [of Huntingtons therapy] completely,” she says. “It opens new treatment strategies that are based in DNA, and so would have a permanent benefit for the rest of someone’s life.” Abdellatif Benraiss, a translational neuroscientist at the University of Rochester in New York who is not involved with the research, cautions that the current technique is not poised for that kind of longevity. Because CRISPR cannot yet discriminate between mutant and healthy
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