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Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington's disease - Nature Biomedical Engineering

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Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington's disease - Nature Biomedical Engineering

The monogenic nature of Huntington's disease (HD) and other neurodegenerative diseases caused by the expansion of glutamine-encoding CAG repeats makes them particularly amenable to gene therapy. Here we show the feasibility of replacing expanded CAG repeats in the mutant HTT allele with a normal CAG repeat in genetically engineered pigs mimicking the selective neurodegeneration seen in patients with HD.

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Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington's disease - Nature Biomedical Engineering

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Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington's disease - Nature Biomedical Engineering

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