A novel CRISPR–Cas9-based gene editing approach for Huntington disease (HD) can inactivate the HD-associated mutant HTT allele without affecting the normal allele. The technique prevented expression of the mutated huntingtin protein in several cell lines.
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A novel CRISPR–Cas9-based gene editing approach for Huntington disease (HD) can inactivate the HD-associated mutant HTT allele without affecting the normal allele. The technique prevented expression of the mutated huntingtin protein in several cell lines.