CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression.

Nucleic Acids Res · 2017 · PMC5435994 · PMID 28334749

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highly significantno p-value reported

Conversely, CAG-13PS produced a marked and highly significant down-regulation of both mRNA and protein, indicating the possibility to target DNA with rather short ONs and still obtain an effect.

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