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Expanded CAG repeats in the crosshairs

Antisense oligomers targeted to CAG repeats allow allele-specific knockdown of the gene that causes Huntington's disease.

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Figure 1: Two nucleic acid-based strategies for allele-specific silencing of mutant genes containing expanded triplet repeats.


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Aronin, N. Expanded CAG repeats in the crosshairs. Nat Biotechnol 27, 451–452 (2009).

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