Allele-specific silencing of dominant disease genes

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Allele-specific silencing of dominant disease genes.

Small interfering RNA (siRNA) holds therapeutic promise for silencing dominantly acting disease genes, particularly if mutant alleles can be targeted selectively. In mammalian cell models we demonstrate that allele-specific silencing of disease genes with siRNA can be achieved by targeting either a linked single-nucleotide polymorphism (SNP) or the disease mutation directly. For a polyglutamine...

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ژورنال

عنوان ژورنال: Proceedings of the National Academy of Sciences

سال: 2003

ISSN: 0027-8424,1091-6490

DOI: 10.1073/pnas.1231012100