Article
Allele-specific silencing of dominant disease genes.
Proceedings of the National Academy of Sciences of the United States of America - 10 Jun 2003
Miller Victor M, Xia Haibin, Marrs Ginger L, Gouvion Cynthia M, Lee Gloria, Davidson Beverly L, Paulson Henry L
Abstract excerpt
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 neurodegenerative...
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