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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...

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Autors principals: Miller, Victor M., Xia, Haibin, Marrs, Ginger L., Gouvion, Cynthia M., Lee, Gloria, Davidson, Beverly L., Paulson, Henry L.
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2003
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC165852/
https://ncbi.nlm.nih.gov/pubmed/12782788
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1231012100
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