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Transthyretin allele selective UNA oligomers for gene silencing
Transthyretin allele selective UNA oligomers for gene silencing
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机译:运甲状腺素蛋白等位基因选择性UNA寡聚体基因沉默
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摘要
This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject.
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