Artificial mirtron-mediated gene knockdown: Functional DMPK silencing in mammalian cells

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Artificial mirtron-mediated gene knockdown: functional DMPK silencing in mammalian cells.

Mirtrons are introns that form pre-miRNA hairpins after splicing to produce RNA interference (RNAi) effectors distinct from Drosha-dependent intronic miRNAs. Here we present a design algorithm for artificial mirtrons and demonstrate, for the first time, efficient gene knockdown of myotonic dystrophy protein kinase (DMPK) target sequences in Renilla luciferase 3' UTR and subsequently pathogenic ...

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ژورنال

عنوان ژورنال: RNA

سال: 2012

ISSN: 1355-8382

DOI: 10.1261/rna.030601.111