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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy
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A simplified system for the effective expression and delivery of functional mature microRNAs in mammalian cells
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Inhibition of hyperglycolysis in mesothelial cells prevents peritoneal fibrosis
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Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice
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