Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice

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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

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Medicine, Genetics, Natural Sciences +8

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

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A gene mutated in X–linked myotubular myopathy defines a new putative tyrosine phosphatase family conserved in yeast

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Molecular Physiology, Genetic Disorder, Natural Sciences +10

Sequence-Specific Inhibition of Small RNA Function

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Systems Biology, Developmental Biology, Small Rna Function +15