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Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
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Incorporation of bridged nucleic acids into CRISPR RNAs improves Cas9 endonuclease specificity
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RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors
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Simultaneous Monitoring of Cell-surface Receptor and Tumor-targeted Photodynamic Therapy via TdT-initiated Poly-G-Quadruplexes
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