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Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.
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RNA Therapeutics Are Stepping Out of the Maze.
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RNA Splicing and Disease: Animal Models to Therapies.
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Wang, Dan
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Target selectivity in mRNA silencing.
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Small RNA combination therapy for lung cancer.
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Khvorova, Anastasia
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Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression.
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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
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An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu,Zn superoxide dismutase.
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Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis.
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Zamore, Phillip
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RNA therapeutics on the rise.
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Trinucleotide repeat expansion and RNA dysregulation in fragile X syndrome: emerging therapeutic approaches.
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RNA interference therapeutics in organ transplantation: The dawn of a new era.
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