Gene Therapy · 2010 · 157 citations · 52 references
Life SciencesMolecular BiologyMolecular GeneticsGene DeliveryGene ExpressionGene TherapyLiver DiseaseMicrorna BiologyMicrorna-mediated RegulationViral Gene ExpressionViral Vector-mediated TransgenesisMicrorna TargetingMicrorna TherapyMicrorna-mediated Gene SilencingMicrorna-target InteractionTransgene Expression
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Identification of Novel Genes Coding for Small Expressed RNAs
Mariana Lagos‐Quintana, Reinhard Rauhut, Winfried Lendeckel et al. · Science · 2001 · 4.9K citations · Full text
Mammalian microRNAs predominantly act to decrease target mRNA levels
Huili Guo, Nicholas T. Ingolia, Jonathan S. Weissman et al. · Nature · 2010 · 3.9K citations · Full text
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text
Medicine, Genetics, Ophthalmology +17