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View article: Systems-level regulation of microRNA networks by miR-130/301 promotes pulmonary hypertension
Systems-level regulation of microRNA networks by miR-130/301 promotes pulmonary hypertension Open
The authors recently became aware that in Figure 3D, lung images from a 10% O 2 -exposed mouse were inadvertently presented in the row labeled as lung images from a 10% O 2 + SU5416-exposed mouse.The authors reviewed the original data and …
View article: Targeting 3′ and 5′ untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression
Targeting 3′ and 5′ untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression Open
Friedreich’s ataxia (FRDA) is a severe multisystem disease caused by transcriptional repression induced by expanded GAA repeats located in intron 1 of the Frataxin (FXN) gene encoding frataxin. FRDA results from decreased levels of frataxi…
View article: mRNA therapeutics: beyond vaccine applications
mRNA therapeutics: beyond vaccine applications Open
The basic principle of mRNA therapeutics involves delivery of in vitro transcribed mRNA into a target cell, where cellular machinery translates the mRNA into a functional protein. In vaccine applications, an mRNA encoding a viral protein w…
View article: Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy
Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy Open
Significance Autosomal recessive mutations or deletions of the gene Survival Motor Neuron 1 ( SMN1 ) cause spinal muscular atrophy, a neurodegenerative disorder. Transcriptional up-regulation of a nearly identical gene, SMN2 , can function…
View article: Identification of metabolically stable 5′-phosphate analogs that support single-stranded siRNA activity
Identification of metabolically stable 5′-phosphate analogs that support single-stranded siRNA activity Open
The ss-siRNA activity in vivo requires a metabolically stable 5'-phosphate analog. In this report we used crystal structure of the 5'-phosphate binding pocket of Ago-2 bound with guide strand to design and synthesize ss-siRNAs containing v…