Matches in SemOpenAlex for { <https://semopenalex.org/work/W2751393297> ?p ?o ?g. }
Showing items 1 to 83 of
83
with 100 items per page.
- W2751393297 endingPage "S229" @default.
- W2751393297 startingPage "S229" @default.
- W2751393297 abstract "Dominant negative mutations within KRT14 are responsible for epidermolysis bullosa simplex (EBS). They lead to loss of stability of the keratin intermediate filament cytoskeleton in keratinocytes in the basal layer of the epidermis and as a result to blister formation. To correct KRT14 mutations, spliceosome-mediated RNA trans-splicing (SMaRT) technology was utilized in EBS patient-derived keratinocytes. SMaRT recombines the mutant RNA molecule with an engineered RNA repair molecule (RNA trans-splicing molecule, RTM) to create a corrected gene product. To achieve this, RTMs carry the wild type sequence of the target site and the necessary core splicing signals to facilitate the trans-splicing reaction. In order to increase the repair efficiency of a previously developed KRT14-specific RTM, randomly fragmented antisense RNAs (asRNAs) and rationally designed antisense oligonucleotides (ASOs) were created to block possibly interfering splice elements in the target region from exon 5 to intron 7 ofthe KRT14 pre-mRNA. We analyzed the functionality of 76 asRNAs and 9 ASOs via a previously established fluorescence-based split-GFP screening system in triple-transfection experiments in HEK293 cells and achieved an increase in trans-splicing efficiency of up to 7-fold as monitored by flow cytometry and confirmed by sqRT-PCR analysis. Western Blot analysis of patient cell lines transfected with the most efficient ASOs showed normalization of K14 expression levels. Our results suggest that inclusion of asRNAs or ASOs can increase trans-splicing efficiency to levels that may be sufficient to overcome the EBS phenotype." @default.
- W2751393297 created "2017-09-15" @default.
- W2751393297 creator A5001554153 @default.
- W2751393297 creator A5001618054 @default.
- W2751393297 creator A5007833937 @default.
- W2751393297 creator A5019955434 @default.
- W2751393297 creator A5028047744 @default.
- W2751393297 creator A5036230131 @default.
- W2751393297 creator A5041951523 @default.
- W2751393297 creator A5047649623 @default.
- W2751393297 creator A5070000759 @default.
- W2751393297 date "2017-10-01" @default.
- W2751393297 modified "2023-10-17" @default.
- W2751393297 title "212 Antisense RNA-mediated improvement of SMaRT therapy for KRT14 correction" @default.
- W2751393297 doi "https://doi.org/10.1016/j.jid.2017.07.209" @default.
- W2751393297 hasPublicationYear "2017" @default.
- W2751393297 type Work @default.
- W2751393297 sameAs 2751393297 @default.
- W2751393297 citedByCount "0" @default.
- W2751393297 crossrefType "journal-article" @default.
- W2751393297 hasAuthorship W2751393297A5001554153 @default.
- W2751393297 hasAuthorship W2751393297A5001618054 @default.
- W2751393297 hasAuthorship W2751393297A5007833937 @default.
- W2751393297 hasAuthorship W2751393297A5019955434 @default.
- W2751393297 hasAuthorship W2751393297A5028047744 @default.
- W2751393297 hasAuthorship W2751393297A5036230131 @default.
- W2751393297 hasAuthorship W2751393297A5041951523 @default.
- W2751393297 hasAuthorship W2751393297A5047649623 @default.
- W2751393297 hasAuthorship W2751393297A5070000759 @default.
- W2751393297 hasBestOaLocation W27513932971 @default.
- W2751393297 hasConcept C104317684 @default.
- W2751393297 hasConcept C129312508 @default.
- W2751393297 hasConcept C143065580 @default.
- W2751393297 hasConcept C153911025 @default.
- W2751393297 hasConcept C185592680 @default.
- W2751393297 hasConcept C2777074287 @default.
- W2751393297 hasConcept C2778405274 @default.
- W2751393297 hasConcept C36823959 @default.
- W2751393297 hasConcept C54009773 @default.
- W2751393297 hasConcept C54355233 @default.
- W2751393297 hasConcept C54458228 @default.
- W2751393297 hasConcept C67705224 @default.
- W2751393297 hasConcept C81885089 @default.
- W2751393297 hasConcept C86803240 @default.
- W2751393297 hasConcept C94671646 @default.
- W2751393297 hasConcept C95444343 @default.
- W2751393297 hasConceptScore W2751393297C104317684 @default.
- W2751393297 hasConceptScore W2751393297C129312508 @default.
- W2751393297 hasConceptScore W2751393297C143065580 @default.
- W2751393297 hasConceptScore W2751393297C153911025 @default.
- W2751393297 hasConceptScore W2751393297C185592680 @default.
- W2751393297 hasConceptScore W2751393297C2777074287 @default.
- W2751393297 hasConceptScore W2751393297C2778405274 @default.
- W2751393297 hasConceptScore W2751393297C36823959 @default.
- W2751393297 hasConceptScore W2751393297C54009773 @default.
- W2751393297 hasConceptScore W2751393297C54355233 @default.
- W2751393297 hasConceptScore W2751393297C54458228 @default.
- W2751393297 hasConceptScore W2751393297C67705224 @default.
- W2751393297 hasConceptScore W2751393297C81885089 @default.
- W2751393297 hasConceptScore W2751393297C86803240 @default.
- W2751393297 hasConceptScore W2751393297C94671646 @default.
- W2751393297 hasConceptScore W2751393297C95444343 @default.
- W2751393297 hasIssue "10" @default.
- W2751393297 hasLocation W27513932971 @default.
- W2751393297 hasOpenAccess W2751393297 @default.
- W2751393297 hasPrimaryLocation W27513932971 @default.
- W2751393297 hasRelatedWork W1905789932 @default.
- W2751393297 hasRelatedWork W1964169827 @default.
- W2751393297 hasRelatedWork W2010971328 @default.
- W2751393297 hasRelatedWork W2026595803 @default.
- W2751393297 hasRelatedWork W2080039255 @default.
- W2751393297 hasRelatedWork W2098032059 @default.
- W2751393297 hasRelatedWork W2110272490 @default.
- W2751393297 hasRelatedWork W2128371536 @default.
- W2751393297 hasRelatedWork W2159095138 @default.
- W2751393297 hasRelatedWork W2342650295 @default.
- W2751393297 hasVolume "137" @default.
- W2751393297 isParatext "false" @default.
- W2751393297 isRetracted "false" @default.
- W2751393297 magId "2751393297" @default.
- W2751393297 workType "article" @default.