2008
Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita
Leachman SA, Hickerson RP, Hull PR, Smith FJ, Milstone LM, Lane EB, Bale SJ, Roop DR, McLean WH, Kaspar RL. Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita. Journal Of Dermatological Science 2008, 51: 151-157. PMID: 18495438, PMCID: PMC2587483, DOI: 10.1016/j.jdermsci.2008.04.003.Peer-Reviewed Original ResearchConceptsPachyonychia congenitaQuantitative RT-PCRRT-PCRReal-time RT-PCRQuantitative real-time RT-PCRClinical efficacyGenetic skin disordersClinical trialsCandidate siRNASkin disordersRNA interferenceNovel therapeuticsPotent inhibitionTherapeutic siRNAsClinical applicationDisordersGenetic disordersPhysiciansCongenitaMutant allelesSiRNAHuman diseasesKeratin disordersPatientsDisease
2007
Single-Nucleotide-Specific siRNA Targeting in a Dominant-Negative Skin Model
Hickerson RP, Smith FJ, Reeves RE, Contag CH, Leake D, Leachman SA, Milstone LM, McLean WH, Kaspar RL. Single-Nucleotide-Specific siRNA Targeting in a Dominant-Negative Skin Model. Journal Of Investigative Dermatology 2007, 128: 594-605. PMID: 17914454, DOI: 10.1038/sj.jid.5701060.Peer-Reviewed Original ResearchConceptsWild-type gene expressionBicistronic reporter constructFilament formationDominant-negative genetic disordersNumerous genetic disordersGenetic disordersKeratin filament formationRNA interferenceMutant formsReporter constructsGene expressionMutant mRNAFluorescent reportersMutant allelesCell culture modelSiRNAsSimultaneous expressionK6aK mutationPachyonychia congenitaExpressionCulture modelHuman keratinocytesSiRNASelective inhibition