2023
Lipid Nanoparticle-Mediated Hit-and-Run Approaches Yield Efficient and Safe In Situ Gene Editing in Human Skin
Bolsoni J, Liu D, Mohabatpour F, Ebner R, Sadhnani G, Tafech B, Leung J, Shanta S, An K, Morin T, Chen Y, Arguello A, Choate K, Jan E, Ross C, Brambilla D, Witzigmann D, Kulkarni J, Cullis P, Hedtrich S. Lipid Nanoparticle-Mediated Hit-and-Run Approaches Yield Efficient and Safe In Situ Gene Editing in Human Skin. ACS Nano 2023, 17: 22046-22059. PMID: 37918441, PMCID: PMC10655174, DOI: 10.1021/acsnano.3c08644.Peer-Reviewed Original ResearchConceptsLipid nanoparticlesLNP compositionGene editing ratesGene editingGene editing toolsIonizable lipidsEfficient deliveryDelivery barriersCas9 RNPMonogenic skin diseasesOff-target effectsNanoparticlesEditing ratesEditing toolsCas9 mRNAGenetic toolsHuman skinEditingHigh 2DSkin tissueExciting advancesEfficientLayerDeliveryEpidermal layer
1998
High-Efficiency Gene Transfer and Pharmacologic Selection of Genetically Engineered Human Keratinocytes
Deng H, Choate K, Lin Q, Khavari P. High-Efficiency Gene Transfer and Pharmacologic Selection of Genetically Engineered Human Keratinocytes. BioTechniques 1998, 25: 274-280. PMID: 9714888, DOI: 10.2144/98252gt02.Peer-Reviewed Original ResearchConceptsCutaneous gene deliveryGene delivery efficiencyEfficient retroviral gene transferGene transferFuture genetic therapiesTherapeutic genesGene deliveryDelivery efficiencyRetroviral gene transferRetroviral vectorsGenetic therapiesLow efficiencyProduction of populationsSomatic cellsEfficiencyHuman keratinocytesArrayGeneticallyBlasticidinTransferDeliveryEpidermolysis bullosaDelivery effortsCapabilityProduction
1997
Sustainability of Keratinocyte Gene Transfer and Cell Survival In Vivo
Choate K, Khavari P. Sustainability of Keratinocyte Gene Transfer and Cell Survival In Vivo. Human Gene Therapy 1997, 8: 895-901. PMID: 9195211, DOI: 10.1089/hum.1997.8.8-895.Peer-Reviewed Original ResearchConceptsGene deliveryCutaneous gene deliveryTherapeutic gene deliveryRetroviral vectorsNormal gene expression levelsBeta-gal expressionMultilayer epidermisGene transferRetroviral long terminal repeatEpidermal progenitor cellsSpecies-specific antibodiesVector sequencesCellular promotersKeratinocyte transglutaminaseDeliveryMarker gene expressionAttractive sitesGene productionType VII collagenHuman keratinocytesEfficiencyHuman involucrinTerminal repeat