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
1996
Corrective gene transfer in the human skin disorder lamellar ichthyosis
Choate K, Medalie D, Morgan J, Khavari P. Corrective gene transfer in the human skin disorder lamellar ichthyosis. Nature Medicine 1996, 2: 1263-1267. PMID: 8898758, DOI: 10.1038/nm1196-1263.Peer-Reviewed Original ResearchConceptsLamellar ichthyosisImmunodeficient mouse xenograft modelPrimary pathophysiologic defectMouse xenograft modelAbnormal epidermal differentiationCorrective gene transferCutaneous gene deliveryPathophysiologic defectsLI patientsEpidermal differentiationFunctional abnormalitiesPotential future approachesImmunodeficient miceXenograft modelSkin diseasesCutaneous barrierDifferentiation marker filaggrinFunction measuresNormal skinEpidermal barrierPatient's skinPrimary keratinocytesPatientsSkinHuman skin