1997
Direct Cutaneous Gene Delivery in a Human Genetic Skin Disease
Choate K, Khavari P. Direct Cutaneous Gene Delivery in a Human Genetic Skin Disease. Human Gene Therapy 1997, 8: 1659-1665. PMID: 9322868, DOI: 10.1089/hum.1997.8.14-1659.Peer-Reviewed Original ResearchConceptsLamellar ichthyosisCutaneous gene deliveryHuman genetic skin diseaseFunctional abnormalitiesDeficient patientsTherapeutic goalsNude miceSkin diseasesIntact skinVivo injectionPlasmid injectionSuprabasal epidermisPatient's skinGenetic skin diseasesAccessible somatic tissueDisease tissuesGene delivery approachSkinTherapeutic gene transferInjectionRetroviral expression vectorDiseaseNaked DNAPartial restorationTissue location
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