2009
First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder
Leachman SA, Hickerson RP, Schwartz ME, Bullough EE, Hutcherson SL, Boucher KM, Hansen CD, Eliason MJ, Srivatsa GS, Kornbrust DJ, Smith FJ, McLean WI, Milstone LM, Kaspar RL. First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder. Molecular Therapy 2009, 18: 442-446. PMID: 19935778, PMCID: PMC2839285, DOI: 10.1038/mt.2009.273.Peer-Reviewed Original ResearchConceptsPachyonychia congenitaPhase Ib clinical trialSubjective patient assessmentPhase Ib trialDose-escalation trialClinical efficacy measuresAutosomal dominant syndromeIb trialAdverse eventsWashout periodPatient assessmentEfficacy measuresPlantar callusClinical trialsPlantar keratodermaOpposite footSkin disordersSkin diseasesClinical settingDominant syndromeSatisfactory treatmentAdditional studiesTrialsGenetic disordersSiRNA
2001
Photodegraded Nifedipine Stimulates Uptake and Retention of Iron in Human Epidermal Keratinocytes
Gruen A, Zhou J, Milstone L, Morton K. Photodegraded Nifedipine Stimulates Uptake and Retention of Iron in Human Epidermal Keratinocytes. Journal Of Investigative Dermatology 2001, 116: 774-777. PMID: 11348469, DOI: 10.1046/j.1523-1747.2001.01318.x.Peer-Reviewed Original ResearchConceptsHuman epidermal keratinocytesEpidermal keratinocytesD washout periodNifedipine treatmentDaily treatmentWashout periodNifedipineRetention of ironEpidermal desquamationIron uptakeKeratinocytesKeratinocyte monolayersSerum proteinsRelevant concentrationsToxic amountsEpidermal culturesTreatmentErythroid cells