2021
Novel RNA Interference (RNAi)-Based Nanomedicines for Treating Viral Infections
Maes N, Zeller S, Kumar P. Novel RNA Interference (RNAi)-Based Nanomedicines for Treating Viral Infections. 2021, 223-264. DOI: 10.1201/9781003125259-8.Peer-Reviewed Original ResearchJapanese encephalitis virusNon-target tissuesHuman papillomavirusPediatric encephalitisTherapeutic modalitiesMajor safety concernNovel RNA interferenceViral infectionReplication-competent lentivirusClinical testingEncephalitis virusInfectious diseasesMajor causeSafety concernsDiseaseVector mobilizationRibonucleic acid interferenceTarget effectsRNA interferenceTargets of microRNAsEncephalitisPapillomavirusVaccineInfectionFlaviviruses
2016
Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease
Kanter J, Walters M, Hsieh M, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Kuypers F, Cavazzana M, Leboulch P, Joseney-Antoine M, Asmal M, Thompson A, Tisdale J. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease. Blood 2016, 128: 1176. DOI: 10.1182/blood.v128.22.1176.1176.Peer-Reviewed Original ResearchSevere sickle cell diseaseSevere SCDBone marrow harvestAdverse eventsSickle cell diseaseCell doseBluebird BioReplication-competent lentivirusHematopoietic stem cellsMyeloablative conditioningPeripheral bloodCell diseaseGrade 3 adverse eventsAdvisory CommitteeMedical directorsPhase 1/2 clinical studyTransfusion-dependent β-thalassemiaSerious adverse eventsLong-term complicationsVector copy numberStart of conditioningSymptoms 1 yearIntegration site analysisGene therapyDP infusion
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