2020
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Walters M, Locatelli F, Thrasher A, Tisdale J, Orchard P, Duncan C, Kühl J, De Oliveira S, Sauer M, Kulozik A, Yannaki E, Hongeng S, Mapara M, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith N, Shi W, Colvin R, McNeil E, Ribeil J, Cavazzana M, Williams D. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy. Transplantation And Cellular Therapy 2020, 26: s38-s39. DOI: 10.1016/j.bbmt.2019.12.104.Peer-Reviewed Original ResearchTransfusion-dependent β-thalassemiaSickle cell diseaseHematopoietic stem cell transplantationCerebral adrenoleukodystrophyStem cell transplantationAdverse eventsAllo-HSCTSafety profileCell transplantationCell diseaseAllogeneic hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantationGene addition therapyLentiviral vectorsBusulfan/cyclophosphamideCommon adverse eventsSecondary graft failureRisk of GVHDLong-term followRisk of complicationsΒ-thalassemiaImproved safety profileDP infusionFebrile neutropeniaAutologous HSCT
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