2020
Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study
Walters M, Kanter J, Kwiatkowski J, Krishnamurti L, Mapara M, Schmidt M, Miller A, Pierciey F, Bonner M, Huang W, Ribeil J, Thompson A, Tisdale J. Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study. Transplantation And Cellular Therapy 2020, 26: s1-s2. DOI: 10.1016/j.bbmt.2019.12.136.Peer-Reviewed Original ResearchPeripheral blood mononuclear cellsGroup C patientsAdverse eventsC patientsMedian HbTotal HbPlerixafor mobilizationNon-hematologic gradeGene therapySerious adverse eventsBlood mononuclear cellsLentiviral vectorsStrong therapeutic benefitFebrile neutropeniaSevere SCDConclusions PatientsGraft failureInitial patientsLast visitMononuclear cellsBusulfan conditioningHemolysis markersAutologous CD34Colony-forming unit assaysTherapeutic benefit
2018
188 Single-Agent Plerixafor Mobilization to Collect Autologous Stem Cells for Use in Gene Therapy for Severe Sickle Cell Disease
Tisdale J, Kanter J, Hsieh M, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Miller A, Pierciey F, Shi W, Asmal M, Thompson A, Walters M. 188 Single-Agent Plerixafor Mobilization to Collect Autologous Stem Cells for Use in Gene Therapy for Severe Sickle Cell Disease. Transplantation And Cellular Therapy 2018, 24: s174. DOI: 10.1016/j.bbmt.2017.12.108.Peer-Reviewed Original Research