2021
Hematopoietic cell transplantation for sickle cell disease: updates and future directions
Krishnamurti L. Hematopoietic cell transplantation for sickle cell disease: updates and future directions. Hematology 2021, 2021: 181-189. PMID: 34889368, PMCID: PMC8791142, DOI: 10.1182/hematology.2021000251.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseCell transplantationAlternative donorsCell diseaseOutcomes of HCTHLA-identical related donorsSymptomatic sickle cell diseaseNew disease-modifying drugsHost disease (GVHD) prophylaxisHLA-identical siblingsAvailable therapeutic optionsDisease-modifying drugsLong-term outcomesCord blood expansionConditioning regimensDisease prophylaxisRelated donorsOrgan damageHematopoietic progenitor cellsTherapeutic optionsExcellent outcomesCareful counselingAutologous transplantationClinical registry
2015
Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease
Kanter J, Walters M, Hsieh M, Thompson A, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Kuypers F, Cavazzana M, Leboulch P, Sandler L, Soni S, Tisdale J. Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease. Blood 2015, 126: 3233. DOI: 10.1182/blood.v126.23.3233.3233.Peer-Reviewed Original ResearchSevere SCDSevere sickle cell diseaseSickle cell diseaseCell diseaseBluebird BioAdverse eventsAutologous CD34Additional subjectsLentiviral vectorsBone marrow harvestVector copy numberIntegration site analysisAdvisory CommitteeGene therapyHematologic engraftmentPRBC transfusionIntravenous busulfanChronic transfusionHb levelsSafety profileAutologous transplantationCell doseStudy visitMonth postProduct infusion