Featured Publications
Combined liver–cytokine humanization comes to the rescue of circulating human red blood cells
Song Y, Shan L, Gbyli R, Liu W, Strowig T, Patel A, Fu X, Wang X, Xu ML, Gao Y, Qin A, Bruscia EM, Tebaldi T, Biancon G, Mamillapalli P, Urbonas D, Eynon E, Gonzalez DG, Chen J, Krause DS, Alderman J, Halene S, Flavell RA. Combined liver–cytokine humanization comes to the rescue of circulating human red blood cells. Science 2021, 371: 1019-1025. PMID: 33674488, PMCID: PMC8292008, DOI: 10.1126/science.abe2485.Peer-Reviewed Original ResearchConceptsRed blood cellsBlood cellsHuman sickle cell diseaseSickle cell diseaseImmunodeficient murine modelKupffer cell densityBone marrow failureMISTRG miceIntrasplenic injectionSCD pathologyCell diseaseMurine modelComplement C3RBC survivalVivo modelHuman cytokinesPreclinical testingHematopoietic stem cellsHuman red blood cellsMarrow failureFumarylacetoacetate hydrolase geneHuman erythropoiesisHuman liverHuman hepatocytesMice
2022
Targeting the EIF2AK1 signaling pathway rescues red blood cell production in SF3B1-mutant myelodysplastic syndromes with ringed sideroblasts
Adema V, Ma F, Kanagal-Shamanna R, Thongon N, Montalban-Bravo G, Yang H, Peslak SA, Wang F, Acha P, Sole F, Lockyer P, Cassari M, Maciejewski JP, Visconte V, Ganan-Gomez I, Song Y, Bueso-Ramos C, Pellegrini M, Tan TM, Bejar R, Carew JS, Halene S, Santini V, Al-Atrash G, Clise-Dwyer K, Garcia-Manero G, Blobel GA, Colla S. Targeting the EIF2AK1 signaling pathway rescues red blood cell production in SF3B1-mutant myelodysplastic syndromes with ringed sideroblasts. Blood Cancer Discovery 2022, 3: 554-567. PMID: 35926182, PMCID: PMC9894566, DOI: 10.1158/2643-3230.bcd-21-0220.Peer-Reviewed Original ResearchConceptsMyelodysplastic syndromeRed blood cell productionSF3B1-mutant myelodysplastic syndromesMDS-RSRinged sideroblastsBlood cell productionSF3B1 mutationsDevelopment of therapiesCell productionRed blood cellsRed blood cell maturationHematologic responseSignificant anemiaTransfusion dependencyIron overloadMDS subtypesElderly populationSide effectsBone marrowCell maturationIssue featurePatientsBlood cellsErythroid precursorsBlood cell maturation
2016
Adding insult to injury: autoimmune haemolytic anaemia complicated by pulmonary embolism
Woodson KA, Lee Y, Gopalratnam K, Halene S. Adding insult to injury: autoimmune haemolytic anaemia complicated by pulmonary embolism. BMJ Case Reports 2016, 2016: bcr2015213575. PMID: 26768839, PMCID: PMC4735441, DOI: 10.1136/bcr-2015-213575.Peer-Reviewed Case Reports and Technical NotesConceptsAutoimmune haemolytic anaemiaOwn red blood cellsPulmonary embolismPulmonary hypertensionVenous thromboembolismHaemolytic anaemiaPatient's own red blood cellsSevere right ventricular dysfunctionNitric oxideRight ventricular dysfunctionNitric oxide treatmentClot burdenHaemodynamic compromiseVentricular dysfunctionRed blood cellsDeadly complicationPathological antibodiesChronic mannerAnemiaBlood cellsHypertensionEmbolismPossible benefitsTreatmentOxide treatment