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
Selective inhibition of MCL1 overcomes venetoclax resistance in a murine model of myelodysplastic syndromes
Fischer MA, Song Y, Arrate MP, Gbyli R, Villaume MT, Smith BN, Childress MA, Stricker TP, Halene S, Savona MR. Selective inhibition of MCL1 overcomes venetoclax resistance in a murine model of myelodysplastic syndromes. Haematologica 2022, 108: 522-531. PMID: 35979721, PMCID: PMC9890032, DOI: 10.3324/haematol.2022.280631.Peer-Reviewed Original ResearchConceptsB-cell lymphoma 2Acute myeloid leukemiaMyeloid cell leukemia-1Myelodysplastic syndromeMDS subtypesHigh-risk myelodysplastic syndromeMCL1 inhibitionRisk myelodysplastic syndromesAnti-apoptotic protein B-cell lymphoma 2Protein B-cell lymphoma 2Effective clinical therapySelective inhibitorMDS patient samplesAttractive therapeutic opportunityBcl-xLExcess blastsOlder patientsClinical trialsMyeloid leukemiaMurine modelImpressive responseSignificant injuryAnti-apoptotic protein Bcl-xLLeukemia survivalLymphoma 2The Coming of Age of Preclinical Models of MDS
Liu W, Teodorescu P, Halene S, Ghiaur G. The Coming of Age of Preclinical Models of MDS. Frontiers In Oncology 2022, 12: 815037. PMID: 35372085, PMCID: PMC8966105, DOI: 10.3389/fonc.2022.815037.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsTranscription factorsEpigenetic modifiersComplex molecular landscapeXenograft modelHematopoietic cellsDeletions/additionsMolecular landscapeRecurrent mutationsCell linesGenetic abnormalitiesMDS cellsXenograft murine modelChromosomal abnormalitiesCellsIneffective hematopoiesisTransgenicHematopoiesisMutationsPreclinical modelsWide spectrumMurine modelHeterogeneous groupLandscapeComing of age
1999
Immune response to green fluorescent protein: implications for gene therapy
Stripecke R, del Carmen Villacres M, Skelton D, Satake N, Halene S, Kohn D. Immune response to green fluorescent protein: implications for gene therapy. Gene Therapy 1999, 6: 1305-1312. PMID: 10455440, DOI: 10.1038/sj.gt.3300951.Peer-Reviewed Original ResearchConceptsCytotoxic T lymphocytesImmune responseDevelopment of CTLImmunodeficient Nu/Nu miceT cell immune responsesNu/nu miceAnti-leukemia responseTransplantable murine modelCell immune responsesT-cell lymphomaLeukemia cell vaccinesCo-express markersMajor histocompatibility complexCell vaccineDendritic cellsLeukemia vaccineImmunocompetent miceLeukemia challengeNu miceT lymphocytesImmune stimulationCell lymphomaMurine modelGene-modified cellsFlow cytometry