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
2020
The Role of RNA Epigenetic Modification in Normal and Malignant Hematopoiesis
Vasic R, Gao Y, Liu C, Halene S. The Role of RNA Epigenetic Modification in Normal and Malignant Hematopoiesis. Current Stem Cell Reports 2020, 6: 144-155. PMID: 33777659, PMCID: PMC7992056, DOI: 10.1007/s40778-020-00178-y.Commentaries, Editorials and LettersHematopoietic stem cellsM6A machineryEpigenetic modificationsRNA modificationsStem cellsNormal hematopoietic stem cellsStem cell fate decisionsCell fate decisionsStem cell functionRNA epigenetic modificationRNA m6A modificationRecognition of RNAImmune cell developmentM6A RNA modificationEraser proteinsFate decisionsM6A readersVariety of functionsGene expressionMalignant hematopoiesisImmune signalingImmune system functionCritical regulatorCell developmentM6A modificationHumanized mice as preclinical models for myeloid malignancies
Gbyli R, Song Y, Halene S. Humanized mice as preclinical models for myeloid malignancies. Biochemical Pharmacology 2020, 174: 113794. PMID: 31926939, DOI: 10.1016/j.bcp.2020.113794.Books
2019
Functional Analysis of Human Hematopoietic Stem Cells In Vivo in Humanized Mice
Song Y, Gbyli R, Fu X, Halene S. Functional Analysis of Human Hematopoietic Stem Cells In Vivo in Humanized Mice. Methods In Molecular Biology 2019, 2097: 273-289. PMID: 31776933, DOI: 10.1007/978-1-0716-0203-4_18.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHematopoietic stem cellsHematopoietic stem cell functionFunctional hematopoietic stem cellsStem cell functionStem cellsCell-intrinsic functionHuman hematopoietic stem cellsOrganismal contextFunctional analysisCell functionIntrinsic functionCell sourceCellular productsChimeric miceStem cell therapyCellsReprogrammingMouse modelMajor advancesCell therapyMiceVivoFunction
2018
Role of alternative splicing in hematopoietic stem cells during development
Gao Y, Vasic R, Halene S. Role of alternative splicing in hematopoietic stem cells during development. Stem Cell Investigation 2018, 5: 26-26. PMID: 30221171, PMCID: PMC6131231, DOI: 10.21037/sci.2018.08.02.Commentaries, Editorials and LettersAlternative splicingSingle genomic locusRNA nuclear exportSplicing of mRNAGenomic lociMature proteinHematopoietic stem cellsNuclear exportSplicing isoformsTranscript stabilityAlternative isoformsTranscript levelsSplicingNormal developmentStem cellsSequencing modalitiesIsoformsTranscriptomeProteomeLociProteinDiversityMRNACellsExport
2016
Single cell transcriptomics reveals unanticipated features of early hematopoietic precursors
Yang J, Tanaka Y, Seay M, Li Z, Jin J, Garmire LX, Zhu X, Taylor A, Li W, Euskirchen G, Halene S, Kluger Y, Snyder MP, Park IH, Pan X, Weissman SM. Single cell transcriptomics reveals unanticipated features of early hematopoietic precursors. Nucleic Acids Research 2016, 45: 1281-1296. PMID: 28003475, PMCID: PMC5388401, DOI: 10.1093/nar/gkw1214.Peer-Reviewed Original ResearchConceptsHematopoietic stem cellsPrecursor cellsInduction of anemiaInterferon response genesG2/M phaseEarly precursor cellsHomeostatic cellsStages of differentiationTranscription factorsSurface markersCell cycle progressionLong-term hematopoietic stem cellsSpecific augmentationAnemic miceMarked increaseEarly hematopoietic precursorsHematopoietic precursorsStem cellsCycle progressionM phaseSingle-cell transcriptomicsCellsCell differentiationHematopoietic stressLineage-specific transcription factors
2012
Gaucher disease gene GBA functions in immune regulation
Liu J, Halene S, Yang M, Iqbal J, Yang R, Mehal WZ, Chuang WL, Jain D, Yuen T, Sun L, Zaidi M, Mistry PK. Gaucher disease gene GBA functions in immune regulation. Proceedings Of The National Academy Of Sciences Of The United States Of America 2012, 109: 10018-10023. PMID: 22665763, PMCID: PMC3382552, DOI: 10.1073/pnas.1200941109.Peer-Reviewed Original ResearchConceptsGaucher diseaseHematopoietic stem cellsImmune regulationDisease severityGBA geneWidespread immune dysregulationB cell recruitmentPeripheral lymphoid organsT cell maturationLyso-GL1Immune dysregulationT helperImmune defectsTh2 cytokinesEarly thymic progenitorsLymphoid organsAntigen presentationGBA deficiencyGBA mutationsSevere diseaseClassic manifestationsClinical observationsGCase deficiencyBone marrowMature thymocytes
2000
Gene Therapy Using Hematopoietic Stem Cells: Sisyphus Approaches the Crest
Halene S, Kohn D. Gene Therapy Using Hematopoietic Stem Cells: Sisyphus Approaches the Crest. Human Gene Therapy 2000, 11: 1259-1267. PMID: 10890736, DOI: 10.1089/10430340050032366.Commentaries, Editorials and Letters
1999
Improved Expression in Hematopoietic and Lymphoid Cells in Mice After Transplantation of Bone Marrow Transduced With a Modified Retroviral Vector
Halene S, Wang L, Cooper R, Bockstoce D, Robbins P, Kohn D. Improved Expression in Hematopoietic and Lymphoid Cells in Mice After Transplantation of Bone Marrow Transduced With a Modified Retroviral Vector. Blood 1999, 94: 3349-3357. PMID: 10552944, PMCID: PMC9071851, DOI: 10.1182/blood.v94.10.3349.422k05_3349_3357.Peer-Reviewed Original ResearchMeSH Keywords3T3 CellsAnimalsBone Marrow TransplantationFemaleGene DosageGene ExpressionGene Transfer TechniquesGenetic TherapyGenetic VectorsGreen Fluorescent ProteinsHematopoietic Stem CellsLeukemia Virus, MurineLuminescent ProteinsLymphocytesMaleMiceMice, Inbred C57BLPolymerase Chain ReactionRetroviridaeTime FactorsTransduction, GeneticConceptsEnhanced green fluorescent proteinHematopoietic cellsMoMuLV LTRMammalian hematopoietic cellsMurine embryonic stem cellsStem cellsEmbryonic stem cellsRetroviral vectorsGreen fluorescent proteinMoloney murine leukemia virusMouse bone marrow transplant modelReliable gene expressionHematopoietic stem cellsStable gene transferMurine leukemia virusGene expressionMND vectorTransduction efficiencyFluorescent proteinCopy numberGene transferIndividual cellsAmount of proteinVector copy numberBone marrow transplant model
1998
Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells
Robbins P, Skelton D, Yu X, Halene S, Leonard E, Kohn D. Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells. Proceedings Of The National Academy Of Sciences Of The United States Of America 1998, 95: 10182-10187. PMID: 9707621, PMCID: PMC21482, DOI: 10.1073/pnas.95.17.10182.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBase SequenceBone Marrow TransplantationColony-Forming Units AssayDNA MethylationDNA PrimersDNA, RecombinantFemaleFounder EffectGene ExpressionGene Transfer TechniquesGenetic VectorsHematopoietic Stem CellsMaleMiceMice, Inbred C57BLMoloney murine leukemia virusRepetitive Sequences, Nucleic AcidRetroviridaeTransduction, GeneticTransplantation, IsogeneicVirus Integration