Featured Publications
A highly efficient and faithful MDS patient-derived xenotransplantation model for pre-clinical studies
Song Y, Rongvaux A, Taylor A, Jiang T, Tebaldi T, Balasubramanian K, Bagale A, Terzi YK, Gbyli R, Wang X, Fu X, Gao Y, Zhao J, Podoltsev N, Xu M, Neparidze N, Wong E, Torres R, Bruscia EM, Kluger Y, Manz MG, Flavell RA, Halene S. A highly efficient and faithful MDS patient-derived xenotransplantation model for pre-clinical studies. Nature Communications 2019, 10: 366. PMID: 30664659, PMCID: PMC6341122, DOI: 10.1038/s41467-018-08166-x.Peer-Reviewed Original ResearchConceptsPatient-derived xenograftsMyelodysplastic syndromeXenotransplantation modelDysplastic morphologyImmunodeficient murine hostsPre-clinical studiesMDS stem cellsMDS subtypesComprehensive preclinical studiesPreclinical studiesTherapeutic efficacyMurine hostSerial transplantationDrug mechanismsMDS researchStem cell propagationStem cellsDifferentiation potentialHematopoietic stem cell nicheGenetic complexityNovel avenuesStem cell nicheCell propagationDisease representationsImmunodeficient
2020
Humanized 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.BooksMeSH KeywordsAnimalsDisease Models, AnimalHematopoietic Stem Cell TransplantationHematopoietic Stem CellsHeterograftsHumansLeukemia, Myeloid, AcuteMiceMice, Inbred NODMice, SCIDMyelodysplastic Syndromes
2018
SRSF2 mutations drive oncogenesis by activating a global program of aberrant alternative splicing in hematopoietic cells
Liang Y, Tebaldi T, Rejeski K, Joshi P, Stefani G, Taylor A, Song Y, Vasic R, Maziarz J, Balasubramanian K, Ardasheva A, Ding A, Quattrone A, Halene S. SRSF2 mutations drive oncogenesis by activating a global program of aberrant alternative splicing in hematopoietic cells. Leukemia 2018, 32: 2659-2671. PMID: 29858584, PMCID: PMC6274620, DOI: 10.1038/s41375-018-0152-7.Peer-Reviewed Original ResearchMeSH KeywordsAlternative SplicingBinding SitesCarcinogenesisCell LineExonsHEK293 CellsHematopoiesisHematopoietic Stem Cell TransplantationHumansMutationMyelodysplastic SyndromesPhenotypeRNARNA SplicingRNA, MessengerSerine-Arginine Splicing FactorsConceptsSplicing factorsRNA processingAlternative splicingGene productsSplicing factor SRSF2Gene regulatory eventsAberrant alternative splicingSplice alterationsRecurrent mutationsSplicing proteinsHITS-CLIPSR familyMRNA splicingSplicing genesHematopoietic differentiationRegulatory eventsImpairs hematopoietic differentiationMolecular explanationWidespread modificationSplicingHematopoietic cellsMutationsBinding eventsOncogenesisProtein
2016
Peripheral blood CD34+ cells efficiently engraft human cytokine knock-in mice
Saito Y, Ellegast JM, Rafiei A, Song Y, Kull D, Heikenwalder M, Rongvaux A, Halene S, Flavell RA, Manz MG. Peripheral blood CD34+ cells efficiently engraft human cytokine knock-in mice. Blood 2016, 128: 1829-1833. PMID: 27543436, PMCID: PMC5054696, DOI: 10.1182/blood-2015-10-676452.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsAnimals, NewbornAntigens, CD34CytokinesGene Knock-In TechniquesHematopoietic Stem Cell TransplantationHematopoietic Stem CellsHumansLymphoid TissueMice, TransgenicConceptsPeripheral blood CD34Higher engraftment levelsColony-stimulating factorBone marrowEngraftment levelsHuman hemato-lymphoid systemBlood CD34Granulocyte-macrophage colony-stimulating factorAdult recipient miceNonobese diabetic/Macrophage colony-stimulating factorSignal regulatory protein αCord blood-derived CD34Blood-derived CD34Regulatory protein αMISTRG miceAdult recipientsRecipient miceDiabetic/T cellsNonhematopoietic organsMyeloid cellsImmune systemCD34Mice
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