2015
Mature T cell responses are controlled by microRNA-142
Sun Y, Oravecz-Wilson K, Mathewson N, Wang Y, McEachin R, Liu C, Toubai T, Wu J, Rossi C, Braun T, Saunders T, Reddy P. Mature T cell responses are controlled by microRNA-142. Journal Of Clinical Investigation 2015, 125: 2825-2840. PMID: 26098216, PMCID: PMC4563679, DOI: 10.1172/jci78753.Peer-Reviewed Original ResearchConceptsCell cyclingE2F transcription factorsAtypical E2F transcription factorMature T cell responsesCell proliferationShort palindromic repeatsUpregulation of genesMiR-142T cell developmentTranscription factorsBioinformatics analysisTarget genesPalindromic repeatsMolecular approachesMolecular mechanismsCell developmentMolecular processesMicroRNA-142Targeted deletionWT T cellsGenesE2F8E2F7Multiple murine modelsT cell proliferationConnective tissue growth factor and integrin αvβ6: A new pair of regulators critical for ductular reaction and biliary fibrosis in mice
Pi L, Robinson P, Jorgensen M, Oh S, Brown A, Weinreb P, Le Trinh T, Yianni P, Liu C, Leask A, Violette S, Scott E, Schultz G, Petersen B. Connective tissue growth factor and integrin αvβ6: A new pair of regulators critical for ductular reaction and biliary fibrosis in mice. Hepatology 2015, 61: 678-691. PMID: 25203810, PMCID: PMC4303530, DOI: 10.1002/hep.27425.Peer-Reviewed Original ResearchMeSH KeywordsAdult Stem CellsAnimalsAntigens, NeoplasmBile Duct NeoplasmsBile Ducts, IntrahepaticCell AdhesionChemical and Drug Induced Liver InjuryCholangiocarcinomaConnective Tissue Growth FactorFemaleFibronectinsHumansIntegrinsLiver CirrhosisMaleMiceMice, KnockoutPyridinesRabbitsRatsTransforming Growth Factor beta1ConceptsConnective tissue growth factorDuctular reactionTissue growth factorIntegrin αvβ6Oval cell activationLiver injuryGrowth factorTamoxifen-inducible Cre-loxP systemCell activationRole of CTGFAlpha-smooth muscle actin stainingRelated liver diseasesSevere liver injuryGreen fluorescent protein reporter miceFibrosis-related genesMuscle actin stainingSirius red stainingPotential therapeutic targetHuman cirrhotic liversEpithelial cell adhesion moleculeDuctular epithelial cellsBiliary fibrosisCre-loxP systemLiver diseaseSerum markers
2013
Pharmacologic inhibition of PKCα and PKCθ prevents GVHD while preserving GVL activity in mice
Haarberg K, Li J, Heinrichs J, Wang D, Liu C, Bronk C, Kaosaard K, Owyang A, Holland S, Masuda E, Tso K, Blazar B, Anasetti C, Beg A, Yu X. Pharmacologic inhibition of PKCα and PKCθ prevents GVHD while preserving GVL activity in mice. Blood 2013, 122: 2500-2511. PMID: 23908466, PMCID: PMC3790515, DOI: 10.1182/blood-2012-12-471938.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsCell SeparationDisease Models, AnimalEnzyme InhibitorsFlow CytometryGraft vs Host DiseaseGraft vs Leukemia EffectHematopoietic Stem Cell TransplantationIsoenzymesLeukemiaLymphocyte ActivationLymphomaMiceMice, Inbred BALB CMice, Inbred C57BLMice, KnockoutProtein Kinase CProtein Kinase C-alphaProtein Kinase C-thetaT-LymphocytesConceptsHematopoietic cell transplantationDonor T cell proliferationAllogeneic hematopoietic cell transplantationT cell proliferationGVL activityGVL effectCytokine productionT cellsPharmacologic inhibitionChemokine/cytokine productionT-cell cytotoxicDonor T cellsPreclinical murine modelsPotential therapeutic targetT cell activationGVHD inductionGVHD preventionPrevents GVHDHost diseaseLeukemia effectSevere graftTherapeutic optionsCell transplantationEffective therapyPharmacologic approachesc‐Rel is an essential transcription factor for the development of acute graft‐versus‐host disease in mice
Yu Y, Wang D, Kaosaard K, Liu C, Fu J, Haarberg K, Anasetti C, Beg A, Yu X. c‐Rel is an essential transcription factor for the development of acute graft‐versus‐host disease in mice. European Journal Of Immunology 2013, 43: 2327-2337. PMID: 23716202, PMCID: PMC3940138, DOI: 10.1002/eji.201243282.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBone Marrow TransplantationCell DifferentiationCell ProliferationForkhead Transcription FactorsGraft vs Host DiseaseImmune ToleranceLymphocyte ActivationMiceMice, Inbred BALB CMice, Inbred C57BLMice, KnockoutProto-Oncogene Proteins c-relTh1 CellsTh17 CellsT-Lymphocytes, RegulatoryTransplantation, HomologousConceptsT cellsAcute GVHDHost diseaseAllogeneic bone marrow transplantationAllogeneic hematopoietic cell transplantationC-RelGVHD target organsHematopoietic cell transplantationRegulatory T cellsBone marrow transplantationAcute graftLeukemia responseTransplant toleranceAllogeneic recipientsMarrow transplantationMinor histocompatibilityCell transplantationTh1 cellsLymphoid organsMurine modelTarget organsTherapeutic interventionsNF-κB familyGraftPotential targetPhosphatidylinositol 3-Kinase–Independent Signaling Pathways Contribute to ICOS-Mediated T Cell Costimulation in Acute Graft-Versus-Host Disease in Mice
Li J, Heinrichs J, Leconte J, Haarberg K, Semple K, Liu C, Gigoux M, Kornete M, Piccirillo C, Suh W, Yu X. Phosphatidylinositol 3-Kinase–Independent Signaling Pathways Contribute to ICOS-Mediated T Cell Costimulation in Acute Graft-Versus-Host Disease in Mice. The Journal Of Immunology 2013, 191: 200-207. PMID: 23729441, PMCID: PMC4318500, DOI: 10.4049/jimmunol.1203485.Peer-Reviewed Original ResearchMeSH KeywordsAcute DiseaseAnimalsDisease Models, AnimalGene Knock-In TechniquesGraft vs Host DiseaseInducible T-Cell Co-Stimulator ProteinLymphocyte ActivationMiceMice, 129 StrainMice, Inbred BALB CMice, Inbred C57BLMice, KnockoutMice, TransgenicPhosphatidylinositol 3-KinaseSignal TransductionT-Lymphocyte SubsetsConceptsCD8 T cellsCD4 T cellsT cellsHost diseaseWild-type CD8 T cellsCD8 T cell compartmentAllogeneic bone marrow transplantationAcute Graft-VersusPathogenic potentialTotal T cellsAlloreactive T cellsBone marrow transplantationT cell compartmentWild-type T cellsIntracellular calcium mobilizationVivo pathogenic potentialT cell costimulationT cell activationKnockout T cellsAcute graftAcute GVHDGraft-VersusSevere GVHDGVHD modelMarrow transplantation
2012
Interleukin-22 Protects Intestinal Stem Cells from Immune-Mediated Tissue Damage and Regulates Sensitivity to Graft versus Host Disease
Hanash A, Dudakov J, Hua G, O’Connor M, Young L, Singer N, West M, Jenq R, Holland A, Kappel L, Ghosh A, Tsai J, Rao U, Yim N, Smith O, Velardi E, Hawryluk E, Murphy G, Liu C, Fouser L, Kolesnick R, Blazar B, van den Brink M. Interleukin-22 Protects Intestinal Stem Cells from Immune-Mediated Tissue Damage and Regulates Sensitivity to Graft versus Host Disease. Immunity 2012, 37: 339-350. PMID: 22921121, PMCID: PMC3477611, DOI: 10.1016/j.immuni.2012.05.028.Peer-Reviewed Original ResearchConceptsIL-23-responsive innate lymphoid cellsIntestinal IL-22IL-22Intestinal stem cellsTissue damageHost diseaseTransplant recipientsIL-22 deficiencyInflammatory intestinal damageDonor immune systemInnate lymphoid cellsBone marrow transplantIL-22 receptorStem cellsILC frequenciesPretransplant conditioningIntestinal damageMarrow transplantCrypt apoptosisLymphoid cellsImmune systemGVHDTissue sensitivityProtective factorsEpithelial integrityDonor- but not host-derived interleukin-10 contributes to the regulation of experimental graft-versus-host disease
Tawara I, Sun Y, Liu C, Toubai T, Nieves E, Evers R, Alrubaie M, Mathewson N, Tamaki H, Reddy P. Donor- but not host-derived interleukin-10 contributes to the regulation of experimental graft-versus-host disease. Journal Of Leukocyte Biology 2012, 91: 667-675. PMID: 22262800, PMCID: PMC3317273, DOI: 10.1189/jlb.1011510.Peer-Reviewed Original ResearchConceptsSuppression of GVHDSeverity of GVHDIL-10Donor TregsBM graftsRegulation of GVHDImmune-regulatory cytokinesInterleukin-10 contributeAcute GVHDClinical GVHDGVHD severityHost diseaseDonor graftsGVHDPreclinical modelsTregsGene polymorphismsExperimental graftCellular subsetsGraftSeverityHost cellsFunctional relevanceHost tissuesIL
2011
Prevention of GVHD while sparing GVL effect by targeting Th1 and Th17 transcription factor T-bet and RORγt in mice
Yu Y, Wang D, Liu C, Kaosaard K, Semple K, Anasetti C, Yu X. Prevention of GVHD while sparing GVL effect by targeting Th1 and Th17 transcription factor T-bet and RORγt in mice. Blood 2011, 118: 5011-5020. PMID: 21856864, PMCID: PMC3208306, DOI: 10.1182/blood-2011-03-340315.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsAntineoplastic AgentsCells, CulturedCombined Modality TherapyGraft vs Host DiseaseGraft vs Leukemia EffectHematopoietic Stem Cell TransplantationLeukemiaMiceMice, Inbred C57BLMice, KnockoutMolecular Targeted TherapyNuclear Receptor Subfamily 1, Group F, Member 3T-Box Domain ProteinsTh1 CellsTh17 CellsTransplantation, HomologousConceptsHematopoietic cell transplantationGVL effectT-betT cellsTranscription factor T-betPrevention of GVHDDonor T cellsCD8 T cellsAmeliorated GVHDGVL activityNaive ThTh17 subsetAdoptive transferCell transplantationTh17 differentiationEffective therapyHematologic malignanciesAllogeneic hostsGVHDMajor MHCTh1Regulatory phenotypeSkewed differentiationTargeted disruptionRORγtAbrogation of donor T-cell IL-21 signaling leads to tissue-specific modulation of immunity and separation of GVHD from GVL
Hanash A, Kappel L, Yim N, Nejat R, Goldberg G, Smith O, Rao U, Dykstra L, Na I, Holland A, Dudakov J, Liu C, Murphy G, Leonard W, Heller G, van den Brink M. Abrogation of donor T-cell IL-21 signaling leads to tissue-specific modulation of immunity and separation of GVHD from GVL. Blood 2011, 118: 446-455. PMID: 21596854, PMCID: PMC3138694, DOI: 10.1182/blood-2010-07-294785.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsGene Knockdown TechniquesGraft vs Host DiseaseGraft vs Leukemia EffectHumansImmunity, InnateInterleukin-21 Receptor alpha SubunitInterleukinsLymphocytes, Tumor-InfiltratingMiceMice, Inbred BALB CMice, Inbred C57BLMice, Inbred DBAMice, KnockoutOrgan SpecificitySignal TransductionTissue DonorsT-LymphocytesTransplantation ImmunologyConceptsSeparation of GVHDDonor T cellsKO T cellsIL-21T cellsTissue-specific modulationGastrointestinal GVHDCytokine productionWild-type donor T cellsDonor regulatory T cellsTh cell cytokine productionPeripheral T cell functionMesenteric lymph nodesRegulatory T cellsTh cell functionIL-21 signalingInflammatory cytokine productionBM transplantation modelT cell functionLymphoma responseLymph nodesProinflammatory cytokinesTransplantation outcomesTransplantation modelGVHDPretransplant CSF-1 therapy expands recipient macrophages and ameliorates GVHD after allogeneic hematopoietic cell transplantation
Hashimoto D, Chow A, Greter M, Saenger Y, Kwan W, Leboeuf M, Ginhoux F, Ochando J, Kunisaki Y, van Rooijen N, Liu C, Teshima T, Heeger P, Stanley E, Frenette P, Merad M. Pretransplant CSF-1 therapy expands recipient macrophages and ameliorates GVHD after allogeneic hematopoietic cell transplantation. Journal Of Experimental Medicine 2011, 208: 1069-1082. PMID: 21536742, PMCID: PMC3092347, DOI: 10.1084/jem.20101709.Peer-Reviewed Original ResearchConceptsDonor allogeneic T cellsDonor T cell expansionAllogeneic hematopoietic cell transplantationAllogeneic T cellsHematopoietic cell transplantationAllo-HCTT cell expansionT cellsAcute GVHDCell transplantationHost macrophagesHost antigen-presenting cellsMacrophage poolPotential prophylactic therapyAlloreactive T cellsAntigen-presenting cellsAcute graftGVHD morbidityGVHD mortalityHost DCsHost diseaseProphylactic therapyRecipient miceGVHDRecipient macrophagesRoles of CD28, CTLA4, and Inducible Costimulator in Acute Graft-versus-Host Disease in Mice
Li J, Semple K, Suh W, Liu C, Chen F, Blazar B, Yu X. Roles of CD28, CTLA4, and Inducible Costimulator in Acute Graft-versus-Host Disease in Mice. Transplantation And Cellular Therapy 2011, 17: 962-969. PMID: 21447398, PMCID: PMC3131782, DOI: 10.1016/j.bbmt.2011.01.018.Peer-Reviewed Original ResearchMeSH KeywordsAbataceptAcute DiseaseAnimalsAntigens, CDAntigens, Differentiation, T-LymphocyteB7-1 AntigenB7-2 AntigenBone Marrow TransplantationCD28 AntigensCTLA-4 AntigenFas Ligand ProteinGraft vs Host DiseaseImmune ToleranceImmunoconjugatesInducible T-Cell Co-Stimulator ProteinInterferon-gammaLymphocyte ActivationMiceMice, Inbred BALB CMice, Inbred C57BLMice, KnockoutRadiation ChimeraT-Lymphocyte SubsetsTransplantation, HomologousTumor Necrosis Factor-alphaConceptsAllogeneic bone marrow transplantationBone marrow transplantationInducible costimulatorRole of CD28T cellsCTLA4 signalsHost diseaseMarrow transplantationMyeloablative allogeneic bone marrow transplantationPathogenic T cell responsesDevelopment of GVHDSeverity of GVHDT cell responsesT cell toleranceAbsence of B7T cell activationAcute graftAcute GVHDICOS signalingPrevents GVHDCTLA4-IgCD28 familyGVHDEffector functionsCell tolerance
2010
Absence of P-Selectin in Recipients of Allogeneic Bone Marrow Transplantation Ameliorates Experimental Graft-versus-Host Disease
Lu S, Holland A, Na I, Terwey T, Alpdogan O, Bautista J, Smith O, Suh D, King C, Kochman A, Hubbard V, Rao U, Yim N, Liu C, Laga A, Murphy G, Jenq R, Zakrzewski J, Penack O, Dykstra L, Bampoe K, Perez L, Furie B, Furie B, van den Brink M. Absence of P-Selectin in Recipients of Allogeneic Bone Marrow Transplantation Ameliorates Experimental Graft-versus-Host Disease. The Journal Of Immunology 2010, 185: 1912-1919. PMID: 20622117, PMCID: PMC3752704, DOI: 10.4049/jimmunol.0903148.Peer-Reviewed Original ResearchConceptsSecondary lymphoid organsDonor T cellsAllogeneic bone marrow transplantationAlloreactive T cellsBone marrow transplantationT cellsWT T cellsP-selectinP-selectin glycoprotein ligand-1P-selectin ligandsMarrow transplantationSmall bowelInflamed tissuesDonor alloreactive T cellsHost disease (GVHD) pathophysiologyGVHD target organsAlloactivated T cellsLigand 1Wild-type recipientsGVHD mortalityGVHD prophylaxisHost diseaseLymphoid organsPeyer's patchesExperimental disease
2008
IL-17 contributes to CD4-mediated graft-versus-host disease
Kappel L, Goldberg G, King C, Suh D, Smith O, Ligh C, Holland A, Grubin J, Mark N, Liu C, Iwakura Y, Heller G, van den Brink M. IL-17 contributes to CD4-mediated graft-versus-host disease. Blood 2008, 113: 945-952. PMID: 18931341, PMCID: PMC2630280, DOI: 10.1182/blood-2008-08-172155.Peer-Reviewed Original ResearchConceptsRecipients of ILT cellsGVHD mortalityHost diseaseIL-17Proinflammatory cytokinesAllogeneic bone marrow transplantAllogeneic BMT modelIL-17 contributesDonor T cellsBone marrow transplantWhole T cellsT-cell recipientsAcute graftGVHD developmentGVT activityAllograft rejectionTh17 cellsIL-17FIL-22Interleukin-17Marrow transplantAutoimmune diseasesTh1 cellsLymphoid organsHistone deacetylase inhibition modulates indoleamine 2,3-dioxygenase–dependent DC functions and regulates experimental graft-versus-host disease in mice
Reddy P, Sun Y, Toubai T, Duran-Struuck R, Clouthier S, Weisiger E, Maeda Y, Tawara I, Krijanovski O, Gatza E, Liu C, Malter C, Mascagni P, Dinarello C, Ferrara J. Histone deacetylase inhibition modulates indoleamine 2,3-dioxygenase–dependent DC functions and regulates experimental graft-versus-host disease in mice. Journal Of Clinical Investigation 2008, 118: 2562-2573. PMID: 18568076, PMCID: PMC2430497, DOI: 10.1172/jci34712.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsAntigens, CDBone Marrow TransplantationCytokinesDendritic CellsEnzyme InhibitorsFemaleGene ExpressionGraft vs Host DiseaseHistone Deacetylase InhibitorsHumansHydroxamic AcidsIndoleamine-Pyrrole 2,3,-DioxygenaseLipopolysaccharidesLymphocyte ActivationMiceMice, Inbred BALB CMice, Inbred C3HMice, Inbred C57BLMice, Inbred StrainsMice, KnockoutRNA, Small InterferingSurvival AnalysisT-LymphocytesVorinostatConceptsDC functionHDAC inhibitorsSuberoylanilide hydroxamic acidHost diseaseExperimental graftBlockade of IDOPretreatment of DCsAllogeneic BM transplantationBM-derived cellsImmune-mediated diseasesExpression of CD40Expression of indoleamineBM transplantation modelExposure of DCsInduction of IDOVivo functional roleHistone deacetylase inhibitionHistone deacetylase inhibitorsMechanism of actionProinflammatory cytokinesBM transplantationWT DCsTransplantation modelImmunomodulatory functionsDeacetylase inhibition
2007
IFN-γ and Fas Ligand Are Required for Graft-versus-Tumor Activity against Renal Cell Carcinoma in the Absence of Lethal Graft-versus-Host Disease
Ramirez-Montagut T, Chow A, Kochman A, Smith O, Suh D, Sindhi H, Lu S, Borsotti C, Grubin J, Patel N, Terwey T, Kim T, Heller G, Murphy G, Liu C, Alpdogan O, van den Brink M. IFN-γ and Fas Ligand Are Required for Graft-versus-Tumor Activity against Renal Cell Carcinoma in the Absence of Lethal Graft-versus-Host Disease. The Journal Of Immunology 2007, 179: 1669-1680. PMID: 17641033, DOI: 10.4049/jimmunol.179.3.1669.Peer-Reviewed Original ResearchConceptsRenal cell carcinomaMurine renal cell carcinomaT cellsCell carcinomaGVT activityHost diseaseRenca cellsIFN-gammaTumor activityAllogeneic bone marrow transplantation modelFas ligandAbsence of graftRecipients of IFNBone marrow transplantation modelMechanism of graftMembrane-bound TNF-alphaTumor-bearing miceLethal graftLethal GVHDSevere GVHDTNF-alphaTransplantation modelTransplanted miceLytic capacitySolid tumorsCCR1/CCL5 (RANTES) receptor-ligand interactions modulate allogeneic T-cell responses and graft-versus-host disease following stem-cell transplantation
Choi S, Hildebrandt G, Olkiewicz K, Hanauer D, Chaudhary M, Silva I, Rogers C, Deurloo D, Fisher J, Liu C, Adams D, Chensue S, Cooke K. CCR1/CCL5 (RANTES) receptor-ligand interactions modulate allogeneic T-cell responses and graft-versus-host disease following stem-cell transplantation. Blood 2007, 110: 3447-3455. PMID: 17641205, PMCID: PMC2200916, DOI: 10.1182/blood-2007-05-087403.Peer-Reviewed Original ResearchConceptsStem cell transplantationT cell responsesCCR1 expressionHost diseaseT cellsAllogeneic T-cell responsesAllogeneic stem cell transplantationCCR1-deficient miceDevelopment of GVHDCytolytic effector functionDonor cellsT cell functionChemokine receptor-ligand interactionsT cell proliferationHost target tissuesReceptor-ligand interactionsActivated T cellsAcute graftGVHD mortalityGVL activityIFNgamma secretionAllo-SCTGVHD severityLeukemic infiltrationLeukemic relapse
2005
A crucial role for antigen-presenting cells and alloantigen expression in graft-versus-leukemia responses
Reddy P, Maeda Y, Liu C, Krijanovski O, Korngold R, Ferrara J. A crucial role for antigen-presenting cells and alloantigen expression in graft-versus-leukemia responses. Nature Medicine 2005, 11: 1244-1249. PMID: 16227991, DOI: 10.1038/nm1309.Peer-Reviewed Original ResearchConceptsBone marrow transplantationAllogeneic bone marrow transplantationGVL responseLeukemia responseAlloantigen expressionInduction of graftAntigen-presenting cellsHost diseaseSerious complicationsDonor APCsMarrow transplantationTumor burdenTumor modelGraftAPCHost originPotent formResponseImmunotherapyCellsComplicationsTransplantationAcuityExpressionTumorsCCR2 is required for CD8-induced graft-versus-host disease
Terwey T, Kim T, Kochman A, Hubbard V, Lu S, Zakrzewski J, Ramirez-Montagut T, Eng J, Muriglan S, Heller G, Murphy G, Liu C, Budak-Alpdogan T, Alpdogan O, van den Brink M. CCR2 is required for CD8-induced graft-versus-host disease. Blood 2005, 106: 3322-3330. PMID: 16037386, PMCID: PMC1895329, DOI: 10.1182/blood-2005-05-1860.Peer-Reviewed Original ResearchConceptsCC chemokine receptor 2Hematopoietic stem cell transplantationDevelopment of GVHDT cellsT cell migrationHost diseaseAllogeneic hematopoietic stem cell transplantationDonor-derived T cellsControl of CD8Donor-derived CD8GVHD target organsMurine bone marrow transplantation modelBone marrow transplantation modelStem cell transplantationChemokine receptor 2IFN-gamma productionWild-type CD8Alloreactive proliferationDonor CD8GVHD morbidityGVT activityTumor effectMajor complicationsCCR2 signalingCell transplantationCritical role of host γδ T cells in experimental acute graft-versus-host disease
Maeda Y, Reddy P, Lowler K, Liu C, Bishop D, Ferrara J. Critical role of host γδ T cells in experimental acute graft-versus-host disease. Blood 2005, 106: 749-755. PMID: 15797996, PMCID: PMC1895173, DOI: 10.1182/blood-2004-10-4087.Peer-Reviewed Original ResearchMeSH KeywordsAcute DiseaseAdoptive TransferAnimalsBone Marrow TransplantationCD40 LigandCell AdhesionCell CommunicationDendritic CellsFemaleGraft vs Host DiseaseInterferon-gammaMiceMice, Inbred BALB CMice, Inbred C57BLMice, KnockoutReceptors, Antigen, T-Cell, gamma-deltaT-Lymphocyte SubsetsTransplantation, HomologousTumor Necrosis Factor-alphaConceptsGammadelta T cellsDendritic cellsT cellsHost diseaseAllostimulatory capacityAllogeneic bone marrow transplantation modelAllogeneic T cell proliferationDonor T cell expansionWild-type B6 recipientsHost antigen-presenting cellsTumor necrosis factor alphaExperimental acute graftWT dendritic cellsNormal dendritic cellsBone marrow transplantation modelBM transplant recipientsΓδ T cellsT cell expansionAntigen-presenting cellsNecrosis factor alphaT cell proliferationAcute graftAcute GVHDB6 recipientsGVHD mortalityImpact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid -Glucosidase in a Model of Glycogen Storage Disease Type II
Cresawn K, Fraites T, Wasserfall C, Atkinson M, Lewis M, Porvasnik S, Liu C, Mah C, Byrne B. Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid -Glucosidase in a Model of Glycogen Storage Disease Type II. Human Gene Therapy 2005, 16: 68-80. PMID: 15703490, DOI: 10.1089/hum.2005.16.68.Peer-Reviewed Original ResearchConceptsGlycogen storage disease type IIImmune-tolerant miceStorage disease type IIAntibody responseContractile functionImmune responseHumoral immune responseHind limb musclesAcid alpha-glucosidaseCardiorespiratory failureType IIAntibody formationReduced glycogenGlycogen reductionLysosomal storage diseaseSoleus muscleGAA levelsMiceEnzyme levelsSkeletal muscleStorage diseaseLevel of activityMuscleRecombinant adenoAlpha-glucosidase