Leukemia Program

The DF/HCC Leukemia Program conducts innovative basic, translational and clinical research to improve the understanding and treatment of leukemia and related disorders, with a focus on discoveries that will lead to improvements in diagnosis, prognosis, and therapy. 

Program members have expertise in identifying oncogenes, defining basic mechanisms of neoplastic transformation in cell culture and animal models of leukemia, analyzing the biology of myeloproliferative neoplasms, developing novel immunotherapeutic approaches, conducting clinical trials of novel therapies, and improving outcomes using stem cell transplantation and adoptive cellular therapies. Program publications include landmark advances that have improved the understanding of leukemia pathobiology and translated gains into improved therapies. To harness the power of interdisciplinary translational science, the Program brings together over 86 investigators representing multiple DF/HCC institutions, departments, and disciplines.

Recent Publications

  • Zhao J, Cato LD, Arora UP, Bao EL, Bryant SC, Williams N, Jia Y, Goldman SR, Nangalia J, Erb MA, Vos SM, Armstrong SA, Sankaran VG. Inherited blood cancer predisposition through altered transcription elongation. Cell 2024. PubMed
  • Ma F, Ghimire L, Ren Q, Fan Y, Chen T, Balasubramanian A, Hsu A, Liu F, Yu H, Xie X, Xu R, Luo HR. Gasdermin E dictates inflammatory responses by controlling the mode of neutrophil death. Nat Commun 2024; 15:386. PubMed
  • Wheeler EC, Martin BJE, Doyle WC, Neaher S, Conway CA, Pitton CN, Gorelov RA, Donahue M, Jann JC, Abdel-Wahab O, Taylor J, Seiler M, Buonamici S, Pikman Y, Garcia JS, Belizaire R, Adelman K, Tothova Z. Splicing modulators impair DNA damage response and induce killing of cohesin-mutant MDS and AML. Sci Transl Med 2024; 16:eade2774. PubMed
  • Paolino J, Dimitrov B, Apsel Winger B, Sandoval-Perez A, Rangarajan AV, Ocasio-Martinez N, Tsai HK, Li Y, Robichaud AL, Khalid D, Hatton C, Gillani R, Polonen P, Dilig A, Gotti G, Kavanagh J, Adhav AA, Gow S, Tsai J, Li Y, Ebert BL, Van Allen EM, Bledsoe J, Kim AS, Tasian SK, Cooper SL, Cooper TM, Hijiya N, Sulis ML, Shukla NN, Magee JA, Mullighan CG, Burke MJ, Luskin MR, Mar BG, Jacobson MP, Harris MH, Stegmaier K, Place AE, Pikman Y. Integration of genomic sequencing drives therapeutic targeting of PDGFRA in T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma. Clin Cancer Res 2023. PubMed
  • Brown DW, Cato LD, Zhao Y, Nandakumar SK, Bao EL, Gardner EJ, Hubbard AK, DePaulis A, Rehling T, Song L, Yu K, Chanock SJ, Perry JRB, Sankaran VG, Machiela MJ. Shared and distinct genetic etiologies for different types of clonal hematopoiesis. Nat Commun 2023; 14:5536. PubMed
  • Versluis J, Saber W, Tsai HK, Gibson CJ, Dillon LW, Mishra A, McGuirk J, Maziarz RT, Westervelt P, Hegde P, Mukherjee D, Martens MJ, Logan B, Horowitz M, Hourigan CS, Nakamura R, Cutler C, Lindsley RC, . Allogeneic Hematopoietic Cell Transplantation Improves Outcome in Myelodysplastic Syndrome Across High-Risk Genetic Subgroups: Genetic Analysis of the Blood and Marrow Transplant Clinical Trials Network 1102 Study. J Clin Oncol 2023. PubMed
  • Hantel A, Luskin MR, Khan I, Warner E, Patel AA, Walsh TP, DeAngelo DJ, Lathan CS, Abel GA. Use, variability, and justification of eligibility criteria for phase II and III clinical trials in acute leukemia. Haematologica 2023. PubMed
  • Kooshesh KA, Foy BH, Sykes DB, Gustafsson K, Scadden DT. Health Consequences of Thymus Removal in Adults. N Engl J Med 2023; 389:406-417. PubMed
  • Tsai JM, Aguirre JD, Li YD, Brown J, Focht V, Kater L, Kempf G, Sandoval B, Schmitt S, Rutter JC, Galli P, Sandate CR, Cutler JA, Zou C, Donovan KA, Lumpkin RJ, Cavadini S, Park PMC, Sievers Q, Hatton C, Ener E, Regalado BD, Sperling MT, Słabicki M, Kim J, Zon R, Zhang Z, Miller PG, Belizaire R, Sperling AS, Fischer ES, Irizarry R, Armstrong SA, Thomä NH, Ebert BL. UBR5 forms ligand-dependent complexes on chromatin to regulate nuclear hormone receptor stability. Mol Cell 2023; 83:2753-2767.e10. PubMed
  • Griffin GK, Booth CAG, Togami K, Chung SS, Ssozi D, Verga JA, Bouyssou JM, Lee YS, Shanmugam V, Hornick JL, LeBoeuf NR, Morgan EA, Bernstein BE, Hovestadt V, van Galen P, Lane AA. Ultraviolet radiation shapes dendritic cell leukaemia transformation in the skin. Nature 2023; 618:834-841. PubMed
  • Martin-Rufino JD, Castano N, Pang M, Grody EI, Joubran S, Caulier A, Wahlster L, Li T, Qiu X, Riera-Escandell AM, Newby GA, Al'Khafaji A, Chaudhary S, Black S, Weng C, Munson G, Liu DR, Wlodarski MW, Sims K, Oakley JH, Fasano RM, Xavier RJ, Lander ES, Klein DE, Sankaran VG. Massively parallel base editing to map variant effects in human hematopoiesis. Cell 2023; 186:2456-2474.e24. PubMed
  • Wong WJ, Emdin C, Bick AG, Zekavat SM, Niroula A, Pirruccello JP, Dichtel L, Griffin G, Uddin MM, Gibson CJ, Kovalcik V, Lin AE, McConkey ME, Vromman A, Sellar RS, Kim PG, Agrawal M, Weinstock J, Long MT, Yu B, Banerjee R, Nicholls RC, Dennis A, Kelly M, Loh PR, McCarroll S, Boerwinkle E, Vasan RS, Jaiswal S, Johnson AD, Chung RT, Corey K, Levy D, Ballantyne C, , Ebert BL, Natarajan P. Clonal haematopoiesis and risk of chronic liver disease. Nature 2023; 616:747-754. PubMed
  • Perner F, Stein EM, Wenge DV, Singh S, Kim J, Apazidis A, Rahnamoun H, Anand D, Marinaccio C, Hatton C, Wen Y, Stone RM, Schaller D, Mowla S, Xiao W, Gamlen HA, Stonestrom AJ, Persaud S, Ener E, Cutler JA, Doench JG, McGeehan GM, Volkamer A, Chodera JD, Nowak RP, Fischer ES, Levine RL, Armstrong SA, Cai SF. MEN1 mutations mediate clinical resistance to menin inhibition. Nature 2023. PubMed
  • Zhao J, Jia Y, Mahmut D, Deik AA, Jeanfavre S, Clish CB, Sankaran VG. Human hematopoietic stem cell vulnerability to ferroptosis. Cell 2023; 186:732-747.e16. PubMed
  • Voit RA, Tao L, Yu F, Cato LD, Cohen B, Fleming TJ, Antoszewski M, Liao X, Fiorini C, Nandakumar SK, Wahlster L, Teichert K, Regev A, Sankaran VG. A genetic disorder reveals a hematopoietic stem cell regulatory network co-opted in leukemia. Nat Immunol 2023; 24:69-83. PubMed
  • Uckelmann HJ, Haarer EL, Takeda R, Wong EM, Hatton C, Marinaccio C, Perner F, Rajput M, Antonissen NJC, Wen Y, Yang L, Brunetti L, Chen CW, Armstrong SA. Mutant NPM1 directly regulates oncogenic transcription in acute myeloid leukemia. 2022. PubMed
  • Wattrus SJ, Smith ML, Rodrigues CP, Hagedorn EJ, Kim JW, Budnik B, Zon LI. Quality assurance of hematopoietic stem cells by macrophages determines stem cell clonality. Science 2022; 377:1413-1419. PubMed
  • Hinze L, Schreek S, Zeug A, Ibrahim NK, Fehlhaber B, Loxha L, Cinar B, Ponimaskin E, Degar J, McGuckin C, Chiosis G, Eckert C, Cario G, Bornhauser B, Bourquin JP, Stanulla M, Gutierrez A. Supramolecular assembly of GSK3α as a cellular response to amino acid starvation. Mol Cell 2022. PubMed
  • Liu YC, Kwon J, Fabiani E, Xiao Z, Liu YV, Follo MY, Liu J, Huang H, Gao C, Liu J, Falconi G, Valentini L, Gurnari C, Finelli C, Cocco L, Liu JH, Jones AI, Yang J, Yang H, Thoms JAI, Unnikrishnan A, Pimanda JE, Pan R, Bassal MA, Voso MT, Tenen DG, Chai L. Demethylation and Up-Regulation of an Oncogene after Hypomethylating Therapy. N Engl J Med 2022; 386:1998-2010. PubMed
  • Aubrey BJ, Cutler JA, Bourgeois W, Donovan KA, Gu S, Hatton C, Perlee S, Perner F, Rahnamoun H, Theall ACP, Henrich JA, Zhu Q, Nowak RP, Kim YJ, Parvin S, Cremer A, Olsen SN, Eleuteri NA, Pikman Y, McGeehan GM, Stegmaier K, Letai A, Fischer ES, Liu XS, Armstrong SA. IKAROS and MENIN coordinate therapeutically actionable leukemogenic gene expression in MLL-r acute myeloid leukemia. 2022; 3:595-613. PubMed
  • Olsen SN, Godfrey L, Healy JP, Choi YA, Kai Y, Hatton C, Perner F, Haarer EL, Nabet B, Yuan GC, Armstrong SA. MLL::AF9 degradation induces rapid changes in transcriptional elongation and subsequent loss of an active chromatin landscape. Mol Cell 2022; 82:1140-1155.e11. PubMed
  • Leick MB, Silva H, Scarfò I, Larson R, Choi BD, Bouffard AA, Gallagher K, Schmidts A, Bailey SR, Kann MC, Jan M, Wehrli M, Grauwet K, Horick N, Frigault MJ, Maus MV. Non-cleavable hinge enhances avidity and expansion of CAR-T cells for acute myeloid leukemia. Cancer Cell 2022. PubMed
  • Harada T, Heshmati Y, Kalfon J, Perez MW, Xavier Ferrucio J, Ewers J, Hubbell Engler B, Kossenkov A, Ellegast JM, Yi JS, Bowker A, Zhu Q, Eagle K, Liu T, Kai Y, Dempster JM, Kugener G, Wickramasinghe J, Herbert ZT, Li CH, Vrabič Koren J, Weinstock DM, Paralkar VR, Nabet B, Lin CY, Dharia NV, Stegmaier K, Orkin SH, Pimkin M. A distinct core regulatory module enforces oncogene expression in KMT2A-rearranged leukemia. Genes Dev 2022; 36:368-389. PubMed
  • Ellegast JM, Alexe G, Hamze A, Lin S, Uckelmann HJ, Rauch PJ, Pimkin M, Ross LS, Dharia NV, Robichaud AL, Saur Conway A, Khalid D, Perry JA, Wunderlich M, Benajiba L, Pikman Y, Nabet B, Gray NS, Orkin SH, Stegmaier K. Unleashing cell-intrinsic inflammation as a strategy to kill AML blasts. 2022. PubMed
  • DeAngelo DJ, Radia DH, George TI, Robinson WA, Quiery AT, Drummond MW, Bose P, Hexner EO, Winton EF, Horny HP, Tugnait M, Schmidt-Kittler O, Evans EK, Lin HM, Mar BG, Verstovsek S, Deininger MW, Gotlib J. Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. Nat Med 2021; 27:2183-2191. PubMed
  • Cha HJ, Uyan Ö, Kai Y, Liu T, Zhu Q, Tothova Z, Botten GA, Xu J, Yuan GC, Dekker J, Orkin SH. Inner nuclear protein Matrin-3 coordinates cell differentiation by stabilizing chromatin architecture. Nat Commun 2021; 12:6241. PubMed
  • Gibson CJ, Kim HT, Zhao L, Murdock HM, Hambley B, Ogata A, Madero-Marroquin R, Wang S, Green L, Fleharty M, Dougan T, Cheng CA, Blumenstiel B, Cibulskis C, Tsuji J, Duran M, Gocke CD, Antin JH, Nikiforow S, DeZern AE, Chen YB, Ho VT, Jones RJ, Lennon NJ, Walt DR, Ritz J, Soiffer RJ, Gondek LP, Lindsley RC. Donor Clonal Hematopoiesis and Recipient Outcomes After Transplantation. J Clin Oncol 2021. PubMed
  • Bachireddy P, Azizi E, Burdziak C, Nguyen VN, Ennis CS, Maurer K, Park CY, Choo ZN, Li S, Gohil SH, Ruthen NG, Ge Z, Keskin DB, Cieri N, Livak KJ, Kim HT, Neuberg DS, Soiffer RJ, Ritz J, Alyea EP, Pe'er D, Wu CJ. Mapping the evolution of T cell states during response and resistance to adoptive cellular therapy. Cell Rep 2021; 37:109992. PubMed
  • Blanco MA, Sykes DB, Gu L, Wu M, Petroni R, Karnik R, Wawer M, Rico J, Li H, Jacobus WD, Jambhekar A, Cheloufi S, Meissner A, Hochedlinger K, Scadden DT, Shi Y. Chromatin-state barriers enforce an irreversible mammalian cell fate decision. Cell Rep 2021; 37:109967. PubMed
  • Niroula A, Sekar A, Murakami MA, Trinder M, Agrawal M, Wong WJ, Bick AG, Uddin MM, Gibson CJ, Griffin GK, Honigberg MC, Zekavat SM, Paruchuri K, Natarajan P, Ebert BL. Distinction of lymphoid and myeloid clonal hematopoiesis. Nat Med 2021. PubMed
  • Lin S, Larrue C, Scheidegger NK, Seong BKA, Dharia NV, Kuljanin M, Wechsler CS, Kugener G, Robichaud AL, Saur Conway A, Mashaka T, Mouche S, Adane B, Ryan JA, Mancias JD, Younger ST, Piccioni F, Lee LH, Wunderlich M, Letai A, Tamburini J, Stegmaier K. An in vivo CRISPR screening platform for prioritizing therapeutic targets in AML. 2021. PubMed
  • Vrooman LM, Blonquist TM, Stevenson KE, Supko JG, Hunt SK, Cronholm SM, Koch V, Kay-Green S, Athale UH, Clavell LA, Cole PD, Harris MH, Kelly KM, Laverdiere C, Leclerc JM, Michon B, Place AE, Schorin MA, Welch JJG, Neuberg DS, Sallan SE, Silverman LB. Efficacy and Toxicity of Pegaspargase and Calaspargase Pegol in Childhood Acute Lymphoblastic Leukemia: Results of DFCI 11-001. J Clin Oncol 2021. PubMed
  • Rossmann MP, Hoi K, Chan V, Abraham BJ, Yang S, Mullahoo J, Papanastasiou M, Wang Y, Elia I, Perlin JR, Hagedorn EJ, Hetzel S, Weigert R, Vyas S, Nag PP, Sullivan LB, Warren CR, Dorjsuren B, Greig EC, Adatto I, Cowan CA, Schreiber SL, Young RA, Meissner A, Haigis MC, Hekimi S, Carr SA, Zon LI. Cell-specific transcriptional control of mitochondrial metabolism by TIF1γ drives erythropoiesis. Science 2021; 372:716-721. PubMed
  • Roux B, Vaganay C, Vargas JD, Alexe G, Benaksas C, Pardieu B, Fenouille N, Ellegast JM, Malolepsza E, Ling F, Sodaro G, Ross L, Pikman Y, Conway AS, Tang Y, Wu T, Anderson DJ, Le Moigne R, Zhou HJ, Luciano F, Hartigan CR, Galinsky I, DeAngelo DJ, Stone RM, Auberger P, Schenone M, Carr SA, Guirouilh-Barbat J, Lopez B, Khaled M, Lage K, Hermine O, Hemann MT, Puissant A, Stegmaier K, Benajiba L. Targeting acute myeloid leukemia dependency on VCP-mediated DNA repair through a selective second-generation small-molecule inhibitor. Sci Transl Med 2021. PubMed
  • Kennedy AL, Myers KC, Bowman J, Gibson CJ, Camarda ND, Furutani E, Muscato GM, Klein RH, Ballotti K, Liu S, Harris CE, Galvin A, Malsch M, Dale D, Gansner JM, Nakano TA, Bertuch A, Vlachos A, Lipton JM, Castillo P, Connelly J, Churpek J, Edwards JR, Hijiya N, Ho RH, Hofmann I, Huang JN, Keel S, Lamble A, Lau BW, Norkin M, Stieglitz E, Stock W, Walkovich K, Boettcher S, Brendel C, Fleming MD, Davies SM, Weller EA, Bahl C, Carter SL, Shimamura A, Lindsley RC. Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome. Nat Commun 2021; 12:1334. PubMed
  • Słabicki M, Yoon H, Koeppel J, Nitsch L, Roy Burman SS, Di Genua C, Donovan KA, Sperling AS, Hunkeler M, Tsai JM, Sharma R, Guirguis A, Zou C, Chudasama P, Gasser JA, Miller PG, Scholl C, Fröhling S, Nowak RP, Fischer ES, Ebert BL. Small-molecule-induced polymerization triggers degradation of BCL6. Nature 2020; 588:164-168. PubMed
  • Pikman Y, Tasian SK, Sulis ML, Stevenson K, Blonquist TM, Apsel Winger B, Cooper TM, Pauly M, Maloney KW, Burke MJ, Brown PA, Gossai N, McNeer JL, Shukla NN, Cole PD, Kahn JM, Chen J, Barth MJ, Magee JA, Gennarini L, Adhav AA, Clinton CM, Ocasio-Martinez N, Gotti G, Li Y, Lin S, Imamovic A, Tognon CE, Patel T, Faust HL, Contreras CF, Cremer A, Cortopassi WA, Garrido Ruiz D, Jacobson MP, Dharia NV, Su A, Robichaud AL, Saur Conway A, Tarlock K, Stieglitz E, Place AE, Puissant A, Hunger SP, Kim AS, Lindeman NI, Gore L, Janeway KA, Silverman LB, Tyner JW, Harris MH, Loh ML, Stegmaier K. Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-risk Leukemias: A Report from the LEAP Consortium. 2021. PubMed
  • Jan M, Scarfò I, Larson RC, Walker A, Schmidts A, Guirguis AA, Gasser JA, Słabicki M, Bouffard AA, Castano AP, Kann MC, Cabral ML, Tepper A, Grinshpun DE, Sperling AS, Kyung T, Sievers QL, Birnbaum ME, Maus MV, Ebert BL. Reversible ON- and OFF-switch chimeric antigen receptors controlled by lenalidomide. Sci Transl Med 2021. PubMed
  • Oki T, Mercier F, Kato H, Jung Y, McDonald TO, Spencer JA, Mazzola MC, van Gastel N, Lin CP, Michor F, Kitamura T, Scadden DT. Imaging dynamic mTORC1 pathway activity in vivo reveals marked shifts that support time-specific inhibitor therapy in AML. Nat Commun 2021; 12:245. PubMed
  • Bhatt S, Pioso MS, Olesinski EA, Yilma B, Ryan JA, Mashaka T, Leutz B, Adamia S, Zhu H, Kuang Y, Mogili A, Louissaint A, Bohl SR, Kim AS, Mehta AK, Sanghavi S, Wang Y, Morris E, Halilovic E, Paweletz CP, Weinstock DM, Garcia JS, Letai A. Reduced Mitochondrial Apoptotic Priming Drives Resistance to BH3 Mimetics in Acute Myeloid Leukemia. Cancer Cell 2020; 38:872-890.e6. PubMed
  • van Gastel N, Spinelli JB, Sharda A, Schajnovitz A, Baryawno N, Rhee C, Oki T, Grace E, Soled HJ, Milosevic J, Sykes DB, Hsu PP, Vander Heiden MG, Vidoudez C, Trauger SA, Haigis MC, Scadden DT. Induction of a Timed Metabolic Collapse to Overcome Cancer Chemoresistance. Cell Metab 2020; 32:391-403.e6. PubMed
  • Cabal-Hierro L, van Galen P, Prado MA, Higby KJ, Togami K, Mowery CT, Paulo JA, Xie Y, Cejas P, Furusawa T, Bustin M, Long HW, Sykes DB, Gygi SP, Finley D, Bernstein BE, Lane AA. Chromatin accessibility promotes hematopoietic and leukemia stem cell activity. Nat Commun 2020; 11:1406. PubMed
  • Christodoulou C, Spencer JA, Yeh SA, Turcotte R, Kokkaliaris KD, Panero R, Ramos A, Guo G, Seyedhassantehrani N, Esipova TV, Vinogradov SA, Rudzinskas S, Zhang Y, Perkins AS, Orkin SH, Calogero RA, Schroeder T, Lin CP, Camargo FD. Live-animal imaging of native haematopoietic stem and progenitor cells. Nature 2020; 578:278-283. PubMed
  • Lundin V, Sugden WW, Theodore LN, Sousa PM, Han A, Chou S, Wrighton PJ, Cox AG, Ingber DE, Goessling W, Daley GQ, North TE. YAP Regulates Hematopoietic Stem Cell Formation in Response to the Biomechanical Forces of Blood Flow. Dev Cell 2020; 52:446-460.e5. PubMed
  • Uckelmann HJ, Kim SM, Wong EM, Hatton C, Giovinazzo H, Gadrey JY, Krivtsov AV, Rücker FG, Döhner K, McGeehan GM, Levine RL, Bullinger L, Vassiliou GS, Armstrong SA. Therapeutic targeting of preleukemia cells in a mouse model of mutant acute myeloid leukemia. Science 2020; 367:586-590. PubMed
  • Shah NJ, Najibi AJ, Shih TY, Mao AS, Sharda A, Scadden DT, Mooney DJ. A biomaterial-based vaccine eliciting durable tumour-specific responses against acute myeloid leukaemia. Nat Biomed Eng 2020; 4:40-51. PubMed
  • Myers KC, Furutani E, Weller E, Siegele B, Galvin A, Arsenault V, Alter BP, Boulad F, Bueso-Ramos C, Burroughs L, Castillo P, Connelly J, Davies SM, DiNardo CD, Hanif I, Ho RH, Karras N, Manalang M, McReynolds LJ, Nakano TA, Nalepa G, Norkin M, Oberley MJ, Orgel E, Pastore YD, Rosenthal J, Walkovich K, Larson J, Malsch M, Elghetany MT, Fleming MD, Shimamura A. Clinical features and outcomes of patients with Shwachman-Diamond syndrome and myelodysplastic syndrome or acute myeloid leukaemia: a multicentre, retrospective, cohort study. Lancet Haematol 2019. PubMed
  • Cremer A, Ellegast JM, Alexe G, Frank ES, Ross L, Chu SH, Pikman Y, Robichaud A, Goodale A, Haupl B, Mohr S, Rao AV, Walker A, Blachly JS, Piccioni F, Armstrong SA, Byrd JC, Oellerich T, Stegmaier K. Resistance mechanisms to SYK inhibition in acute myeloid leukemia. 2019. PubMed
  • Brunner AM, Blonquist TM, DeAngelo DJ, McMasters M, Fell G, Hermance NM, Winer ES, Lindsley RC, Hobbs GS, Amrein PC, Hock HR, Steensma DP, Garcia JS, Luskin MR, Stone RM, Ballen KK, Rosenblatt J, Avigan D, Nahas MR, Mendez LM, McAfee SL, Moran JA, Bergeron M, Foster J, Bertoli C, Manning AL, McGregor KL, Fishman KM, Kuo FC, Baltay MT, Macrae M, Burke M, Behnan T, Wey MC, Som TT, Ramos AY, Rae J, Lombardi Story J, Nelson N, Logan E, Connolly C, Neuberg DS, Chen YB, Graubert TA, Fathi AT. Alisertib plus induction chemotherapy in previously untreated patients with high-risk, acute myeloid leukaemia: a single-arm, phase 2 trial. Lancet Haematol 2019. PubMed
  • Krivtsov AV, Evans K, Gadrey JY, Eschle BK, Hatton C, Uckelmann HJ, Ross KN, Perner F, Olsen SN, Pritchard T, McDermott L, Jones CD, Jing D, Braytee A, Chacon D, Earley E, McKeever BM, Claremon D, Gifford AJ, Lee HJ, Teicher BA, Pimanda JE, Beck D, Perry JA, Smith MA, McGeehan GM, Lock RB, Armstrong SA. A Menin-MLL Inhibitor Induces Specific Chromatin Changes and Eradicates Disease in Models of MLL-Rearranged Leukemia. Cancer Cell 2019; 36:660-673.e11. PubMed
  • Pouliot GP, Degar J, Hinze L, Kochupurakkal B, Vo CD, Burns MA, Moreau L, Ganesa C, Roderick J, Peirs S, Menten B, Loh ML, Hunger SP, Silverman LB, Harris MH, Stevenson KE, Weinstock DM, Weng AP, Van Vlierberghe P, D'Andrea AD, Gutierrez A. Fanconi-BRCA pathway mutations in childhood T-cell acute lymphoblastic leukemia. PLoS ONE 2019; 14:e0221288. PubMed
  • DeFilipp Z, Li S, Avigan D, Armand P, Ho VT, Koreth J, Nikiforow S, Alyea EP, Ritz J, Boussiotis V, Rosenblatt J, Brown J, McAfee S, Dey BR, El-Jawahri A, Spitzer TR, Chen YB, Soiffer RJ, Antin JH, Ballen KK, Cutler CS. A phase II study of reduced intensity double umbilical cord blood transplantation using fludarabine, melphalan, and low dose total body irradiation. Bone Marrow Transplant 2019. PubMed
  • Boettcher S, Miller PG, Sharma R, McConkey M, Leventhal M, Krivtsov AV, Giacomelli AO, Wong W, Kim J, Chao S, Kurppa KJ, Yang X, Milenkowic K, Piccioni F, Root DE, Rücker FG, Flamand Y, Neuberg D, Lindsley RC, Jänne PA, Hahn WC, Jacks T, Döhner H, Armstrong SA, Ebert BL. A dominant-negative effect drives selection of missense mutations in myeloid malignancies. Science 2019; 365:599-604. PubMed
  • Baryawno N, Przybylski D, Kowalczyk MS, Kfoury Y, Severe N, Gustafsson K, Kokkaliaris KD, Mercier F, Tabaka M, Hofree M, Dionne D, Papazian A, Lee D, Ashenberg O, Subramanian A, Vaishnav ED, Rozenblatt-Rosen O, Regev A, Scadden DT. A Cellular Taxonomy of the Bone Marrow Stroma in Homeostasis and Leukemia. Cell 2019; 177:1915-1932.e16. PubMed
  • Yan C, Brunson DC, Tang Q, Do D, Iftimia NA, Moore JC, Hayes MN, Welker AM, Garcia EG, Dubash TD, Hong X, Drapkin BJ, Myers DT, Phat S, Volorio A, Marvin DL, Ligorio M, Dershowitz L, McCarthy KM, Karabacak MN, Fletcher JA, Sgroi DC, Iafrate JA, Maheswaran S, Dyson NJ, Haber DA, Rawls JF, Langenau DM. Visualizing Engrafted Human Cancer and Therapy Responses in Immunodeficient Zebrafish. Cell 2019; 177:1903-1914.e14. PubMed
  • Hinze L, Pfirrmann M, Karim S, Degar J, McGuckin C, Vinjamur D, Sacher J, Stevenson KE, Neuberg DS, Orellana E, Stanulla M, Gregory RI, Bauer DE, Wagner FF, Stegmaier K, Gutierrez A. Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Cancer Cell 2019; 35:664-676.e7. PubMed
  • Wong WJ, Baltay M, Getz A, Fuhrman K, Aster JC, Hasserjian RP, Pozdnyakova O. Gene expression profiling distinguishes prefibrotic from overtly fibrotic myeloproliferative neoplasms and identifies disease subsets with distinct inflammatory signatures. PLoS ONE 2019; 14:e0216810. PubMed
  • Mugoni V, Panella R, Cheloni G, Chen M, Pozdnyakova O, Stroopinsky D, Guarnerio J, Monteleone E, Lee JD, Mendez L, Menon AV, Aster JC, Lane AA, Stone RM, Galinsky I, Zamora JC, Lo-Coco F, Bhasin MK, Avigan D, Longo L, Clohessy JG, Pandolfi PP. Vulnerabilities in mIDH2 AML confer sensitivity to APL-like targeted combination therapy. Cell Res 2019; 29:446-459. PubMed
  • Bolaños-Meade J, Reshef R, Fraser R, Fei M, Abhyankar S, Al-Kadhimi Z, Alousi AM, Antin JH, Arai S, Bickett K, Chen YB, Damon LE, Efebera YA, Geller NL, Giralt SA, Hari P, Holtan SG, Horowitz MM, Jacobsohn DA, Jones RJ, Liesveld JL, Logan BR, MacMillan ML, Mielcarek M, Noel P, Pidala J, Porter DL, Pusic I, Sobecks R, Solomon SR, Weisdorf DJ, Wu J, Pasquini MC, Koreth J. Three prophylaxis regimens (tacrolimus, mycophenolate mofetil, and cyclophosphamide; tacrolimus, methotrexate, and bortezomib; or tacrolimus, methotrexate, and maraviroc) versus tacrolimus and methotrexate for prevention of graft-versus-host disease with Lancet Haematol 2019; 6:e132-e143. PubMed
  • Shah NJ, Mao AS, Shih TY, Kerr MD, Sharda A, Raimondo TM, Weaver JC, Vrbanac VD, Deruaz M, Tager AM, Mooney DJ, Scadden DT. An injectable bone marrow-like scaffold enhances T cell immunity after hematopoietic stem cell transplantation. Nat Biotechnol 2019; 37:293-302. PubMed
  • Czechowicz A, Palchaudhuri R, Scheck A, Hu Y, Hoggatt J, Saez B, Pang WW, Mansour MK, Tate TA, Chan YY, Walck E, Wernig G, Shizuru JA, Winau F, Scadden DT, Rossi DJ. Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation. Nat Commun 2019; 10:617. PubMed
  • Mowery CT, Reyes JM, Cabal-Hierro L, Higby KJ, Karlin KL, Wang JH, Kimmerling RJ, Cejas P, Lim K, Li H, Furusawa T, Long HW, Pellman D, Chapuy B, Bustin M, Manalis SR, Westbrook TF, Lin CY, Lane AA. Trisomy of a Down Syndrome Critical Region Globally Amplifies Transcription via HMGN1 Overexpression. Cell Rep 2018; 25:1898-1911.e5. PubMed
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Myeloid Malignancies

SPORE Grants, or Specialized Programs of Research Excellence are awarded by the National Cancer Institute to promote collaborative, interdisciplinary translational cancer research. With about 60 SPORE's nationwide, DF/HCC holds 7 of these grants.

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