Publications

Publications from our lab.

  • Durbin AD, Wang T, Wimalasena VK, Zimmerman MW, Li D, Dharia NV, Mariani L, Shendy NAM, Nance S, Patel AG, Shao Y, Mundada M, Maxham L, Park PMC, Sigua LH, Morita K, Saur Conway A, Robichaud AL, Perez-Atayde AR, Bikowitz MJ, Quinn TR, Wiest OG, Easton J, Schonbrunn E, Bulyk ML, Abraham BJ, Stegmaier K, Look AT, Qi J. EP300 selectively controls the enhancer landscape of MYCN-amplified neuroblastoma. Cancer Discov. 2021 Nov 12. 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. Cancer Discov. 2021 Sep 16. PubMed
  • Pikman Y, Ocasio-Martinez N, Alexe G, Dimitrov B, Kitara S, Diehl FF, Robichaud AL, Conway AS, Ross L, Su A, Ling F, Qi J, Roti G, Lewis CA, Puissant A, Vander Heiden MG, Stegmaier K. Targeting serine hydroxymethyltransferases 1 and 2 for T-cell acute lymphoblastic leukemia therapy. Leukemia. 2021 Aug 02. PubMed
  • Seong BKA, Dharia NV, Lin S, Donovan KA, Chong S, Robichaud A, Conway A, Hamze A, Ross L, Alexe G, Adane B, Nabet B, Ferguson FM, Stolte B, Wang EJ, Sun J, Darzacq X, Piccioni F, Gray NS, Fischer ES, Stegmaier K. TRIM8 modulates the EWS/FLI oncoprotein to promote survival in Ewing sarcoma. Cancer Cell. 2021 Jul 22. PubMed
  • Neggers JE, Paolella BR, Asfaw A, Rothberg MV, Skipper TA, Yang A, Kalekar RL, Krill-Burger JM, Dharia NV, Kugener G, Kalfon J, Yuan C, Dumont N, Gonzalez A, Abdusamad M, Li YY, Spurr LF, Wu WW, Durbin AD, Wolpin BM, Piccioni F, Root DE, Boehm JS, Cherniack AD, Tsherniak A, Hong AL, Hahn WC, Stegmaier K, Golub TR, Vazquez F, Aguirre AJ. Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Cell Rep. 2021 Jul 13; 36(2):109367. PubMed
  • Adane B, Alexe G, Seong BKA, Lu D, Hwang EE, Hnisz D, Lareau CA, Ross L, Lin S, Dela Cruz FS, Richardson M, Weintraub AS, Wang S, Iniguez AB, Dharia NV, Conway AS, Robichaud AL, Tanenbaum B, Krill-Burger JM, Vazquez F, Schenone M, Berman JN, Kung AL, Carr SA, Aryee MJ, Young RA, Crompton BD, Stegmaier K. STAG2 loss rewires oncogenic and developmental programs to promote metastasis in Ewing sarcoma. Cancer Cell. 2021 Jun 14; 39(6):827-844.e10. PubMed
  • Gillani R, Seong BKA, Crowdis J, Conway JR, Dharia NV, Alimohamed S, Haas BJ, Han K, Park J, Dietlein F, He MX, Imamovic A, Ma C, Bassik MC, Boehm JS, Vazquez F, Gusev A, Liu D, Janeway KA, McFarland JM, Stegmaier K, Van Allen EM. Gene Fusions Create Partner and Collateral Dependencies Essential to Cancer Cell Survival. Cancer Res. 2021 Aug 01; 81(15):3971-3984. PubMed
  • Yi JS, Sias-Garcia O, Nasholm N, Hu X, Iniguez AB, Hall MD, Davis M, Guha R, Moreno-Smith M, Barbieri E, Duong K, Koach J, Qi J, Bradner JE, Stegmaier K, Weiss WA, Gustafson WC. The synergy of BET inhibitors with aurora A kinase inhibitors in MYCN-amplified neuroblastoma is heightened with functional TP53. Neoplasia. 2021 06; 23(6):624-633. PubMed
  • Malone CF, Dharia NV, Kugener G, Forman AB, Rothberg MV, Abdusamad M, Gonzalez A, Kuljanin M, Robichaud AL, Saur Conway A, Dempster JM, Paolella BR, Dumont N, Hovestadt V, Mancias JD, Younger ST, Root DE, Golub TR, Vazquez F, Stegmaier K. Selective modulation of a pan-essential protein as a therapeutic strategy in cancer. Cancer Discov. 2021 Apr 21. PubMed
  • Jiang B, Jiang J, Kaltheuner IH, Iniguez AB, Anand K, Ferguson FM, Ficarro SB, Seong BKA, Greifenberg AK, Dust S, Kwiatkowski NP, Marto JA, Stegmaier K, Zhang T, Geyer M, Gray NS. Structure-activity relationship study of THZ531 derivatives enables the discovery of BSJ-01-175 as a dual CDK12/13 covalent inhibitor with efficacy in Ewing sarcoma. Eur J Med Chem. 2021 Oct 05; 221:113481. 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 03 31; 13(587). PubMed
  • Su XA, Ma D, Parsons JV, Replogle JM, Amatruda JF, Whittaker CA, Stegmaier K, Amon A. RAD21 is a driver of chromosome 8 gain in Ewing sarcoma to mitigate replication stress. Genes Dev. 2021 Mar 25. PubMed
  • Dharia NV, Kugener G, Guenther LM, Malone CF, Durbin AD, Hong AL, Howard TP, Bandopadhayay P, Wechsler CS, Fung I, Warren AC, Dempster JM, Krill-Burger JM, Paolella BR, Moh P, Jha N, Tang A, Montgomery P, Boehm JS, Hahn WC, Roberts CWM, McFarland JM, Tsherniak A, Golub TR, Vazquez F, Stegmaier K. A first-generation pediatric cancer dependency map. Nat Genet. 2021 Apr; 53(4):529-538. PubMed
  • Pearson AD, DuBois SG, Buenger V, Kieran M, Stegmaier K, Bandopadhayay P, Bennett K, Bourdeaut F, Brown PA, Chesler L, Clymer J, Fox E, French CA, Germovsek E, Giles FJ, Bender JG, Hattersley MM, Ludwinski D, Luptakova K, Maris J, McDonough J, Nikolova Z, Smith M, Tsiatis AC, Vibhakar R, Weiner S, Yi JS, Zheng F, Vassal G. Bromodomain and extra-terminal inhibitors-A consensus prioritisation after the Paediatric Strategy Forum for medicinal product development of epigenetic modifiers in children-ACCELERATE. Eur J Cancer. 2021 Mar; 146:115-124. 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. Cancer Discov. 2021 Feb 09. PubMed
  • Neggers JE, Paolella BR, Asfaw A, Rothberg MV, Skipper TA, Yang A, Kalekar RL, Krill-Burger JM, Dharia NV, Kugener G, Kalfon J, Yuan C, Dumont N, Gonzalez A, Abdusamad M, Li YY, Spurr LF, Wu WW, Durbin AD, Wolpin BM, Piccioni F, Root DE, Boehm JS, Cherniack AD, Tsherniak A, Hong AL, Hahn WC, Stegmaier K, Golub TR, Vazquez F, Aguirre AJ. Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Cell Rep. 2020 Dec 15; 33(11):108493. PubMed
  • Pearson AD, Stegmaier K, Bourdeaut F, Reaman G, Heenen D, Meyers ML, Armstrong SA, Brown P, De Carvalho D, Jabado N, Marshall L, Rivera M, Smith M, Adamson PC, Barone A, Baumann C, Blackman S, Buenger V, Donoghue M, Duncan AD, Fox E, Gadbaw B, Hattersley M, Ho P, Jacobs I, Kelly MJ, Kieran M, Lesa G, Ligas F, Ludwinski D, McDonough J, Nikolova Z, Norga K, Senderowicz A, Taube T, Weiner S, Karres D, Vassal G. Paediatric Strategy Forum for medicinal product development of epigenetic modifiers for children: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2020 11; 139:135-148. PubMed
  • Nabet B, Ferguson FM, Seong BKA, Kuljanin M, Leggett AL, Mohardt ML, Robichaud A, Conway AS, Buckley DL, Mancias JD, Bradner JE, Stegmaier K, Gray NS. Rapid and direct control of target protein levels with VHL-recruiting dTAG molecules. Nat Commun. 2020 09 18; 11(1):4687. PubMed
  • Su A, Ling F, Vaganay C, Sodaro G, Benaksas C, Dal Bello R, Forget A, Pardieu B, Lin KH, Rutter JC, Bassil CF, Fortin G, Pasanisi J, Antony-Debré I, Alexe G, Benoist JF, Pruvost A, Pikman Y, Qi J, Schlageter MH, Micol JB, Roti G, Cluzeau T, Dombret H, Preudhomme C, Fenouille N, Benajiba L, Golan HM, Stegmaier K, Lobry C, Wood KC, Itzykson R, Puissant A. The Folate Cycle Enzyme MTHFR Is a Critical Regulator of Cell Response to MYC-Targeting Therapies. Cancer Discov. 2020 Dec; 10(12):1894-1911. PubMed
  • Marchesini M, Gherli A, Montanaro A, Patrizi L, Sorrentino C, Pagliaro L, Rompietti C, Kitara S, Heit S, Olesen CE, Møller JV, Savi M, Bocchi L, Vilella R, Rizzi F, Baglione M, Rastelli G, Loiacono C, La Starza R, Mecucci C, Stegmaier K, Aversa F, Stilli D, Lund Winther AM, Sportoletti P, Bublitz M, Dalby-Brown W, Roti G. Blockade of Oncogenic NOTCH1 With the SERCA Inhibitor CAD204520 in T Cell Acute Lymphoblastic Leukemia. Cell Chem Biol. 2020 Apr 15:S2451-9456(20)30116-1. PubMed
  • Pikman Y, Conway AS, Robichaud AL, Kitara S, Church AJ, Kennedy AL, Silverman LB, Billett AL, Weinstock DM, Harris MH, Stegmaier K. Targeting EZH2 for the Treatment of Hepatosplenic T-cell Lymphoma. Blood Adv. 2020 Apr 14;4(7):1265-1269. PubMed
  • Schauer NJ, Liu X, Magin RS, Doherty LM, Chan WC, Ficarro SB, Hu W, Roberts RM, Iacob RE, Stolte B, Giacomelli AO, Perera S, McKay K, Boswell SA, Weisberg EL, Ray A, Chauhan D, Dhe-Paganon S, Anderson KC, Griffin JD, Li J, Hahn WC, Sorger PK, Engen JR, Stegmaier K, Marto JA, Buhrlage SJ. Selective USP7 Inhibition Elicits Cancer Cell Killing Through a p53-dependent Mechanism. Sci Rep. 2020 Mar 24;10(1):5324. PubMed
  • Cremer A, Stegmaier K. Targeting DUBs to Degrade Oncogenic Proteins. Br J Cancer. 2020 Apr;122(8):1121-1123. PubMed
  • Elgamal OA, Mehmood A, Jeon JY, Carmichael B, Lehman A, Orwick SJ, Truxall J, Goettl VM, Wasmuth R, Tran M, Mitchell S, Lapalombella R, Eathiraj S, Schwartz B, Stegmaier K, Baker SD, Hertlein E, Byrd JC. Preclinical Efficacy for a Novel Tyrosine Kinase Inhibitor, ArQule 531 Against Acute Myeloid Leukemia. J Hematol Oncol. 2020 Jan 28;13(1):8. PubMed
  • Cremer A, Ellegast JM, Alexe G, Frank ES, Ross L, Chu SH, Pikman Y, Robichaud A, Goodale A, Häupl B, Mohr S, Rao AV, Walker AR, Blachly JS, Piccioni F, Armstrong SA, Byrd JC, Oellerich T, Stegmaier K. Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia. Cancer Discov. 2020 Feb;10(2):214-231. PubMed
  • Tarumoto Y, Lin S, Wang J, Milazzo JP, Xu Y, Lu B, Yang Z, Wei Y, Polyanskaya S, Wunderlich M, Gray NS, Stegmaier K, Vakoc CR. Salt-inducible kinase inhibition suppresses acute myeloid leukemia progression in vivo. Blood. 2020 Jan 2;135(1):56-70. PubMed
  • Oberlick EM, Rees MG, Seashore-Ludlow B, Vazquez F, Nelson GM, Dharia NV, Weir BA, Tsherniak A, Ghandi M, Krill-Burger JM, Meyers RM, Wang X, Montgomery P, Root DE, Bieber JM, Radko S, Cheah JH, Hon CS, Shamji AF, Clemons PA, Park PJ, Dyer MA, Golub TR, Stegmaier K, Hahn WC, Stewart EA, Schreiber SL, Roberts CWM. Small-Molecule and CRISPR Screening Converge to Reveal Receptor Tyrosine Kinase Dependencies in Pediatric Rhabdoid Tumors. Cell Rep. 2019 Aug 27; 28(9):2331-2344.e8. PubMed
  • Selvanathan SP, Graham GT, Grego AR, Baker TM, Hogg JR, Simpson M, Batish M, Crompton B, Stegmaier K, Tomazou EM, Kovar H, Üren A, Toretsky JA. EWS-FLI1 modulated alternative splicing of ARID1A reveals novel oncogenic function through the BAF complex. Nucleic Acids Res. 2019 Aug 08. PubMed
  • Hanlon K, Thompson A, Pantano L, Hutchinson JN, Al-Obeidi A, Wang S, Bliss-Moreau M, Helble J, Alexe G, Stegmaier K, Bauer DE, Croker BA. Single-cell cloning of human T-cell lines reveals clonal variation in cell death responses to chemotherapeutics. Cancer Genet. 2019 Sep; 237:69-77. PubMed
  • Bandopadhayay P, Piccioni F, O'Rourke R, Ho P, Gonzalez EM, Buchan G, Qian K, Gionet G, Girard E, Coxon M, Rees MG, Brenan L, Dubois F, Shapira O, Greenwald NF, Pages M, Balboni Iniguez A, Paolella BR, Meng A, Sinai C, Roti G, Dharia NV, Creech A, Tanenbaum B, Khadka P, Tracy A, Tiv HL, Hong AL, Coy S, Rashid R, Lin JR, Cowley GS, Lam FC, Goodale A, Lee Y, Schoolcraft K, Vazquez F, Hahn WC, Tsherniak A, Bradner JE, Yaffe MB, Milde T, Pfister SM, Qi J, Schenone M, Carr SA, Ligon KL, Kieran MW, Santagata S, Olson JM, Gokhale PC, Jaffe JD, Root DE, Stegmaier K, Johannessen CM, Beroukhim R. Neuronal differentiation and cell-cycle programs mediate response to BET-bromodomain inhibition in MYC-driven medulloblastoma. Nat Commun. 2019 06 03; 10(1):2400. PubMed
  • Brien GL, Stegmaier K, Armstrong SA. Targeting chromatin complexes in fusion protein-driven malignancies. Nat Rev Cancer. 2019 05; 19(5):255-269. PubMed
  • Ruiz-Gutierrez M, Bölükbasi ÖV, Alexe G, Kotini AG, Ballotti K, Joyce CE, Russell DW, Stegmaier K, Myers K, Novina CD, Papapetrou EP, Shimamura A. Therapeutic discovery for marrow failure with MDS predisposition using pluripotent stem cells. JCI Insight. 2019 Apr 30; 5. 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 Apr 15; 35(4):664-676.e7. PubMed
  • Wang S, Hwang EE, Guha R, O'Neill AF, Melong N, Veinotte CJ, Conway Saur A, Wuerthele K, Shen M, McKnight C, Alexe G, Lemieux ME, Wang A, Hughes E, Xu X, Boxer MB, Hall MD, Kung A, Berman JN, Davis MI, Stegmaier K, Crompton BD. High-throughput Chemical Screening Identifies Focal Adhesion Kinase and Aurora Kinase B Inhibition as a Synergistic Treatment Combination in Ewing Sarcoma. Clin Cancer Res. 2019 Jul 15; 25(14):4552-4566. PubMed
  • DuBois SG, Corson LB, Stegmaier K, Janeway KA. Ushering in the next generation of precision trials for pediatric cancer. Science. 2019 Mar 15; 363(6432):1175-1181. PubMed
  • Bensinger D, Stubba D, Cremer A, Kohl V, Waßmer T, Stuckert J, Engemann V, Stegmaier K, Schmitz K, Schmidt B. Virtual Screening Identifies Irreversible FMS-like Tyrosine Kinase 3 Inhibitors with Activity toward Resistance-Conferring Mutations. J Med Chem. 2019 Mar 14; 62(5):2428-2446. PubMed
  • Howard TP, Arnoff TE, Song MR, Giacomelli AO, Wang X, Hong AL, Dharia NV, Wang S, Vazquez F, Pham MT, Morgan AM, Wachter F, Bird GH, Kugener G, Oberlick EM, Rees MG, Tiv HL, Hwang JH, Walsh KH, Cook A, Krill-Burger JM, Tsherniak A, Gokhale PC, Park PJ, Stegmaier K, Walensky LD, Hahn WC, Roberts CWM. MDM2 and MDM4 Are Therapeutic Vulnerabilities in Malignant Rhabdoid Tumors. Cancer Res. 2019 May 01; 79(9):2404-2414. PubMed
  • Liu S, Marneth AE, Alexe G, Walker SR, Gandler HI, Ye DQ, Labella K, Mathur R, Toniolo PA, Tillgren M, Gokhale PC, Barbie D, Mullally A, Stegmaier K, Frank DA. The kinases IKBKE and TBK1 regulate MYC-dependent survival pathways through YB-1 in AML and are targets for therapy. Blood Adv. 2018 12 11; 2(23):3428-3442. PubMed
  • Iniguez AB, Alexe G, Wang EJ, Roti G, Patel S, Chen L, Kitara S, Conway A, Robichaud AL, Stolte B, Bandopadhayay P, Goodale A, Pantel S, Lee Y, Cheff DM, Hall MD, Guha R, Davis MI, Menard M, Nasholm N, Weiss WA, Qi J, Beroukhim R, Piccioni F, Johannessen C, Stegmaier K. Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling. Cancer Cell. 2018 12 10; 34(6):922-938.e7. PubMed
  • Guenther LM, Dharia NV, Ross L, Conway A, Robichaud AL, Catlett JL, Wechsler CS, Frank ES, Goodale A, Church AJ, Tseng YY, Guha R, McKnight CG, Janeway KA, Boehm JS, Mora J, Davis MI, Alexe G, Piccioni F, Stegmaier K. A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma. Clin Cancer Res. 2019 Feb 15; 25(4):1343-1357. PubMed
  • Benajiba L, Alexe G, Su A, Raffoux E, Soulier J, Hemann MT, Hermine O, Itzykson R, Stegmaier K, Puissant A. Creatine kinase pathway inhibition alters GSK3 and WNT signaling in EVI1-positive AML. Leukemia. 2019 03; 33(3):800-804. PubMed
  • Witwicki RM, Ekram MB, Qiu X, Janiszewska M, Shu S, Kwon M, Trinh A, Frias E, Ramadan N, Hoffman G, Yu K, Xie Y, McAllister G, McDonald R, Golji J, Schlabach M, deWeck A, Keen N, Chan HM, Ruddy D, Rejtar T, Sovath S, Silver S, Sellers WR, Jagani Z, Hogarty MD, Roberts C, Brown M, Stegmaier K, Long H, Shivdasani RA, Pellman D, Polyak K. TRPS1 Is a Lineage-Specific Transcriptional Dependency in Breast Cancer. Cell Rep. 2018 10 30; 25(5):1255-1267.e5. PubMed
  • Guerra RM, Bird GH, Harvey EP, Dharia NV, Korshavn KJ, Prew MS, Stegmaier K, Walensky LD. Precision Targeting of BFL-1/A1 and an ATM Co-dependency in Human Cancer. Cell Rep. 2018 09 25; 24(13):3393-3403.e5. PubMed
  • Bohnenberger H, Kaderali L, Ströbel P, Yepes D, Plessmann U, Dharia NV, Yao S, Heydt C, Merkelbach-Bruse S, Emmert A, Hoffmann J, Bodemeyer J, Reuter-Jessen K, Lois AM, Dröge LH, Baumeister P, Walz C, Biggemann L, Walter R, Häupl B, Comoglio F, Pan KT, Scheich S, Lenz C, Küffer S, Bremmer F, Kitz J, Sitte M, Beißbarth T, Hinterthaner M, Sebastian M, Lotz J, Schildhaus HU, Wolff H, Danner BC, Brandts C, Büttner R, Canis M, Stegmaier K, Serve H, Urlaub H, Oellerich T. Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. EMBO Mol Med. 2018 09; 10(9). PubMed
  • Shulman DS, Klega K, Imamovic-Tuco A, Clapp A, Nag A, Thorner AR, Van Allen E, Ha G, Lessnick SL, Gorlick R, Janeway KA, Leavey PJ, Mascarenhas L, London WB, Vo KT, Stegmaier K, Hall D, Krailo MD, Barkauskas DA, DuBois SG, Crompton BD. Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. Br J Cancer. 2018 08; 119(5):615-621. PubMed
  • Durbin AD, Zimmerman MW, Dharia NV, Abraham BJ, Iniguez AB, Weichert-Leahey N, He S, Krill-Burger JM, Root DE, Vazquez F, Tsherniak A, Hahn WC, Golub TR, Young RA, Look AT, Stegmaier K. Selective gene dependencies in MYCN-amplified neuroblastoma include the core transcriptional regulatory circuitry. Nat Genet. 2018 09; 50(9):1240-1246. PubMed
  • Pikman Y, Stegmaier K. Targeted therapy for fusion-driven high-risk acute leukemia. Blood. 2018 Jul 26. pii: blood-2018-04-784157. PubMed
  • Stolte B, Iniguez AB, Dharia NV, Robichaud AL, Conway AS, Morgan AM, Alexe G, Schauer NJ, Liu X, Bird GH, Tsherniak A, Vazquez F, Buhrlage SJ, Walensky LD, Stegmaier K. Genome-scale CRISPR-Cas9 screen identifies druggable dependencies in TP53 wild-type Ewing sarcoma. J Exp Med. 2018 Aug 6;215(8):2137-2155. PubMed
  • Zhang C, D'Alessandro A, Wellendorf AM, Mohmoud F, Serrano-Lopez J, Perentesis JP, Komurov K, Alexe G, Stegmaier K, Whitsett JA, Grimes HL, Cancelas JA. KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. Oncotarget. 2018 Jul 3;9(51):29665-29679. PubMed
  • Place AE, Pikman Y, Stevenson KE, Harris MH, Pauly M, Sulis ML, Hijiya N, Gore L, Cooper TM, Loh ML, Roti G, Neuberg DS, Hunt SK, Orloff-Parry S, Stegmaier K, Sallan SE, Silverman LB. Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. Pediatr Blood Cancer. 2018 Mar 30:e27062. PubMed
  • Wagner FF, Benajiba L, Campbell AJ, Weïwer M, Sacher JR, Gale JP, Ross L, Puissant A, Alexe G, Conway A, Back M, Pikman Y, Galinsky I, DeAngelo DJ, Stone RM, Kaya T, Shi X, Robers MB, Machleidt T, Wilkinson J, Hermine O, Kung A, Stein AJ, Lakshminarasimhan D, Hemann MT, Scolnick E, Zhang YL, Pan JQ, Stegmaier K, Holson EB. Exploiting an Asp-Glu "switch" in glycogen synthase kinase 3 to design paralog-selective inhibitors for use in acute myeloid leukemia. Sci Transl Med. 2018 Mar 07; 10(431). PubMed
  • Gorthi A, Romero JC, Loranc E, Cao L, Lawrence LA, Goodale E, Iniguez AB, Bernard X, Masamsetti VP, Roston S, Lawlor ER, Toretsky JA, Stegmaier K, Lessnick SL, Chen Y, Bishop AJR. EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 Mar 15; 555(7696):387-391. PubMed
  • Baldoni S, Del Papa B, Dorillo E, Aureli P, De Falco F, Rompietti C, Sorcini D, Varasano E, Cecchini D, Zei T, Di Tommaso A, Rosati E, Alexe G, Roti G, Stegmaier K, Di Ianni M, Falzetti F, Sportoletti P. Bepridil exhibits anti-leukemic activity associated with NOTCH1 pathway inhibition in chronic lymphocytic leukemia. Int J Cancer. 2018 Mar 06. PubMed
  • Iniguez AB, Stolte B, Wang EJ, Conway AS, Alexe G, Dharia NV, Kwiatkowski N, Zhang T, Abraham BJ, Mora J, Kalev P, Leggett A, Chowdhury D, Benes CH, Young RA, Gray NS, Stegmaier K. EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. Cancer Cell. 2018 Feb 12; 33(2):202-216.e6. PubMed
  • Chen L, Alexe G, Dharia NV, Ross L, Iniguez AB, Conway AS, Wang EJ, Veschi V, Lam N, Qi J, Gustafson WC, Nasholm N, Vazquez F, Weir BA, Cowley GS, Ali LD, Pantel S, Jiang G, Harrington WF, Lee Y, Goodale A, Lubonja R, Krill-Burger JM, Meyers RM, Tsherniak A, Root DE, Bradner JE, Golub TR, Roberts CW, Hahn WC, Weiss WA, Thiele CJ, Stegmaier K. CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. J Clin Invest. 2018 Jan 02; 128(1):446-462. PubMed
  • Roti G, Qi J, Kitara S, Sanchez-Martin M, Saur Conway A, Varca AC, Su A, Wu L, Kung AL, Ferrando AA, Bradner JE, Stegmaier K. Leukemia-specific delivery of mutant NOTCH1 targeted therapy. J Exp Med. 2018 Jan 02; 215(1):197-216. PubMed
  • Meyers RM, Bryan JG, McFarland JM, Weir BA, Sizemore AE, Xu H, Dharia NV, Montgomery PG, Cowley GS, Pantel S, Goodale A, Lee Y, Ali LD, Jiang G, Lubonja R, Harrington WF, Strickland M, Wu T, Hawes DC, Zhivich VA, Wyatt MR, Kalani Z, Chang JJ, Okamoto M, Stegmaier K, Golub TR, Boehm JS, Vazquez F, Root DE, Hahn WC, Tsherniak A. Computational correction of copy number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells. Nat Genet. 2017 Dec;49(12):1779-1784. PubMed
  • LaRochelle JR, Fodor M, Ellegast JM, Liu X, Vemulapalli V, Mohseni M, Stams T, Buhrlage SJ, Stegmaier K, LaMarche MJ, Acker MG, Blacklow SC. Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. Bioorg Med Chem. 2017 Dec 15;25(24):6479-6485. PubMed
  • Lobbardi R, Pinder J, Martinez-Pastor B, Theodorou M, Blackburn JS, Abraham BJ, Namiki Y, Mansour M, Abdelfattah NS, Molodtsov A, Alexe G, Toiber D, de Waard M, Jain E, Boukhali M, Lion M, Bhere D, Shah K, Gutierrez A, Stegmaier K, Silverman LB, Sadreyev RI, Asara JM, Oettinger MA, Haas W, Look AT, Young RA, Mostoslavsky R, Dellaire G, Langenau DM. TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. Cancer Discov. 2017 Nov; 7(11):1336-1353. PubMed
  • Malone CF, Stegmaier K. Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. Cancer Cell. 2017 09 11; 32(3):271-273. PubMed
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  • Harris MH, DuBois SG, Glade Bender JL, Kim A, Crompton BD, Parker E, Dumont IP, Hong AL, Guo D, Church A, Stegmaier K, Roberts CW, Shusterman S, London WB, MacConaill LE, Lindeman NI, Diller L, Rodriguez-Galindo C, Janeway KA. Multicenter Feasibility Study of Tumor Molecular Profiling to Inform Therapeutic Decisions in Advanced Pediatric Solid Tumors: The Individualized Cancer Therapy (iCat) Study. JAMA Oncol 2016. PubMed
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  • Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen SR, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber TA, Golub TR, Stegmaier K, Loh ML. The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet 2015; 47:1326-33. PubMed
  • Kennedy AL, Vallurupalli M, Chen L, Crompton B, Cowley G, Vazquez F, Weir BA, Tsherniak A, Parasuraman S, Kim S, Alexe G, Stegmaier K. Functional, chemical genomic, and super-enhancer screening identify sensitivity to cyclin D1/CDK4 pathway inhibition in Ewing sarcoma. 2015. PubMed
  • Deveau AP, Forrester AM, Coombs AJ, Wagner GS, Grabher C, Chute IC, Léger D, Mingay M, Alexe G, Rajan V, Liwski R, Hirst M, Steigmaier K, Lewis SM, Look AT, Berman JN. Epigenetic therapy restores normal hematopoiesis in a zebrafish model of NUP98-HOXA9-induced myeloid disease. Leukemia 2015. PubMed
  • Boros K, Puissant A, Back M, Alexe G, Bassil CF, Sinha P, Tholouli E, Stegmaier K, Byers RJ, Rodig SJ. Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. 2015; 6:25575-87. PubMed
  • Marques Howarth M, Simpson D, Ngok SP, Nieves B, Chen R, Siprashvili Z, Vaka D, Breese MR, Crompton BD, Alexe G, Hawkins DS, Jacobson D, Brunner AL, West R, Mora J, Stegmaier K, Khavari P, Sweet-Cordero EA. Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. J Clin Invest 2014; 124:5275-90. PubMed
  • Crompton BD, Stewart C, Taylor-Weiner A, Alexe G, Kurek KC, Calicchio ML, Kiezun A, Carter SL, Shukla SA, Mehta SS, Thorner AR, de Torres C, Lavarino C, Suñol M, McKenna A, Sivachenko A, Cibulskis K, Lawrence MS, Stojanov P, Rosenberg M, Ambrogio L, Auclair D, Seepo S, Blumenstiel B, DeFelice M, Imaz-Rosshandler I, Schwarz-Cruz Y Celis A, Rivera MN, Rodriguez-Galindo C, Fleming MD, Golub TR, Getz G, Mora J, Stegmaier K. The genomic landscape of pediatric Ewing sarcoma. 2014. PubMed
  • Moore NF, Azarova AM, Bhatnagar N, Ross KN, Drake LE, Frumm S, Liu QS, Christie AL, Sanda T, Chesler L, Kung AL, Gray NS, Stegmaier K, George RE. Molecular rationale for the use of PI3K/AKT/mTOR pathway inhibitors in combination with crizotinib in ALK-mutated neuroblastoma. 2014; 5:8737-49. PubMed
  • Lane AA, Chapuy B, Lin CY, Tivey T, Li H, Townsend EC, van Bodegom D, Day TA, Wu SC, Liu H, Yoda A, Alexe G, Schinzel AC, Sullivan TJ, Malinge S, Taylor JE, Stegmaier K, Jaffe JD, Bustin M, te Kronnie G, Izraeli S, Harris MH, Stevenson KE, Neuberg D, Silverman LB, Sallan SE, Bradner JE, Hahn WC, Crispino JD, Pellman D, Weinstock DM. Triplication of a 21q22 region contributes to B cell transformation through HMGN1 overexpression and loss of histone H3 Lys27 trimethylation. Nat Genet 2014. PubMed
  • Stegmaier K. Targeting Csnk1a1 in leukemia. J Exp Med 2014; 211:594. PubMed
  • Deangelo DJ, Neuberg D, Amrein PC, Berchuck J, Wadleigh M, Sirulnik LA, Galinsky I, Golub T, Stegmaier K, Stone RM. A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. Leuk Res 2014; 38:430-4. PubMed
  • Knoechel B, Roderick JE, Williamson KE, Zhu J, Lohr JG, Cotton MJ, Gillespie SM, Fernandez D, Ku M, Wang H, Piccioni F, Silver SJ, Jain M, Pearson D, Kluk MJ, Ott CJ, Shultz LD, Brehm MA, Greiner DL, Gutierrez A, Stegmaier K, Kung AL, Root DE, Bradner JE, Aster JC, Kelliher MA, Bernstein BE. An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. Nat Genet 2014; 46:364-70. PubMed
  • Puissant A, Fenouille N, Alexe G, Pikman Y, Bassil CF, Mehta S, Du J, Kazi JU, Luciano F, Rönnstrand L, Kung AL, Aster JC, Galinsky I, Stone RM, DeAngelo DJ, Hemann MT, Stegmaier K. SYK is a critical regulator of FLT3 in acute myeloid leukemia. Cancer Cell 2014; 25:226-42. PubMed
  • Liu S, Yin L, Stroopinsky D, Rajabi H, Puissant A, Stegmaier K, Avigan D, Kharbanda S, Kufe D, Stone R. MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. Blood 2014; 123:734-42. PubMed
  • Lawrence MS, Stojanov P, Polak P, Kryukov GV, Cibulskis K, Sivachenko A, Carter SL, Stewart C, Mermel CH, Roberts SA, Kiezun A, Hammerman PS, McKenna A, Drier Y, Zou L, Ramos AH, Pugh TJ, Stransky N, Helman E, Kim J, Sougnez C, Ambrogio L, Nickerson E, Shefler E, Cortés ML, Auclair D, Saksena G, Voet D, Noble M, DiCara D, Lin P, Lichtenstein L, Heiman DI, Fennell T, Imielinski M, Hernandez B, Hodis E, Baca S, Dulak AM, Lohr J, Landau DA, Wu CJ, Melendez-Zajgla J, Hidalgo-Miranda A, Koren A, McCarroll SA, Mora J, Lee RS, Crompton B, Onofrio R, Parkin M, Winckler W, Ardlie K, Gabriel SB, Roberts CW, Biegel JA, Stegmaier K, Bass AJ, Garraway LA, Meyerson M, Golub TR, Gordenin DA, Sunyaev S, Lander ES, Getz G. Mutational heterogeneity in cancer and the search for new cancer-associated genes. Nature 2013; 499:214-8. PubMed
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  • Crompton BD, Carlton AL, Thorner AR, Christie AL, Du J, Calicchio ML, Rivera MN, Fleming MD, Kohl NE, Kung AL, Stegmaier K. High-throughput tyrosine kinase activity profiling identifies FAK as a candidate therapeutic target in Ewing sarcoma. Cancer Res 2013. PubMed
  • Roti G, Carlton A, Ross KN, Markstein M, Pajcini K, Su AH, Perrimon N, Pear WS, Kung AL, Blacklow SC, Aster JC, Stegmaier K. Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. Cancer Cell 2013; 23:390-405. PubMed
  • Puissant A, Frumm SM, Alexe G, Bassil CF, Qi J, Chanthery YH, Nekritz EA, Zeid R, Gustafson WC, Greninger P, Garnett MJ, McDermott U, Benes CH, Kung AL, Weiss WA, Bradner JE, Stegmaier K. Targeting MYCN in neuroblastoma by BET bromodomain inhibition. 2013; 3:308-23. PubMed
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  • Aste-Amézaga M, Zhang N, Lineberger JE, Arnold BA, Toner TJ, Gu M, Huang L, Vitelli S, Vo KT, Haytko P, Zhao JZ, Baleydier F, L'Heureux S, Wang H, Gordon WR, Thoryk E, Andrawes MB, Tiyanont K, Stegmaier K, Roti G, Ross KN, Franlin LL, Wang H, Wang F, Chastain M, Bett AJ, Audoly LP, Aster JC, Blacklow SC, Huber HE. Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. PLoS ONE 2010; 5:e9094. PubMed
  • Hahn CK, Berchuck JE, Ross KN, Kakoza RM, Clauser K, Schinzel AC, Ross L, Galinsky I, Davis TN, Silver SJ, Root DE, Stone RM, DeAngelo DJ, Carroll M, Hahn WC, Carr SA, Golub TR, Kung AL, Stegmaier K. Proteomic and genetic approaches identify Syk as an AML target. Cancer Cell 2009; 16:281-94. PubMed
  • Corsello SM,Roti G,Ross KN,Chow KT,Galinsky I,Deangelo DJ,Stone RM,Kung AL,Golub TR,Stegmaier K. Identification of AML1-ETO modulators by chemical genomics. Blood 2009; 113:6193-205. PubMed
  • DuBois SG,Krailo MD,Lessnick SL,Smith R,Chen Z,Marina N,Grier HE,Stegmaier K. Phase II study of intermediate-dose cytarabine in patients with relapsed or refractory Ewing sarcoma: a report from the Children's Oncology Group. Pediatr Blood Cancer 2008; 52:324-7. PubMed
  • Hahn CK,Ross KN,Warrington IM,Mazitschek R,Kanegai CM,Wright RD,Kung AL,Golub TR,Stegmaier K. Expression-based screening identifies the combination of histone deacetylase inhibitors and retinoids for neuroblastoma differentiation. Proc Natl Acad Sci U S A 2008; 105:9751-6. PubMed
  • Haining WN,Angelosanto J,Brosnahan K,Ross K,Hahn C,Russell K,Drury L,Norton S,Nadler L,Stegmaier K. High-throughput gene expression profiling of memory differentiation in primary human T cells. BMC Immunol 2008; 9:44. PubMed
  • Hieronymus H, Lamb J, Ross KN, Peng XP, Clement C, Rodina A, Nieto M, Du J, Stegmaier K, Raj SM, Maloney KN, Clardy J, Hahn WC, Chiosis G, Golub TR. Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. Cancer Cell 2006; 10:321-30. PubMed
  • Peck D, Crawford ED, Ross KN, Stegmaier K, Golub TR, Lamb J. A Method for High-Throughput Gene Expression Signature Analysis. Genome Biol 2006; 7:R61. PubMed
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