Publications

Project Publications

Werner J, Lee AG, Zhang C, Abelson S, Xirenayi S, Rivera J, Yousuf K, Shin H, Patiño-Escobar B, Bachl S, Mandal K, Barpanda A, Ramos E, Izgutdina A, Chaudhuri S, Temple WC, Bhatnagar S, Dardis JK, Meyer J, Morales C, Meshinchi S, Loh ML, Braun B, Tasian SK, Wiita AP, Stieglitz E. Cellular immunotherapy targeting CLL-1 for juvenile myelomonocytic leukemia. Nat Commun. 2025 Apr 23;16(1):3804. doi: 10.1038/s41467-025-59040-6. PMCID: PMC12019388.

Decker M, Huang BJ, Ware T, Boone C, Tang M, Ybarra J, Ballapuram AC, Taran KA, Chen PY, Amendáriz M, Leung CJ, Harris M, Tjoa K, Hongo H, Abelson S, Rivera J, Ngo N, Herbst DM, Suciu RM, Guijas C, Sedighi K, Andalis T, Roche E, Xie B, Liu Y, Smith CC, Stieglitz E, Niphakis MJ, Cravatt BF, Shannon K. In vivo validation of the palmitoylation cycle as a therapeutic target in NRAS-mutant cancer. bioRxiv. [Preprint]. 2025 Mar 21:2025.03.20.644389. doi: 10.1101/2025.03.20.644389. PMCID: PMC11957127.

Gross AM, O'Sullivan Coyne G, Dombi E, Tibery C, Herrick WG, Martin S, Angus SP, Shern JF, Rhodes SD, Foster JC, Rubinstein LV, Baldwin A, Davis C, Dixon SAH, Fagan M, Ong MJ, Wolters PL, Tamula MA, Reid O, Sankaran H, Fang F, Govindharajulu JP, Browne AT, Kaplan RN, Heisey K, On TJ, Xuei X, Zhang X, Johnson BC, Parchment RE, Clapp DW, Srivastava AK, Doroshow JH, Chen AP, Widemann BC. Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial. Nat Med. 2025 Jan;31(1):105-115. doi: 10.1038/s41591-024-03361-4. Epub 2025 Jan 6. PMID: 39762421.

Sait SF, Tang KH, Angus SP, Brown R, Sun D, Xie X, Iltis C, Lien M, D Socci N, Bale TA, Davis C, Dixon SAH, Zhang C, Wade Clapp D, Neel BG, Parada LF. Hydroxychloroquine prevents resistance and potentiates the antitumor effect of SHP2 inhibition in NF1-associated malignant peripheral nerve sheath tumors. Proc Natl Acad Sci U S A. 2025 Jan 7;122(1):e2407745121. doi: 10.1073/pnas.2407745121. Epub 2024 Dec 30. PMCID: PMC11725864 (available on 2025-06-30).

Xie XP, Ganbold M, Li J, Lien M, Chipman ME, Wang T, Jayewickreme CD, Pedraza AM, Bale T, Tabar V, Brennan C, Sun D, Sharma R, Parada LF. Glioblastoma functional heterogeneity and enrichment of cancer stem cells with tumor recurrence. Neuron. 2024 Dec 18;112(24):4017-4032.e6. doi: 10.1016/j.neuron.2024.10.012. Epub 2024 Nov 6. PMCID: PMC11659040 (available on 2025-12-18).

Chen YJ, Iyer SV, Hsieh DC, Li B, Elias HK, Wang T, Li J, Ganbold M, Lien MC, Peng YC, Xie XP, Jayewickreme CD, van den Brink MRM, Brady SF, Lim SK, Parada LF. Gliocidin is a nicotinamide-mimetic prodrug that targets glioblastoma. Nature. 2024 Dec;636(8042):466-473.doi: 10.1038/s41586-024-08224-z. Epub 2024 Nov 20. PMCID: PMC11665509

Zhang L, Maalouf A, Makri SC, Banerjee J, Suru A, Tam AJ, Calizo A, Pollard K, Wang J, Danilova L, Ioannou M, Levin AS, Morris CD, Rhee DS, Belzberg AJ, Blakeley JO, Ladle BH, Pardoll DM, Lucas CG, Rodriguez FJ, Gross JM, Anders RA, Pratilas CA, Llosa NJ. Multidimensional Immunotyping of Human NF1-Associated Peripheral Nerve Sheath Tumors Uncovers Tumor-Associated Macrophages as Key Drivers of Immune Evasion in the Tumor Microenvironment. Clin Cancer Res. 2024 Dec 2;30(23):5459-5472. doi: 10.1158/1078-0432.CCR-24-1454. PubMed PMID: 39321200; PubMed Central PMCID: PMC11866061.

Stieglitz E, Lee AG, Angus SP, Davis C, Barkauskas DA, Hall D, Kogan SC, Meyer J, Rhodes SD, Tasian SK, Xuei X, Shannon K, Loh ML, Fox E, Weigel BJ. Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: a Report from the Children's Oncology Group. Cancer Discov. 2024 Sep 4;14(9):1590-1598. doi: 10.1158/2159-8290.CD-23-1376. PMCID: PMC11374478 (available on 2025-03-04).

Wintering A, Hecht A, Meyer J, Wong EB, Hübner J, Abelson S, Feldman K, Kennedy VE, Peretz CAC, French DL, Maguire JA, Jobaliya C, Vasquez MR, Desai S, Dulman R, Nemecek E, Haines H, Hammad M, El Haddad A, Kogan SC, Abdullaev Z, Chehab FF, Tasian SK, Smith CC, Loh ML, Stieglitz E. LNK/ SH2B3 as a novel driver in juvenile myelomonocytic leukemia. Haematologica. 2024 Aug 1;109(8):2533-2541. doi: 10.3324/haematol.2023.283776. PMCID: PMC11290546.

Na B, Shah SR, Vasudevan HN. Past, Present, and Future Therapeutic Strategies for NF-1-Associated Tumors. Curr Oncol Rep. 2024 Jun;26(6):706-713. doi: 10.1007/s11912-024-01527-4. Epub 2024 May 6. PMCID: PMC11169015.

Somatilaka BN, Madana L, Sadek A, Chen Z, Chandrasekaran S, McKay RM, Le LQ. STING activation reprograms the microenvironment to sensitize NF1-related malignant peripheral nerve sheath tumors for immunotherapy. J Clin Invest. 2024 Mar 19;134(10):e176748. doi: 10.1172/JCI176748. PMCID: PMC11093615

Mitchell DK, Burgess B, White EE, Smith AE, Sierra Potchanant EA, Mang H, Hickey BE, Lu Q, Qian S, Bessler W, Li X, Jiang L, Brewster K, Temm C, Horvai A, Albright EA, Fishel ML, Pratilas CA, Angus SP, Clapp DW, Rhodes SD. Spatial Gene-Expression Profiling Unveils Immuno-oncogenic Programs of NF1-Associated Peripheral Nerve Sheath Tumor Progression. Clin Cancer Res. 2024 Mar 1;30(5):1038-1053. doi: 10.1158/1078-0432.CCR-23-2548. PMCID: PMC11095977 (available on 2024-09-01).

Furnari FB, Anastasaki C, Bian S, Fine HA, Koga T, Le LQ, Rodriguez FJ, Gutmann DH. Stem cell modeling of nervous system tumors. Dis Model Mech. 2024 Feb 1;17(2):dmm050533. doi: 10.1242/dmm.050533. Epub 2024 Feb 14. PMCID: PMC10886724.

Vasudevan HN, Payne E, Delley CL, John Liu S, Mirchia K, Sale MJ, Lastella S, Nunez MS, Lucas CG, Eaton CD, Casey-Clyde T, Magill ST, Chen WC, Braunstein SE, Perry A, Jacques L, Reddy AT, Pekmezci M, Abate AR, McCormick F, Raleigh DR. Functional interactions between neurofibromatosis tumor suppressors underlie Schwann cell tumor de-differentiation and treatment resistance. Nat Commun. 2024 Jan 12;15(1):477. doi: 10.1038/s41467-024-44755-9. PMCID: PMC10786885.

Mitchell DK, Brewster K, Makri SC, Khan J, Albright EA, Horvai A, Mang H, Lu Q, Dixon SAH, White E, Saadatzadeh MR, Bijangi-Vishehsaraei K, Gampala S, Hickey BE, Leffew H, Li X, Jiang L, Ciesielski MD, Bessler WK, Collier CD, Cohen-Gadol A, Fishel ML, Pratilas CA, Pollok KE, Angus SP, Rhodes S, Clapp DW. DLK1 distinguishes subsets of NF1-associated malignant peripheral nerve sheath tumors with divergent molecular signatures. PMCID: PMC12081192.

Gross AM, Dombi E, Wolters PL, Baldwin A, Dufek A, Herrera K, Martin S, Derdak J, Heisey KS, Whitcomb PM, Steinberg SM, Venzon DJ, Fisher MJ, Kim A, Bornhorst M, Weiss BD, Blakeley JO, Smith MA, Widemann BC. Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas. Neuro Oncol. 2023 Oct 3;25(10):1883-1894. doi: 10.1093/neuonc/noad086. PMCID: PMC10547508 (available on 2024-04-28).

Stieglitz E, Gu CJ, Richardson M, Kita R, Santaguida MT, Ali KA, Strachan DC, Dhar A, Yam G, Anderson W, Anderson E, Hübner J, Tasian SK, Loh ML, Lacher MD. Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. JCO Precis Oncol.2023 Sep:7:e2300302. doi: 10.1200/PO.23.00302. PMCID: PMC10645413.

Flint AC, Mitchell DK, Angus SP, Smith AE, Bessler W, Jiang L, Mang H, Li X, Lu Q, Rodriguez B, Sandusky GE, Masters AR, Zhang C, Dang P, Koenig J, Johnson GL, Shen W, Liu J, Aggarwal A, Donoho GP, Willard MD, Bhagwat SV, Clapp DW, Rhodes SD. Combined CDK4/6 and ERK1/2 Inhibition Enhances Antitumor Activity in NF1-Associated Plexiform Neurofibroma. Clin Cancer Res. 2023 Sep 1;29(17):3438-3456. doi: 10.1158/1078-0432.CCR-22-2854. PMCID: PMC11060649.

Chen PY, Huang BJ, Harris M, Boone C, Wang W, Carias H, Mesiona B, Mavrici D, Kohler AC, Bollag G, Zhang C, Zhang Y, Shannon K. Structural and functional analyses of a germline KRAS T50I mutation provide insights into Raf activation. CI Insight. 2023 Sep 8;8(17):e168445.doi: 10.1172/jci.insight.168445. PMCID: PMC10544224.

Jiang C, McKay RM, Lee SY, Romo CG, Blakeley JO, Haniffa M, Serra E, Steensma MR, Largaespada D, Le LQ. Cutaneous Neurofibroma Heterogeneity: Factors that Influence Tumor Burden in Neurofibromatosis Type 1. J Invest Dermatol. 2023 Aug;143(8):1369-1377. doi: 10.1016/j.jid.2022.12.027. Epub 2023 Jun 15. PMID: 37318402. -- NIHMS1998517

Flint AC, Mitchell DK, Angus SP, Smith AE, Bessler W, Jiang L, Mang H, Li X, Lu Q, Rodriguez B, Sandusky GE, Masters AR, Zhang C, Dang P, Koenig J, Johnson GL, Shen W, Liu J, Aggarwal A, Donoho GP, Willard MD, Bhagwat SV, Clapp DW, Rhodes SD. Combined CDK4/6 and ERK1/2 Inhibition Enhances Antitumor Activity in NF1-Associated Plexiform Neurofibroma. Clin Cancer Res. 2023 Sep 1;29(17):3438-3456. doi: 10.1158/1078-0432.CCR-22-2854. PMCID: PMC11060649.

Cuevas-Navarro A, Wagner M, Van R, Swain M, Mo S, Columbus J, Allison MR, Cheng A, Messing S, Turbyville TJ, Simanshu DK, Sale MJ, McCormick F, Stephen AG, Castel P. RAS-dependent RAF-MAPK hyperactivation by pathogenic RIT1 is a therapeutic target in Noonan syndrome-associated cardiac hypertrophy. Sci Adv. 2023 Jul 14;9(28):eadf4766. doi: 10.1126/sciadv.adf4766. Epub 2023 Jul 14. PMCID: PMC10348673.

Jiang C, Kumar A, Yu Z, Shipman T, Wang Y, McKay RM, Xing C, Le LQ. Basement membrane proteins in extracellular matrix characterize NF1 neurofibroma development and response to MEK inhibitor. J Clin Invest. 2023 Jun 15;133(12):e168227.doi: 10.1172/JCI168227. PMCID: PMC10266775.

Kotch C, Wagner K, Broad JH, Dombi E, Minturn JE, Phillips P, Smith K, Li Y, Jacobs IN, Elden LM, Fisher MJ, Belasco J. Vinblastine/Methotrexate for Debilitating and Progressive Plexiform Neurofibroma in Children and Young Adults with Neurofibromatosis Type 1: A Phase 2 Study. Cancers (Basel). 2023 May 5;15(9):2621. doi: 10.3390/cancers15092621. PMCID: PMC10177272.

Chipman ME, Wang Z, Sun D, Pedraza AM, Bale TA, Parada LF. Tumor progression is independent of tumor-associated macrophages in cell lineage-based mouse models of glioblastoma. Proc Natl Acad Sci U S A. 2023 Apr 18;120(16):e2222084120.doi: 10.1073/pnas.2222084120. Epub 2023 Apr 11. PMCID: PMC10120014.

Levinson A, Tjoa K, Huang BJ, Meyer LK, Kim MO, Brady SW, Zhang J, Shannon KM, Wandler A. Opposing Effects of KDM6A and JDP2 on Glucocorticoid Sensitivity in T-ALL. Blood Adv. 2023 Mar 10;bloodadvances.2021006881. doi: 10.1182/bloodadvances.2021006881. Online ahead of print. PMID: 36897249.

Paria N, Khalid A, Shen B, Lemoine B, Chan J, Kidane YH, Oxendine I, Cornelia R, Wise CA, Rios JJ. Molecular Dissection of Somatic Skeletal Disease in Neurofibromatosis Type 1. J Bone Miner Res. 2023 Feb;38(2):288-299. doi: 10.1002/jbmr.4755. Epub 2022 Dec 13. PMCID: PMC9898201.

Gross AM, Glassberg B, Wolters PL, Dombi E, Baldwin A, Fisher MJ, Kim A, Bornhorst M, Weiss BD, Blakeley JO, Whitcomb P, Paul SM, Steinberg SM, Venzon DJ, Martin S, Carbonell A, Heisey K, Therrien J, Kapustina O, Dufek A, Derdak J, Smith MA, Widemann BC. Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity. Neuro Oncol. 2022 Nov 2;24(11):1978-1988. doi: 10.1093/neuonc/noac109. PMCID: PMC9629448.

de Blank PMK, Gross AM, Akshintala S, Blakeley JO, Bollag G, Cannon A, Dombi E, Fangusaro J, Gelb BD, Hargrave D, Kim A, Klesse LJ, Loh M, Martin S, Moertel C, Packer R, Payne JM, Rauen KA, Rios JJ, Robison N, Schorry EK, Shannon K, Stevenson DA, Stieglitz E, Ullrich NJ, Walsh KS, Weiss BD, Wolters PL, Yohay K, Yohe ME, Widemann BC, Fisher MJ. MEK inhibitors for neurofibromatosis type 1 manifestations: Clinical evidence and consensus. Neuro Oncol. 2022 Nov 2;24(11):1845-1856.doi: 10.1093/neuonc/noac165.PMCID: PMC9629420.

Bennett NK, Nakaoka HJ, Laurent D, Okimoto RA, Sei Y, Horvai AE, Bivona TG, Ten Hoeve J, Graeber TG, Nakamura K, Nakamura JL. Primary and metastatic tumors exhibit systems-level differences in dependence on mitochondrial respiratory function. PLoS Biol. 2022 Sep 22;20(9):e3001753.doi: 10.1371/journal.pbio.3001753. eCollection 2022 Sep. PMCID: PMC9498964.

Anastasaki C, Mo J, Chen JK, Chatterjee J, Pan Y, Scheaffer SM, Cobb O, Monje M, Le LQ, Gutmann DH. Neuronal hyperexcitability drives central and peripheral nervous system tumor progression in models of neurofibromatosis-1. Nat Commun. 2022 May 19;13(1):2785. doi: 10.1038/s41467-022-30466-6. PMCID: PMC9120229.

Somatilaka BN, Sadek A, McKay RM, Le LQ. Malignant peripheral nerve sheath tumor: models, biology, and translation. Oncogene. 2022 Apr;41(17):2405-2421. doi: 10.1038/s41388-022-02290-1. Epub 2022 Apr 7. PMCID: PMC9035132.

Castel P. Defective protein degradation in genetic disorders. Biochim Biophys Acta Mol Basis Dis 2022 May 1;1868(5):166366. doi: 10.1016/j.bbadis.2022.166366. Epub 2022 Feb 11. PMCID: PMC8977116.

Mo J, Moye SL, McKay RM, Le LQ. Neurofibromin and suppression of tumorigenesis: beyond the GAP. Oncogene. 2022 Feb;41(9):1235-1251. doi: 10.1038/s41388-021-02156-y. Epub 2022 Jan 23. PMCID: PMC9063229.

Behnert A, Meyer J, Parsa JY, Hechmer A, Loh ML, Olshen A, de Smith AJ, Stieglitz E. Exploring the genetic and epigenetic origins of juvenile myelomonocytic leukemia using newborn screening samples. Leukemia. 2022 Jan;36(1):279-282.doi: 10.1038/s41375-021-01331-0. Epub 2021 Jun 28.PMCID: PMC8720242.

Morales CE, Stieglitz E, Kogan SC, Loh ML, Braun BS. Nf1 and Sh2b3 mutations cooperate in vivo in a mouse model of juvenile myelomonocytic leukemia. Blood Adv. 2021 Sep 28;5(18):3587-3591. doi: 10.1182/bloodadvances.2020003754. PMCID: PMC8945579.

Jiang C, McKay RM, Le LQ. Tumorigenesis in neurofibromatosis type 1: role of the microenvironment. Oncogene. 2021 Sep;40(39):5781-5787. doi: 10.1038/s41388-021-01979-z. Epub 2021 Aug 3. PMCID: PMC8713356.

Bui A, Jiang C, McKay RM, Klesse LJ, Le LQ. Insights into the Pathogenesis of NF1-Associated Neoplasms. JID Innov. 2021 Sep;1(3):100044. doi: 10.1016/j.xjidi.2021.100044. Epub 2021 Aug 20. PMCID: PMC8486282.

Sun D, Xie XP, Zhang X, Wang Z, Sait SF, Iyer SV, Chen YJ, Brown R, Laks DR, Chipman ME, Shern JF, Parada LF. Stem-like cells drive NF1-associated MPNST functional heterogeneity and tumor progression. Cell Stem Cell. 2021 Aug 5;28(8):1397-1410.e4. doi: 10.1016/j.stem.2021.04.029. Epub 2021 May 18. PMCID: PMC8349880.

Laurent D, Smith AE, Bessler WK, Mendonca M, Chin-Sinex H, Descovich M, Horvai AE, Clapp DW, Nakamura JL. Irradiation of Nf1 mutant mouse models of spinal plexiform neurofibromas drives pathologic progression and decreases survival. Neurooncol Adv. 2021 Apr 23;3(1):vdab063.doi: 10.1093/noajnl/vdab063. eCollection Jan-Dec 2021. PMCID: PMC8193912.

Gampala S, Shah F, Zhang C, Rhodes SD, Babb O, Grimard M, Wireman RS, Rad E, Calver B, Bai RY, Staedtke V, Hulsey EL, Saadatzadeh MR, Pollok KE,Tong Y, Smith AE, Clapp DW, Tee AR, Kelley MR, Fishel ML. Exploring transcriptional regulators Ref-1 and STAT3 as therapeutic targets in malignant peripheral nerve sheath tumors. Br J Cancer. 2021 Apr;124(9):1566-1580.doi: 10.1038/s41416-021-01270-8. Epub 2021 Mar 3. PMCID: PMC8076291

Wang J, Pollard K, Calizo A, Pratilas CA. Activation of Receptor Tyrosine Kinases Mediates Acquired Resistance to MEK Inhibition in Malignant Peripheral Nerve Sheath Tumors. Cancer Res. 2021 Feb 1;81(3):747-762. doi: 10.1158/0008-5472.CAN-20-1992. Epub 2020 Nov 17. PMCID: PMC7854512.

Pikman Y, Stieglitz E. Targeting the Ras pathway in pediatric hematologic malignancies. Curr Opin Pediatr. 2021 Feb 1;33(1):49-58. doi: 10.1097/MOP.0000000000000981. Epub 2020 Dec 29. PMCID: PMC7880552.

Fisher MJ, Shih CS, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler WK, He Y, Park SJ, Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR; Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp DW. Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial. Nat Med 2021 Jan;27(1):165-173. doi: 10.1038/s41591-020-01193-6. Epub 2021 Jan 13. PMCID: PMC8275010.

Brosseau JP, Sathe AA, Wang Y, Nguyen T, Glass DA 2nd, Xing C, Le LQ. Human cutaneous neurofibroma matrisome revealed by single-cell RNA sequencing. Acta Neuropathol Commun. 2021 Jan 7;9(1):11. doi: 10.1186/s40478-020-01103-4. PMCID: PMC7792184.

Mo J, Anastasaki C, Chen Z, Shipman T, Papke J, Yin K, Gutmann DH, Le LQ. Humanized neurofibroma model from induced pluripotent stem cells delineates tumor pathogenesis and developmental origins. J Clin Invest. 2021 Jan 4;131(1):e139807. doi: 10.1172/JCI139807. PMCID: PMC7773354.

Schönung M, Meyer J, Nöllke P, Olshen AB, Hartmann M, Murakami N, Wakamatsu M, Okuno Y, Plass C, Loh ML, Niemeyer CM, Muramatsu H, Flotho C, Stieglitz E, Lipka DB. International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia. Clin Cancer Res. 2021 Jan 1;27(1):158-168. doi: 10.1158/1078-0432.CCR-20-3184. Epub 2020 Nov 2. PMCID: PMC7785676.

Ma Y, Gross AM, Dombi E, Pemov A, Choi K, Chaney K, Rhodes SD, Angus SP, Sciaky N, Clapp DW, Ratner N, Widemann BC, Rios JJ, Elefteriou F. A molecular basis for neurofibroma-associated skeletal manifestations in NF1. Genet Med. 2020 Nov;22(11):1786-1793. doi: 10.1038/s41436-020-0885-3. Epub 2020 Jun 30. PMCID: PMC8106869.

Wang Z, Sun D, Chen YJ, Xie X, Shi Y, Tabar V, Brennan CW, Bale TA, Jayewickreme CD, Laks DR, Alcantara Llaguno S, Parada LF. Cell Lineage-Based Stratification for Glioblastoma. Cancer Cell. 2020 Sep 14;38(3):366-379.e8. doi: 10.1016/j.ccell.2020.06.003. Epub 2020 Jul 9. PMCID: PMC7494533.

Pucciarelli D, Angus SP, Huang B, Zhang C, Nakaoka HJ, Krishnamurthi G, Bandyopadhyay S, Clapp DW, Shannon K, Johnson GL, Nakamura JL. Nf1 mutant tumors undergo transcriptome and kinome re-modeling after inhibition of either mTOR or MEK. Mol Cancer Ther. 2020 Aug 26;molcanther.1017.2019. doi: 10.1158/1535-7163.MCT-19-1017. [Online ahead of print]. PMCID: PMC7907257.

Bennett NK, Nguyen MK, Darch MA, Nakaoka HJ, Cousineau D, Ten Hoeve J, Graeber TG, Schuelke M, Maltepe E, Kampmann M, Mendelsohn BA, Nakamura JL, Nakamura K. Defining the ATPome reveals cross-optimization of metabolic pathways. Nat Commun. 2020 Aug 28;11(1):4319. doi: 10.1038/s41467-020-18084-6. PMCID: PMC7455733.

Armstrong AE, Rhodes SD, Smith A, Chen S, Bessler W, Ferguson MJ, Jiang L, Li X, Yuan J, Yang X, Yang FC, Robertson KA, Ingram DA, Blakeley JO, Clapp DW. Early administration of imatinib mesylate reduces plexiform neurofibroma tumor burden with durable results after drug discontinuation in a mouse model of neurofibromatosis type 1. Pediatr Blood Cancer. 2020 Aug;67(8):e28372. doi: 10.1002/pbc.28372. Epub 2020 May 27. PMCID: PMC32459399.

Mund JA, Park S, Smith AE, He Y, Jiang L, Hawley E, Roberson MJ, Mitchell DK, Abu-Sultanah M, Yuan J, Bessler WK, Sandusky G, Chen S, Zhang C, Rhodes SD, Clapp DW. Genetic disruption of the small GTPase RAC1 prevents plexiform neurofibroma formation in mice with neurofibromatosis type 17. J Biol Chem. 2020 Jul 17;295(29):9948-9958.doi: 10.1074/jbc.RA119.010981. Epub 2020 May 29. PMCID: PMC7380178.

Brosseau JP, Liao CP, Le LQ. Translating current basic research into future therapies for neurofibromatosis type 1. Br J Cancer. 2020 Jul;123(2):178-186. doi: 10.1038/s41416-020-0903-x. Epub 2020 May 22. PMCID: PMC7374719.

Lemberg KM, Wang J, Pratilas CA. From Genes to -Omics: The Evolving Molecular Landscape of Malignant Peripheral Nerve Sheath Tumor. Genes (Basel). 2020 Jun 24;11(6). doi: 10.3390/genes11060691. Review. PMCID: PMC7349243.

Muskens IS, de Smith AJ, Zhang C, Hansen HM, Morimoto L, Metayer C, Ma X, Walsh KM, Wiemels JL. Germline cancer predisposition variants and pediatric glioma: a population-based study in California. Neuro Oncol. 2020 Jun 9;22(6):864-874. doi: 10.1093/neuonc/noaa014. PMCID: PMC7283023.

Zambetti NA, Firestone AJ, Remsberg JR, Huang BJ, Wong JC, Long AM, Predovic M, Suciu RM, Inguva A, Kogan SC, Haigis KM, Cravatt BF, Shannon K. Genetic disruption of N-RasG12D palmitoylation perturbs hematopoiesis and prevents myeloid transformation in mice. Blood. 2020 May 14;135(20):1772-1782. doi: 10.1182/blood.2019003530. PMCID: PMC7225687

Scherer A, Stephens VR, McGivney GR, et al. Distinct Tumor Microenvironments Are a Defining Feature of Strain-Specific CRISPR/Cas9-Induced MPNSTs. Genes (Basel). 2020;11(5):583. Published 2020 May 23. doi:10.3390/genes11050583 PMCID: PMC7288323.

Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, Weiss B, Kim A, Bornhorst M, Shah AC, Martin S, Roderick MC, Pichard DC, Carbonell A, Paul SM, Therrien J, Kapustina O, Heisey K, Clapp DW, Zhang C, Peer CJ, Figg WD, Smith M, Glod J, Blakeley JO, Steinberg SM, Venzon DJ, Doyle LA, Widemann BC. Selumetinib in Children with Inoperable Plexiform Neurofibromas. N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735. Epub 2020 Mar 18. PMCID: PMC7305659.

Wong JC, Perez-Mancera PA, Huang TQ, Kim J, Grego-Bessa J, del pilar Alzamora M, Kogan SC, Sharir A, Keefe SH, Morales C, Schanze D, Zenker M, Sheppard D, Klein OD, Tuveson DA, Braun BS*, Shannon K*. Kras^P34R and Kras^T58I mutations induce distinct RASopathy phenotypes in mice. JCI Insight Sep 29:140495. doi: 10.1172/jci.insight.140495. PMCID: PMC7710308.

Brosseau JP, Le LQ. Heterozygous Tumor Suppressor Microenvironment in Cancer Development. Trends Cancer. 2019 Sep;5(9):541-546. doi: 10.1016/j.trecan.2019.07.004. Epub 2019 Aug 15. Review. PMCID: PMC6724550.

Burks CA, Rhodes, RD, Besser WK, Chen S, Smith A, Gehlhausen JR, Hawley ET, Jiang L, Li X, Yuan J, Lu Q, Jacobsen M, Sandusky GE, Jones DR, Clapp DW, Blakeley JO. Ketotifen Modulates Mast Cell Chemotaxis to Kit-Ligand, but Does Not Impact Mast Cell Numbers, Degranulation, or Tumor Behavior in Neurofibromas of Nf1-Deficient Mice. Molecular Cancer Therapeutics. Mol Cancer Ther. 2019 Sep 16. pii: molcanther.0123.2019. doi: 10.1158/1535-7163.MCT-19-0123. [Epub ahead of print]. -- PMCID: PMC7482139.

Chao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E. Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia. 2019 Sep 12. doi: 10.1038/s41375-019-0549-y. [Epub ahead of print]. PMCID: PMC6995757.

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