Experimental Hematology and Cancer Biology

Publications

Ali, S; Ehrman, JM; Merchan-Sala, P; Matthew Kofron, J; Waclaw, RR; Campbell, K. A central role for Islr2 (Linx) in direct pathway striatal projection neurons for the correct formation of the internal capsule and cerebral peduncle. Developmental Biology. 2026.

Anandappa, AJ; Xiao, W; Miles, LA. Single cell sequencing in acute myeloid leukemia: Linking genotype to functional phenotype for precision risk stratification and treatment decisions. Human Pathology. 2026; 106174.

Babic, I; Bryan, N; Cunningham, C; Sampson, A; Starczynowski, D; Nurmemmedov, E. MICRO-TAG enzyme complementation enables quantification of cellular drug-target engagement in temperature series. SLAS Discovery. 2026; 38:100291.

Batschelett, MA; Migneault, KY; Aschbacher-Smith, L; Cecil, KM; Huddleston, DA; Gilbert, DL; Ratner, N; Difrancesco, MW. Multiecho Resting-State Functional Magnetic Resonance Imaging Analysis of Attention-Deficit/Hyperactivity Disorder Severity and Motor Dysfunction in a Pediatric Sample of Neurofibromatosis Type 1. Pediatric Neurology. 2026; 181:136-147.

Bischoff, LJ; Sherpa, C; Schrenk, S; Boscolo, E. Constitutive, Mosaic Expression of TIE2 p.L914F During Mouse Development Causes Venous Malformation. The FASEB Journal. 2026; 40(9):e71862.

Cai, M; Duan, X; Jorgensen, R; Wunderlich, M; Guo, F; Zheng, Y. Abstract B013: Targeting CDC42 increases TCF1-mediated “stemness” and rejuvenates human T cells to enhance CAR-T efficacy. Cancer immunology research. 2026; 14(2_Supplement):b013-b013.

Chlon, T. Rescue gone rogue: the ERCC6L2 syndrome and TP53 mutations. Blood. 2026; 147(15):1651-1652.

Dezern, AE; Gillis, N; Otterstatter, M; Abel, G; Padron, E; Deeg, HJ; Al Baghdadi, T; Liu, J; Xie, Z; Zhang, L; Lee, C; Difronzo, N; Walter, M; Sekeres, MA. A novel approach to defining progression in MDS and precursor myeloid conditions in the MDS Natural History Study. Blood advances. 2026; 10(8):2743-2753.

Drucker, M; Lee, D; Bowman, M; Zhang, X; Kain, BN; Nicolet, D; Bandopadhyay, R; Singh, V; Wang, Z; Stone, RM; Grimes, HL; Eisfeld, A-K; Bowman, RL; Miles, LA. Genotype-immunophenotype relationships in NPM1-mutated AML clonal evolution uncovered by single-cell multiomic analysis. Blood. 2026; 147(26):3209-3216.

Duong, N; Khan, DH; Thomas, GE; Feng, Y; Hurren, R; Lee, JB; St-Germain, J; Drimmer, L; Yan, Y; Zhang, LX; Jacobs, MG; Goede, D; Spadavecchio, V; Schimmer, AD. Intracellular IL-23R is necessary for mitotic spindle formation and viability in AML. Leukemia. 2026; 40(5):906-917.

Eskandari, N; Pappano, E; Ruiz, MAD; Mahmud, N; Ren, G; Ivy, ZK; Gaitonde, S; Gordeuk, VR; Gladwin, MT; Malik, P; Quinn, CT; Saraf, SL. Renin-Angiotensin-Aldosterone System Inhibition Exacerbates Anemia in Sickle Cell Disease. Blood. 2026.

Fobare, S; Sharpe, C; Quinn, K; Bryant, K; Miles, LA; Bowman, RL; Cheney, C; Furby, C; Long, M; Fyock, K; Perentesis, JP; Eisfeld, A-K; Hertlein, E; Byrd, JC. Characterization of the clonal hierarchy and immunophenotype of PTPN11 mutations in acute myeloid leukemia. JCI Insight. 2026; 11(4).

Gilbert, DL; Aschbacher-Smith, LE; Migneault, KY; Huddleston, DA; Cecil, KM; Beebe, DW; Zhang, B; Ratner, N; Prada, CE. A Randomized, Double-Blind, Pilot Study of N-Acetylcysteine for Motor and Cognitive Symptoms in Youth With Neurofibromatosis Type 1. Pediatric Neurology. 2026; 180:129-137.

Gopalan, L; Na, Y; Hu, L; Hall, A; Kim, M-O; Dombi, E; Szabo, S; Ratner, N; Huang, G; Wu, J. Valosin-Containing Protein Contributes to Plexiform Neurofibroma Formation and Represents a Novel Therapeutic Target. Cells. 2026; 15(9).

Grover, KE; Cappel, ZR; Volz, AM; Cotella, EM; Smallwood, K; Berryhill, CA; Raje, KR; Fisher, AA; Casper, MCT; Nardini, D; Prada, CE; Weaver, KN; Waclaw, RR; Robinson, JE. Genetic activation of ERK2 recapitulates core neurodevelopmental features of Rasopathy syndromes in mice. HGG Advances. 2026; 7(3):100621.

Hashimoto, M; Feng, X; Bai, J; Zeng, H; Li, T; Li, J; Umemoto, T; Andreassen, PR; Huang, G. Insights and modulation of RNA polymerase-dependent R-loop and dsRNA in Fanconi anemia hematopoietic stem cells. JCI Insight. 2026; 11(7).

Kassim, AA; Thompson, AA; Malik, P. Exploring Affordable Curative Therapy for Sickle Cell Disease in Africa: A Comprehensive Overview. American Journal of Hematology. 2026; 101 Suppl 1(Suppl 1):56-74.

Kirstein, A; Ionascu, D; Lamba, MAS; Ruffolo, BM; Crail, KJ; Mascia, AE; Sertorio, MG; Hinrichs, BH; Zender, C; Lehn, MA; Wise-Draper, TM; Perentesis, JP; Zheng, Y; Wells, SI. In vitro determination of patient-specific variation challenges the universal RBE gold standard for proton radiation therapy. Clinical and Translational Radiation Oncology. 2026; 56:101046.

Koo, J; Langenberg, L; Zhao, X; Setchell, KR; Lake, KE; Luebbering, N; Walter, E; Lane, A; Myers, KC; Reynaud, D; Sabulski, A; Teusink-Cross, A; Jodele, S; Davies, SM. Lipid dysregulation after hematopoietic stem cell transplant. Haematologica. 2026; 111(5):1634-1645.

Koo, J; Langenberg, L; Walter, E; Zhao, X; Setchell, K; Lake, KE; Khandelwal, P; Lane, A; Manjrekar, P; Myers, KC; Reynaud, D; Sabulski, A; Teusink-Cross, A; Jodele, S; Davies, SM. Lower Isn't Better: Baseline LDL-C Identifies Endothelial Vulnerability and Outcomes after HSCT. Elsevier; 2026:s594.

Lacher, RK; Henson, K; Wathen, LN; Jones, C; Arnold, T; Rice, MR; Carles, HM; White, AR; Ramsuchit, E; Murrey, D; Tiwari, D; Erickson, CA; Pedapati, EV; Gross, C. FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome. Gene Therapy. 2026.

Lanzkron, S; Coleman-Cowger, VH; Thompson, AA; Carroll, CP; Clemons, T; Debaun, M; Kanter, J; Malik, P; Manwani, D; Pierciey, FJ; Walters, MC; Alai, S. Cure Sickle Cell Initiative recommendations on common data elements for sickle cell disease gene therapy trials. Blood advances. 2026; 10(11):3960-3965.

Lee, YS; Good, JD; Awais, N; Eschle, BK; Lineberry, MS; Lin, J; Wunderlich, M; Thibodeau, J; Edwards, H; Teo, JL; Garcia, JS; Lane, AA; Jones, CL; Van Galen, P. Synergistic targeting of eIF4A-mediated translation initiation and apoptosis in acute myeloid leukemia. Blood Neoplasia. 2026; 3(2):100202.

Lee, D; Abu-Shihab, Y; Plas, K; Nicolet, D; Mrózek, K; Routbort, MJ; Patel, KP; Walker, CJ; Buss, J; Stiff, AR; Tyner, JW; Loghavi, S; Eisfeld, A-K; Miles, LA. Prognostic and therapeutic implications of BRAF mutations in acute myeloid leukemia. Leukemia. 2026.

Li, X; Aguilar-Navarro, AG; Nader, M; Jahangiri, S; Lee, HS; Edun, G; Gower, M; Yang, D; Fernandez, M; Lin, B; Kim, Y-M; Adams, RH; Sivaraj, KK; Tikhonova, AN. IFNγ Drives Long-Term Bone Marrow Niche Dysfunction Following Doxorubicin-Based Chemotherapy. Blood. 2026.

Ling, T; O'brien, C; St-Germain, JR; Rondeau, V; Shi, M; Berman, JM; Cepa, A; Saez Raez, P; Wunderlich, M; Carter, KM; Hope, KJ; D'alessandro, A; Raught, B; Jones, CL. Glutaredoxin 2 is essential for AML survival through mitochondrial permeability transition pore regulation. Blood. 2026; 147(8):863-876.

Mejía-Ramírez, E; Picazo, PI; Walter, B; Montserrat-Vazquez, S; Affuso, F; Wieser, S; Pezzano, F; Reymond, L; Castillo-Robles, J; Matteini, F; Ruprecht, V; Zheng, Y; Petrone, P; Florian, MC. Targeting RhoA nuclear mechanoactivity rejuvenates aged hematopoietic stem cells. Nature Aging. 2026; 6(1):68-87.

Nalapareddy, K; Haslam, DB; Kissmann, A-K; Alenghat, T; Stahl, S; Rosenau, F; Zheng, Y; Geiger, H. Microbiota from young mice restore the function of aged ISCs. Stem Cell Reports. 2026; 21(2):102788.

Novitzky-Basso, I; Xu, C; Chiarello, C; Reisz, JA; D'alessandro, A; Bader, GD; Mattsson, J; Jones, C. Metabolomic Signatures of Relapse and Survival in AML Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation. Hematology Reports. 2026; 18(2).

Rai, P; Roy, S; Arumugam, P; Konstantinidis, DG; Ponny, SR; Eiymo Mwa Mpollo, M-S; Shrestha, A; Kalfa, TA; Malik, P. Angiotensin signaling is essential for stress erythropoiesis but causes retention of dysfunctional mitochondria in RBCs. JCI Insight. 2026; 11(9).

Raut, NGR; Sprague, KL; Adlakha, A; Rupert, AR; Hofmann, MC; Queme, LF; Ratner, N; Jankowski, MP. MAPK-dependent release of GDNF from Schwann cells mediates tumor-independent pain in neurofibromatosis 1. Science Signaling. 2026; 19(939):eaee5174.

Ravindran, R; Kedei, N; Youn, E-K; Choi, K; Volz, A; Pundavela, J; Largaespada, DA; Shern, JF; Ratner, N. Abstract 2269: Two-step mechanism of plexiform neurofibroma formation: Role of the NF-κB pathway in neurofibroma formation. Cancer Research. 2026; 86(7_Supplement):2269-2269.

Ren, L; Zhang, H; Bobileva, O; Nai, F; Bi, H; Chan, A; Baker, GE; Dong, L; Guarin, D; Hu, W; Chen, J; Caflisch, A; Li, X; Su, R. Targeting the METTL1/m7G axis as a therapeutic strategy in myeloid leukemia. Blood. 2026; 147(25):3069-3085.

Roome, RB; Yadav, A; Flores, L; Puarr, AK; Nardini, D; Richardson, A; Waclaw, RR; Arkell, RM; Menon, V; Johnson, JE; Levine, AJ. Ontogeny of the spinal cord dorsal horn. Science. 2026; 391(6781):eadx5781.

Sagar, K; Boscolo, E. Advances in genetics, signaling, and modeling of venous malformations. Frontiers in Cardiovascular Medicine. 2026; 13:1770126.

Tang, W; Li, Y; Boscolo, E; Kohane, DS; Cullion, K. Polymeric rapamycin nanoparticles encapsulating ponatinib cause regression of venous malformations in mice. Science Translational Medicine. 2026; 18(848):eaeb7597.

Uible, EE; Choi, I; Clough, CA; Hassan, A; Anandappa, AJ; Fisher, J; Karki, B; Hueneman, K; Choi, K; Vick, EJ; Pasare, C; Cunningham, JT; Volk, AG; Starczynowski, DT. Scaffolding-dependent CASP1 constrains excessive cell-intrinsic inflammatory signaling in leukemia. Cell Chemical Biology. 2026; 33(1):59-73.e10.

Williams, KB; Larsson, AT; Keller, BJ; Chaney, KE; Williams, RL; Bhunia, MM; Draper, GM; Jubenville, TA; Hudson, WA; Georg, GI; Moertel, CL; Ratner, N; Largaespada, DA. Pharmacogenomic Synthetic Lethal Screens Reveal Hidden Vulnerabilities and New Therapeutic Approaches for Treatment of NF1-Associated Tumors. Molecular Cancer Therapeutics. 2026; 25(2):230-243.

Woodburn, SC; Kuhn, A; Bosis, K; Dadodsky, DT; Mueller, E; Bollinger, JL; Gonzalez, RMS; Robinson, JE; Vollmer, LL; Wohleb, ES. Microglial brain-derived neurotrophic factor (BDNF) supports the behavioral and synaptogenic effects of ketamine. Brain, Behavior, and Immunity. 2026; 106886.

Yang, Y; Liu, X; Wei, J; Qin, J; Tan, Q; Ji, J; Guo, F; Zheng, Y; Bi, F; Liu, M; Wang, G. In vivo expression of CD3/CD19 bispecific T-cell engager and α-PD-L1-Fc enables effective and durable immunotherapy for CD19+/PD-L1+ leukemia. Leukemia. 2026; 40(2):348-359.

Yue, M; Wang, Y; Gu, J; Zhang, C; Chen, Y; Gao, A; Choi, K; Wu, Z; Wang, Z; Pundavela, J; Ratner, N; Yu, Y. Decreased PP2A expression and activity represent a therapeutic target for plexiform neurofibroma. Acta Neuropathologica Communications. 2026.

Zhang, X; Xue, J; Zhao, C; Zeng, C; Zhong, J; Yu, G; Yang, X; Ling, Y; Li, D; Yang, J; Lu, QR; Deng, Y; Tang, Z; Lu, F. Oligodendrocyte transcription factor 2 orchestrates glioblastoma immune evasion by suppressing CXCL10 and CD8+ T cell activation. Journal of Clinical Investigation. 2026; 136(5).

Zhang, Z; Fan, C; Jorgensen, R; Sylvestre, P; Adam, M; Zheng, Y. Loss of polarity by Cdc42 depletion and oncogenic Kras activation in the mouse intestinal epithelia leads to a necrotizing enterocolitis (NEC)-like disease. Nature Communications. 2026; 17(1).