6,406 Total Number of Publications
375 Number of Publications This Year
 
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Locoregional delivery of CAR T cells to the cerebrospinal fluid for treatment of metastatic medulloblastoma and ependymoma

Recurrent medulloblastoma and ependymoma are universally lethal, with no approved targeted therapies and few candidates presently under clinical evaluation. Nearly all recurrent medulloblastomas and posterior fossa group A (PFA) ependymomas are located adjacent to and bathed by the cerebrospinal fluid, presenting an opportunity for locoregional therapy, bypassing the blood-brain barrier.…
Donovan, LK;Delaidelli, A;Joseph, SK;Bielamowicz, K;Fousek, K;Holgado, BL;Manno, A;Srikanthan, D;Gad, AZ;Van Ommeren, R;Przelicki, D;Richman, C;Ramaswamy, V;Daniels, C;Pallota, JG;Douglas, T;Joynt, ACM;Haapasalo, J;Nor, C;Vladoiu, MC;Kuzan-Fischer, CM;Garzia, L;Mack, SC;Varadharajan, S;Baker, ML;Hendrikse, L;Ly, M;Kharas, K;Balin, P;Wu, X;Qin, L;Huang, N;Stucklin, AG;Morrissy, AS;Cavalli, FMG;Luu, B;Suarez, R;De Antonellis, P;Michealraj, A;Rastan, A;Hegde, M;Komosa, M;Sirbu, O;Kumar, SA;Abdullaev, Z;Faria, CC;Yip, S;Hukin, J;Tabori, U;Hawkins, C;Aldape, K;Daugaard, M;Maris, JM;Sorensen, PH;Ahmed, N;Taylor, MD;
Nat. Med. ():
Nat. Med.
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Protective autophagy elicited by RAFMEKERK inhibition suggests a treatment strategy for RAS-driven cancers

Pancreatic ductal adenocarcinoma (PDA) was responsible for ~44,000 deaths in the United States in 2018 and is the epitome of a recalcitrant cancer driven by a pharmacologically intractable oncoprotein, KRAS1-4. Downstream of KRAS, the RAFMEKERK signaling pathway plays a central role in pancreatic carcinogenesis5. However, paradoxically, inhibition of this pathway…
Kinsey, CG;Camolotto, SA;Boespflug, AM;Guillen, KP;Foth, M;Truong, A;Schuman, SS;Shea, JE;Seipp, MT;Yap, JT;Burrell, LD;Lum, DH;Whisenant, JR;Gilcrease, GW;Cavalieri, CC;Rehbein, KM;Cutler, SL;Affolter, KE;Welm, AL;Welm, BE;Scaife, CL;Snyder, EL;McMahon, M;
Nat. Med. 25(4):620-627
Nat. Med.
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Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors

Atypical teratoid/rhabdoid tumors (ATRTs) typically arise in the central nervous system (CNS) of children under 3 years of age. Despite intensive multimodal therapy (surgery, chemotherapy and, if age permits, radiotherapy), median survival is 17 months1,2. We show that ATRTs robustly express B7-H3/CD276 that does not result from the inactivating mutations…
Theruvath, J;Sotillo, E;Mount, CW;Graef, CM;
Nature Medicine ():
Nature Medicine
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Tumor-derived IFN triggers chronic pathway agonism and sensitivity to ADAR loss

Interferons (IFNs) are cytokines that play a critical role in limiting infectious and malignant diseases 1-4 . Emerging data suggest that the strength and duration of IFN signaling can differentially impact cancer therapies, including immune checkpoint blockade 5-7 . Here, we characterize the output of IFN signaling, specifically IFN-stimulated gene…
Liu, H;Golji, J;Brodeur, LK;Chung, FS;Chen, JT;deBeaumont, RS;Bullock, CP;Jones, MD;Kerr, G;Li, L;Rakiec, DP;Schlabach, MR;Sovath, S;Growney, JD;Pagliarini, RA;Ruddy, DA;MacIsaac, KD;Korn, JM;McDonald, ER;
Nat. Med. ():
Nat. Med.
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Mitochondrial DNA variants modulate N-formylmethionine, proteostasis and risk of late-onset human diseases

Mitochondrial DNA (mtDNA) variants influence the risk of late-onset human diseases, but the reasons for this are poorly understood. Undertaking a hypothesis-free analysis of 5,689 blood-derived biomarkers with mtDNA variants in 16,220 healthy donors, here we show that variants defining mtDNA haplogroups Uk and H4 modulate the level of circulating…
Cai, N;Gomez-Duran, A;Yonova-Doing, E;Kundu, K;Burgess, AI;Golder, ZJ;Calabrese, C;Bonder, MJ;Camacho, M;Lawson, RA;Li, L;Williams-Gray, CH;ICICLE-PD Study Group, ;Di Angelantonio, E;Roberts, DJ;Watkins, NA;Ouwehand, WH;Butterworth, AS;Stewart, ID;Pietzner, M;Wareham, NJ;Langenberg, C;Danesh, J;Walter, K;Rothwell, PM;Howson, JMM;Stegle, O;Chinnery, PF;Soranzo, N;
():,2021
SSRN Electronic Journal
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CAR T cells with dual targeting of CD19 and CD22 in adult patients with recurrent or refractory B cell malignancies: a phase 1 trial

Despite impressive progress, more than 50% of patients treated with CD19-targeting chimeric antigen receptor T cells (CAR19) experience progressive disease. Ten of 16 patients with large B cell lymphoma (LBCL) with progressive disease after CAR19 treatment had absent or low CD19. Lower surface CD19 density pretreatment was associated with progressive…
Spiegel, JY;Patel, S;Muffly, L;Hossain, NM;Oak, J;Baird, JH;Frank, MJ;Shiraz, P;Sahaf, B;Craig, J;Iglesias, M;Younes, S;Natkunam, Y;Ozawa, MG;Yang, E;Tamaresis, J;Chinnasamy, H;Ehlinger, Z;Reynolds, W;Lynn, R;Rotiroti, MC;Gkitsas, N;Arai, S;Johnston, L;Lowsky, R;Majzner, RG;Meyer, E;Negrin, RS;Rezvani, AR;Sidana, S;Shizuru, J;Weng, WK;Mullins, C;Jacob, A;Kirsch, I;Bazzano, M;Zhou, J;Mackay, S;Bornheimer, SJ;Schultz, L;Ramakrishna, S;Davis, KL;Kong, KA;Shah, NN;Qin, H;Fry, T;Feldman, S;Mackall, CL;Miklos, DB;
():,2021
SSRN Electronic Journal
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A compendium of kinetic modulatory profiles identifies ferroptosis regulators

Cell death can be executed by regulated apoptotic and nonapoptotic pathways, including the iron-dependent process of ferroptosis. Small molecules are essential tools for studying the regulation of cell death. Using time-lapse imaging and a library of 1,833 bioactive compounds, we assembled a large compendium of kinetic cell death modulatory profiles…
Conlon, M;Poltorack, CD;Forcina, GC;Armenta, DA;Mallais, M;Perez, MA;Wells, A;Kahanu, A;Magtanong, L;Watts, JL;Pratt, DA;Dixon, SJ;
():,2021
Nature chemical biology
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Systematic identification of biomarker-driven drug combinations to overcome resistance

The ability to understand and predict variable responses to therapeutic agents may improve outcomes in patients with cancer. We hypothesized that the basal gene-transcription state of cancer cell lines, coupled with cell viability profiles of small molecules, might be leveraged to nominate specific mechanisms of intrinsic resistance and to predict…
Rees, MG;Brenan, L;do Carmo, M;Duggan, P;Bajrami, B;Arciprete, M;Boghossian, A;Vaimberg, E;Ferrara, SJ;Lewis, TA;Rosenberg, D;Sangpo, T;Roth, JA;Kaushik, VK;Piccioni, F;Doench, JG;Root, DE;Johannessen, CM;
():,2022
Nature chemical biology
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Macrophages, rather than DCs, are responsible for inflammasome activity in the GM-CSF BMDC model

Inflammasomes are one of the most important mechanisms for innate immune defense against microbial infection but are also known to drive various inflammatory disorders via processing and release of the cytokine IL-1. As research into the regulation and effects of inflammasomes in disease has rapidly expanded, a variety of cell…
Erlich, Z;Shlomovitz, I;Edry-Botzer, L;Cohen, H;Frank, D;Wang, H;Lew, AM;Lawlor, KE;Zhan, Y;Vince, JE;Gerlic, M;
Nat. Immunol. ():
Nat. Immunol.
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Targeted glycan degradation potentiates the anticancer immune response in vivo

Currently approved immune checkpoint inhibitor therapies targeting the PD-1 and CTLA-4 receptor pathways are powerful treatment options for certain cancers; however, most patients across cancer types still fail to respond. Consequently, there is interest in discovering and blocking alternative pathways that mediate immune suppression. One such mechanism is an upregulation…
Gray, MA;Stanczak, MA;Mantuano, NR;Xiao, H;Pijnenborg, JFA;Malaker, SA;Miller, CL;Weidenbacher, PA;Tanzo, JT;Ahn, G;Woods, EC;Läubli, H;Bertozzi, CR;
():,2020
Nat. Chem. Biol.
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