Results 31 to 40 of about 6,416 (143)
Real‐World Effectiveness and Safety of Pegzilarginase Treatment in Arginase 1 Deficiency
JIMD Reports, Volume 67, Issue 3, May 2026.ABSTRACT
Pegzilarginase is the first disease‐modifying drug for arginase 1 deficiency. In clinical trials, pegzilarginase effectively normalised plasma arginine (pArg), which was associated with clinically relevant improvements in neuromotor outcomes. We report from a French early access scheme the first report on experience from pegzilarginase therapy Anaïs Brassier, Alina Arion, Jean‐Baptiste Arnoux, Magalie Barth, Dries Dobbelaere, Magali Gorce, Michael Grövdal, Cécile Laroche, Olivia Rime, Manuel Schiff, Mattias Rudebeck +10 morewiley +1 more sourceImproving PARP inhibitor efficacy in bladder cancer without genetic BRCAness by combination with PLX51107
Molecular Oncology, Volume 20, Issue 3, Page 779-803, March 2026.Clinical trials on PARP inhibitors in urothelial carcinoma (UC) showed limited efficacy and a lack of predictive biomarkers. We propose SLFN5, SLFN11, and OAS1 as UC‐specific response predictors. We suggest Talazoparib as the better PARP inhibitor for UC than Olaparib.Jutta Schmitz, Anna L. Bartkowiak, Michael Rose, Nora Kolks, Patrick Petzsch, Vandana Solanki, Anne Stoffel, Bianca Faßbender, Leandra Lepping, Julka Volkamer, Karl Köhrer, Marc Seifert, Tokameh Mahmoudi, Tahlita C. M. Zuiverloon, Günter Niegisch, Michèle J. Hoffmann +15 morewiley +1 more sourcePARP AND PARG INHIBITION SELECTIVELY IMPAIR METABOLISM OF B-ALL CELL LINES AND INDUCE CYTOTOXICITY THROUGH DISTINCT CELL-DEATH PATHWAYS
Hematology, Transfusion and Cell TherapyBackground: Adult patients afflicted with Acute Lymphoblastic Leukemia (ALL) still represent a subset of poor prognosis, with a 5-year overall survival of around 46%.CB Machado, KS Esteves, SCG Lima, IP Furtado, M Brandão, FMCP Pessoa, BMD Nogueira, MEA Moraes, LEB Souza, CFA Moreira-Nunes +9 moredoaj +1 more sourceA ribose-functionalized NAD+ with unexpected high activity and selectivity for protein poly-ADP-ribosylation. [PDF]
, 2019 Nicotinamide adenine dinucleotide (NAD+)-dependent ADP-ribosylation plays important roles in physiology and pathophysiology. It has been challenging to study this key type of enzymatic post-translational modification in particular for protein poly-ADP ...Chen, Jingwen, Cheng, Qinqin, Dai, Zhefu, Evdokimov, Nikolai M, Lam, Albert T, Louie, Stan G, Lu, Yanran, Pei, Hua, Zhang, Xiao-Nan, Zhang, Yong +9 morecore +1 more sourceTSG101 Promotes SIAH1 Auto‐Ubiquitination to Drive Migration and Invasion in Hepatocellular Carcinoma Cells
Journal of Cellular and Molecular Medicine, Volume 30, Issue 6, March 2026.ABSTRACT
Our previous studies have demonstrated that seven in absentia homologue 1 (SIAH1), an E3 ubiquitin ligase, is downregulated in hepatocellular carcinoma (HCC) and regulates substrate ubiquitination. However, the molecular mechanisms governing reduced SIAH1 expression in HCC remain unclear.Jie Qiu, Xixi Gu, Kai Wu, Jinzhao Wu, Chenrui Xu, Wengang Shan, Hengliang Shi +6 morewiley +1 more sourceIntrinsic signaling pathways modulate targeted protein degradation
Nature CommunicationsTargeted protein degradation is a groundbreaking modality in drug discovery; however, the regulatory mechanisms are still not fully understood. Here, we identify cellular signaling pathways that modulate the targeted degradation of the anticancer target ...Yuki Mori, Yoshino Akizuki, Rikuto Honda, Miyu Takao, Ayaka Tsuchimoto, Sota Hashimoto, Hiroaki Iio, Masakazu Kato, Ai Kaiho-Soma, Yasushi Saeki, Jun Hamazaki, Shigeo Murata, Toshikazu Ushijima, Naoko Hattori, Fumiaki Ohtake +14 moredoaj +1 more sourcePotential biological role of poly (ADP-ribose) polymerase (PARP) in male gametes [PDF]
, 2009 Maintaining the integrity of sperm DNA is vital to reproduction and male fertility. Sperm contain a number of molecules and pathways for the repair of base excision, base mismatches and DNA strand breaks.Ashok Agarwal, Reda Z Mahfouz, Rakesh K Sharma, Oli Sarkar, Devna Mangrola, Premendu P Mathur, T Ogawa, F Suzuki-Toyota, K Toshimori, RJ Aitken, I Amiri, F Kappes, DN Tripathi, D Sakkas, P Patrizio, E Seli, J Tesarik, MM El-Domyati, RM Laberge, RM Laberge, F Leduc, RK Sharma, A Agarwal, A Agarwal, A Agarwal, A Agarwal, M Bungum, J Erenpreiss, M Muratori, SE Lewis, K Tremellen, TE Schmid, KC Chitra, P Mathur, AK Olsen, JC Rockett, B Saradha, N Desai, A Agarwal, D Gero, JP Gagne, AM Reed, S Di Meglio, AP Sinha Hikim, ML Meyer-Ficca, L Atorino, D Sakkas, O Stahl, RZ Mahfouz, SE Lewis, A Zini, S Horak, Z Li, MH Nasr-Esfahani, C Niederberger, C Paul, V Schreiber, C Flohr, JC Ame, PO Hassa, DA Wacker, V Schreiber, A Augustin, M Baumgartner, BW Cherney, BC Woodhouse, D D'Amours, PO Hassa, MF Langelier, Z Tao, V Schreiber, E Pion, M Altmeyer, T Zaremba, N Ogata, J Diefenbach, H Mendoza-Alvarez, P Adamietz, GG Poirier, L Atorino, H Mendoza-Alvarez, K Yoshihara, AI Scovassi, M Kameoka, K Huang, PI Bauer, U Kolthur-Seetharam, G Ermak, T Schwerdtle, R Midorikawa, E Mocchegiani, E Kun, L Geraets, TM Kauppinen, Y Ariumi, M Audebert, J Chen, P Kotoglou, JB Leppard, F Dantzer, JL Parsons, D Sagan, D Quenet, O Mortusewicz, O Ullrich, J Mathieu, JT Heeres, FJ Oliver, M Zerfaoui, SH Kaufmann, D D'Amours, SW Yu, F Tramontano, A Tulin, NA Berger, SA Andrabi, M Kanai, P Chang, M Kanai, E Earle, A Saxena, MR Faraone-Mennella, WL Kraus, WL Kraus, D Quenet, JY Huang, GG Poirier, M Rouleau, R Krishnakumar, SL Oei, T Yazawa, K Ye, K Mizuno, O Varisli, M Tanori, V Spina-Purrello, W Yu, NA Thornberry, O Ullrich, E Klenova, T Guastafierro, E Winstall, L Davidovic, DW Koh, C Blenn, AE Fisher, JS Baum, CG Print, VA Codelia, Y Ohashi, V Spina-Purrello, M Tewari, P Pacher, PJ Duriez, F Hadziselimovic, WW Lin, SK Kim, J Tesarik, BB Maymon, F Dantzer, AP Hikim, M Miura, R Aguilar-Quesada, V Pentikainen, A Chiarugi, S Di Meglio, P Quesada, D Wouters-Tyrou, WS Ward, L Marcon, ML Meyer-Ficca, A Kaipia, SL Taylor, R Jha, M Benchaib, J Kopeika, M Meseguer, C O'Flaherty, A Edelstein, JR Spermon, RT Mitchell, T Wang, Y Wang, GJ Olsen, IV Kovtun, IV Kovtun, IV Kovtun, E Trushina, D Rybaczek, F Leduc, P Quesada, F Tramontano, S Jaroudi, C Marchetti, U Paasch, S Grunewald, C Almeida, SL Weng, U Paasch, L Faleiro, B Buelow, B Xu, G Blanc-Layrac, MM El-Domyati, RZ Mahfouz, JC Kefer, K Makker, M Reddy, R Mahfouz, S Vaithinathan, O Sarkar, B Saradha, O Sarkar, K Erdelyi, H Chen, PG Chandak, SF El-Khamisy, Y Liang, M Ljungman, SH Huang, P Lin, Q Ma, S Beneke, O Cohausz, HJ Chiu, F Dantzer, M Gomez, S Smith, CM Simbulan-Rosenthal, F d'Adda di Fagagna, RJ Aitken, RJ Aitken, RZ Mahfouz, S Adhikari, CA de la Lastra, P Zhang +235 morecore +1 more sourceSabotaged Integral HSC Heterogeneity Underlies Essential Thrombocythemia Development
Advanced Science, Volume 13, Issue 7, 3 February 2026.Single‐cell RNA sequencing (scRNA‐seq) maps how distinct driver mutations remodel hematopoietic stem cell (HSC) programs across essential thrombocythemia (ET). Comparative analysis uncovers both shared and subtype‐specific molecular signatures, identifies a triple‐negative (TN)‐associated HSC population enriched with malignant traits, and reveals the ...Jingyuan Tong, Di Wang, Haoze Song, Ting Sun, Yanhong Zhao, Wenjing Gu, Lexuan Lin, Yitong Zhao, Yipeng Liang, Xu Jin, Rongfeng Fu, Mankai Ju, Jie Gao, Jinfa Ma, Chengjie Gao, Anqi Zhang, Zhijian Xiao, Erlie Jiang, Renchi Yang, Shihui Ma, Lei Zhang, Lihong Shi +21 morewiley +1 more source