Research Progress in Diseases Caused by STAT1 Gain-of-Function Mutations by LI Linpeng, MA Jing, GU Hao, SHU Zhou, MAO Huawei

“…For the treatment of STAT1-GOF, such as targeted therapy with ruxolitinib for STAT1 hyperphosphorylation, immunomodulatory therapy and hematopoietic stem cell transplantation for different self-immune systems, certain curative effects can be obtained. …”
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Inborn errors of immunity (primary immunodeficiencies) by Vy H. D. Kim, Julia E. M. Upton, Beata Derfalvi, Kyla J. Hildebrand, Christine McCusker

“…The treatment of IEIs is complex and generally requires both supportive and definitive strategies, including but not limited to, immunoglobulin replacement therapy, antibiotic prophylaxis, immune response modifiers, and hematopoietic stem cell transplantation. This article provides an overview of the major categories of IEIs and strategies for the appropriate diagnosis and management of these disorders.…”
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Hepatic sinusoidal obstruction syndrome (SOS) associated with checkpoint inhibitor therapy by Marianna Litterio, Nicole Marchetti, Francesco Carubbi, Marianna Tudini, Luciano Mutti, Claudio Ferri

“…Sinusoidal obstruction syndrome (SOS) is a distinctive and potentially fatal form of hepatic injury that mainly occurs after hematopoietic-stem cell transplantation but also due to many other conditions including drug or toxin exposure. …”
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Parallel single-cell metabolic analysis and extracellular vesicle profiling reveal vulnerabilities with prognostic significance in acute myeloid leukemia by Dorian Forte, Roberto Maria Pellegrino, Paolo Falvo, Paulina Garcia-Gonzalez, Husam B. R. Alabed, Filippo Maltoni, Davide Lombardi, Samantha Bruno, Martina Barone, Federico Pasini, Francesco Fabbri, Ivan Vannini, Benedetta Donati, Gianluca Cristiano, Chiara Sartor, Simona Ronzoni, Alessia Ciarrocchi, Sandra Buratta, Lorena Urbanelli, Carla Emiliani, Simona Soverini, Lucia Catani, Francesco Bertolini, Rafael José Argüello, Michele Cavo, Antonio Curti

“…Extracellular vesicles from AML are mainly enriched in stem cell markers and express antioxidant GPX3, with their profiles showing potential prognostic value. …”
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Accelerating biopharmaceutical cell line selection with label-free multimodal nonlinear optical microscopy and machine learning by Jindou Shi, Alexander Ho, Corey E. Snyder, Eric J. Chaney, Janet E. Sorrells, Aneesh Alex, Remben Talaban, Darold R. Spillman, Marina Marjanovic, Minh Doan, Gary Finka, Steve R. Hood, Stephen A. Boppart

“…The techniques have potential for broader single-cell characterization applications in stem cell research, immunology, cancer biology and beyond.…”
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Spontaneous high clonal expansion of Wilms’ tumor gene 1-specific cytotoxic T-lymphocytes in patients with Wilms’ tumor gene 1-expressing solid tumor by Soyoko Morimoto, Yukie Tanaka, Jun Nakata, Fumihiro Fujiki, Kana Hasegawa, Hiroko Nakajima, Sumiyuki Nishida, Akihiro Tsuboi, Naoki Hosen, Naoki Kagawa, Motohiko Maruno, Akira Myoui, Takayuki Enomoto, Shuichi Izumoto, Mitsugu Sekimoto, Naoya Hashimoto, Toshiki Yoshimine, Atsushi Kumanogoh, Yusuke Oji, Yoshihiro Oka, Haruo Sugiyama

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A synthetic biological secondary metabolite, Lycogenâ¢, produced and extracted from Rhodobacter sphaeroides WL-APD911 in an optimizatioal scale-up strategy by Cheng-Chin Wang, Shi-Ying Huang, Shu-Hung Huang, Zhi-Hong Wen, Jyun-Ying Huang, Wen-Sheng Liu, Hui-Min David Wang

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Human respiratory organoids sustained reproducible propagation of human rhinovirus C and elucidation of virus-host interaction by Cun Li, Yifei Yu, Zhixin Wan, Man Chun Chiu, Jingjing Huang, Shuxin Zhang, Xiaoxin Zhu, Qiaoshuai Lan, Yanlin Deng, Ying Zhou, Wei Xue, Ming Yue, Jian-Piao Cai, Cyril Chik-Yan Yip, Kenneth Kak-Yuen Wong, Xiaojuan Liu, Yang Yu, Lin Huang, Hin Chu, Jasper Fuk-Woo Chan, Hans Clevers, Kwok Yung Yuen, Jie Zhou

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Necrotizing Fasciitis in Paroxysmal Nocturnal Hemoglobinuria by Pusem Patir, Yakup Isik, Yigit Turk, Mehmet Can Ugur, Cengiz Ceylan, Gulnur Gorgun, Nihal Mete Gokmen, Guray Saydam, Fahri Sahin

“…Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, progressive, and life-threatening hematopoietic stem cell disorder characterized by complement-mediated intravascular hemolysis and a prothrombotic state. …”
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Adult Hippocampal Neurogenesis in Parkinson’s Disease: Impact on Neuronal Survival and Plasticity by Martin Regensburger, Iryna Prots, Beate Winner

“…The pathology of the adult neurogenic niches in human PD patients is still mostly elusive, but different preclinical models have shown profound alterations of adult neurogenesis. Alterations in stem cell proliferation, differentiation, and survival as well as neurite outgrowth and spine formation have been related to different aspects in PD pathogenesis. …”
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Allogeneic CD33-directed CAR-NKT cells for the treatment of bone marrow-resident myeloid malignancies by Yan-Ruide Li, Ying Fang, Siyue Niu, Yichen Zhu, Yuning Chen, Zibai Lyu, Enbo Zhu, Yanxin Tian, Jie Huang, Valerie Rezek, Scott Kitchen, Tzung Hsiai, Jin J. Zhou, Pin Wang, Wanxing Chai-Ho, Sunmin Park, Christopher S. Seet, Caspian Oliai, Lili Yang

“…In this study, we characterize primary patient samples and identify a unique therapeutic opportunity for CAR-engineered invariant natural killer T (CAR-NKT) cells. Using stem cell gene engineering and a clinically guided culture method, we generate allogeneic CD33-directed CAR-NKT cells with high yield, purity, and robustness. …”
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Translocation (6;15)(q12;q15): A Novel Mutation in a Patient with Therapy-Related Myelodysplastic Syndrome by Saba F. Ali, Rebecca J. Sonu, Denis M. Dwyre, Brian A. Jonas, Hooman H. Rashidi

“…Patients with t-MDS, a group of heterogeneous stem cell related disorders resulting as a latent complication of cytotoxic and/or radiation therapy, generally tend to have a poorer prognosis than de novo MDS. …”
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High frequency CCR5 editing in human hematopoietic stem progenitor cells protects xenograft mice from HIV infection by Daniel T. Claiborne, Zachary Detwiler, Steffen S. Docken, Todd D. Borland, Deborah Cromer, Amanda Simkhovich, Youdiil Ophinni, Ken Okawa, Timothy Bateson, Tao Chen, Wesley Hudson, Radiana Trifonova, Miles P. Davenport, Tony W. Ho, Christian L. Boutwell, Todd M. Allen

“…Abstract The only cure of HIV has been achieved in a small number of people who received a hematopoietic stem cell transplant (HSCT) comprising allogeneic cells carrying a rare, naturally occurring, homozygous deletion in the CCR5 gene. …”
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A patient-based iPSC-derived hepatocyte model of alcohol-associated cirrhosis reveals bioenergetic insights into disease pathogenesis by Bani Mukhopadhyay, Cheryl Marietta, Pei-Hong Shen, Abdul Oiseni, Faridoddin Mirshahi, Maria Mazzu, Colin Hodgkinson, Eli Winkler, Qiaoping Yuan, Daniel Miranda, George Kunos, Arun J. Sanyal, David Goldman

“…Induced pluripotent stem cell-derived hepatocytes (iHLCs) from patients with AC and healthy controls differ transcriptomically, bioenergetically and histologically. …”
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Intelligent in-cell electrophysiology: Reconstructing intracellular action potentials using a physics-informed deep learning model trained on nanoelectrode array recordings by Keivan Rahmani, Yang Yang, Ethan Paul Foster, Ching-Ting Tsai, Dhivya Pushpa Meganathan, Diego D. Alvarez, Aayush Gupta, Bianxiao Cui, Francesca Santoro, Brenda L. Bloodgood, Rose Yu, Csaba Forro, Zeinab Jahed

“…This study presents an AI-supported technique that leverages thousands of synchronous eAP and iAP pairs from stem-cell-derived cardiomyocytes on NEAs. Our analysis revealed strong correlations between specific eAP and iAP features, such as amplitude and spiking velocity, indicating that extracellular signals could be reliable indicators of intracellular activity. …”
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N-Cadherin promotes cardiac regeneration by potentiating pro-mitotic β-Catenin signaling in cardiomyocytes by Yi-Wei Tsai, Yi-Shuan Tseng, Yu-Shuo Wu, Wei-Lun Song, Min-Yi You, Yun-Chia Hsu, Wen-Pin Chen, Wei-Han Huang, Jia-Ci Chng, Chai-Ling Lim, Ke-Hsuan Wei, Shih-Lei Ben Lai, Wen-Chih Lee, Kai-Chien Yang

“…N-Cadherin knockdown reduces, whereas overexpression increases, the proliferation activity of neonatal mouse CMs and human induced pluripotent stem cell-derived CMs. Mechanistically, N-Cadherin binds and stabilizes pro-mitotic transcription regulator β-Catenin, driving CM self-renewal. …”
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The different faces of GATA2 deficiency: implications for therapy and surveillance by Luca Vinci, Brigitte Strahm, Carsten Speckmann, Carsten Speckmann, Miriam Erlacher, Miriam Erlacher

“…Due to the elevated risk of progression, nearly all individuals with GATA2-related MDS eventually undergo a hematopoietic stem cell transplantation (HSCT) at some point in their lives. …”
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Secondary Primary Malignancies in Multiple Myeloma: An Old Nemesis Revisited by Jay Yang, Howard R. Terebelo, Jeffrey A. Zonder

“…High-dose chemotherapy with autologous stem cell support became widely accepted for myeloma in the 1990s. …”
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Sequential Kinase Inhibition (Idelalisib/Ibrutinib) Induces Clinical Remission in B-Cell Prolymphocytic Leukemia Harboring a 17p Deletion by H. Coelho, M. Badior, T. Melo

“…Herein, we describe, for the first time, a case of B-cell prolymphocytic leukemia harboring a 17p deletion in a 48-year-old man that was successfully treated sequentially with idelalisib-rituximab/ibrutinib followed by allogeneic hematopoietic stem cell transplant (allo-HSCT). After 5 months of therapy with idelalisib-rituximab, clinical remission was achieved, but the development of severe diarrhea led to its discontinuation. …”
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Septic Shock Induced by Bacterial Prostatitis with Morganella morganii subsp. morganii in a Posttransplantation Patient by Xiaofan Li, Jianhui Chen

“…Bacterial infection is a common complication after Hematopoietic Stem Cell Transplantation (HSCT). Morganella morganii is ubiquitous Gram-negative facultative anaerobe, which may cause many kinds of opportunistic infection. …”
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