tbuckley/cabot-search huggingface dataset:
Abstract 546: S-palmitoylation Mediates Caveolae Localization and Limits Cysteine Oxidation of Gc-1 in Cardiomyocytes
Authors: Christos Tzimas, Ilaria Russo, Wen Dun, Sevde Yilmaz, Sudarsan Rajan, Emily J Tsai
Journal: Circulation Research (2020)

Objectives: The nitric oxide (NO) receptor soluble guanylyl cyclase (GC-1) can localize to cell membrane microdomains, including caveolae. In failing hearts, GC-1 relocalizes away from caveolae and becomes oxidized. How this occurs is unknown. We hypothesized that S-palmitoylation, a reversible, enzyme-catalyzed, post-translational modification that enhances membrane affinity of protein substrates, mediates caveolae-localization and modulates redox regulation of GC-1. Methods: Adult male C57BL/6 mice were subjected to transverse aortic constriction (TAC) versus thoracotomy (Sham). Modified acyl-biotin exchange (ABE) assays were performed to assess S-palmitoylation of GC-1 in failing TAC vs non-failing Sham hearts. Using bioinformatics, we identified 5 conserved cysteines in the GC-1α subunit as potential S-palmitoylation sites. We created cysteine-serine mutants of GC-1α at each and all 5 sites (C3S, C15S, C176S, C497S, C595S, CS*). Mutants and WT GC-1α were expressed in neonatal rat cardiomyocytes (NRCM) and their palmitoylation status compared by ABE. We assessed localization and oxidation of GC-1α (CS* vs WT) by confocal microscopy and redox Western, respectively. Results: In TAC hearts, S-palmitoylation of GC-1α was half of that in Sham ( p =0.02). Likewise, palmitoyl-transferase ZDHHC16 was downregulated in TAC vs Sham by 31% ( p =0.01) at the transcript level and by 56% ( p =0.03) at the protein level. In NRCMs, S-palmitoylation of CS* was markedly diminished (6.9%, p &lt;0.001), while that of C15S, C176S, and C497S were modestly diminished (23.8%, p=0.03; 23.2%, p=0.01; 22.8%, p=0.01), compared to WT (54.7%). Moreover, S-palmitoylation of WT but not CS* was decreased by palmitoylation inhibitor 2-BP (17.8%, p&lt;0.0001). Confocal microscopy showed more diffuse cytosolic distribution of CS* compared to WT. Redox Westerns revealed increased oxidation of CS* vs WT upon H 2 O 2 treatment (1.7-fold increase, p=0.008). Conclusions: Our data support that GC-1α is S-palmitoylated at C15, C176, and C497. S-palmitoylation of GC-1α at these 3 cysteines appears to protect GC-1α against oxidation and to mediate its membrane localization.
Long-Term Outcomes and Health-Related Quality of Life (HRQoL) By Response Status for Bortezomib, Melphalan, and Prednisone (VMP) ± Daratumumab (DARA) in Alcyone
Authors: Paula Rodriguez-Otero, Mario Boccadoro, Roman Hajek, Tomoaki Fujisaki, Jae Hoon Lee, Joaquin Martinez-Lopez, Paulo Lucio, Zsolt Nagy, Ganna Usenko, Anna Marina Liberati, Mihaela Lazaroiu, Dariusz Woszczyk, Hiroyuki Takamatsu, Joanna Romejko-Jarosinska, Stefan Tobias Knop, Astrid Pavlovsky, Cecily Forsyth, Takayuki Ishikawa, Katharine S. Gries, Huiling Pei, Anupa Kudva, Jon Ukropec, Susan Wroblewski, Robin Carson, Meletios A Dimopoulos
Journal: Blood (2020)
Covid-19: NHS hospitals are urged to recruit more patients to Recovery trial to find what treatments work
Authors: Gareth Iacobucci
Journal: BMJ (2020)

Covid-19: NHS hospitals are urged to recruit more patients to Recovery trial to find what treatments work Gareth IacobucciThe head of the UK's Recovery trial has urged NHS hospitals to recruit more patients to the study in order to speed up the development of treatments for covid-19.Recovery (randomised evaluation of covid-19 therapy) is the world's largest randomised trial of covid-19 treatments, recruiting around 150 patients a day.
Prognostication for Advanced Stage Hodgkin Lymphoma (HL) in the Modern Era: A Project from the Hodgkin Lymphoma International Study for Individual Care (HoLISTIC) Consortium
Authors: Angie Mae Rodday, Susan K Parsons, Carlton Scharman, Ranjana Advani, Massimo Federico, Jonathan W. Friedberg, Andrea Gallamini, David C. Hodgson, Peter Hoskin, Martin Hutchings, Peter Johnson, Kara M. Kelly, Brian K. Link, John A. Radford, Pier Luigi Zinzani, James R. Cerhan, John Raemaekers, Andrew M. Evens, On Behalf Of The HoLISTIC Consortium
Journal: Blood (2020)
PTRF/cavin-1 remodels phospholipid metabolism to promote tumor proliferation and suppress immune responses in glioblastoma by stabilizing cPLA2
Authors: Kaikai Yi, Qi Zhan, Qixue Wang, Yanli Tan, Chuan Fang, Yunfei Wang, Junhu Zhou, Chao Yang, Yansheng Li, Chunsheng Kang
Journal: Neuro-Oncology (2020)

Metabolism remodeling is a hallmark of glioblastoma (GBM) that regulates tumor proliferation and the immune microenvironment. Previous studies have reported that increased polymerase 1 and transcript release factor (PTRF) levels are associated with a worse prognosis in glioma patients. However, the biological role and the molecular mechanism of PTRF in GBM metabolism remain unclear.The relationship between PTRF and lipid metabolism in GBM was detected by nontargeted metabolomics profiling and subsequent lipidomics analysis. Western blotting, quantitative real-time PCR, and immunoprecipitation were conducted to explore the molecular mechanism of PTRF in lipid metabolism. A sequence of in vitro and in vivo experiments (both xenograft tumor and intracranial tumor mouse models) were used to detect the tumor-specific impacts of PTRF.Here, we show that PTRF triggers a cytoplasmic phospholipase A2 (cPLA2)-mediated phospholipid remodeling pathway that promotes GBM tumor proliferation and suppresses tumor immune responses. Research in primary cell lines from GBM patients revealed that cells overexpressing PTRF show increased cPLA2 activity-resulting from increased protein stability-and exhibit remodeled phospholipid composition. Subsequent experiments revealed that PTRF overexpression alters the endocytosis capacity and energy metabolism of GBM cells. Finally, in GBM xenograft and intracranial tumor mouse models, we showed that inhibiting cPLA2 activity blocks tumor proliferation and prevents PTRF-induced reduction in CD8+ tumor-infiltrating lymphocytes.The PTRF-cPLA2 lipid remodeling pathway promotes tumor proliferation and suppresses immune responses in GBM. In addition, our findings highlight multiple new therapeutic targets for GBM.
Abstract MP173: Loss of DCHS1 Promotes Mitral Valve Prolapse Through Cytoskeleton Destabilization
Authors: Reece Moore, Kelsey Moore, Rebecca Stairley, Diana B Fulmer, Lilong Guo, Russell A Norris
Journal: Circulation Research (2020)

Introduction: Mitral valve prolapse (MVP) is a major source of morbidity and mortality and is becoming one of the most common indications for cardiac surgery. Our group has previously reported that MVP is associated with missense mutations in DCHS1 , a gene widely implicated in tissue development and organization. Purpose: We recently performed two-hybrid screens and co-immunoprecipitation assays that define a novel complex between DCHS1, LIX1L, and SEPT9 (DLS). Our current studies investigate the role of this interaction in mitral valve morphogenesis. Methods: Wild-type (WT), DCHS1, and/or LIX1L heterozygous neonate murine heart tissue was isolated for primary culture of cardiac fibroblasts (CFs) and analyzed with immunocytochemistry, collagen, compaction, fibrin pillar to post assays, and actin sedimentation assays. Nuclear morphology and chromatin density were conducted for functional read-outs of cytoskeletal aberrations. Morphologic and functional analyses of murine mitral valves were completed. Results: Loss of actin architecture and collagen compaction capability were seen in CFs deficient of DCHS1 and/or LIX1L. Delivery of a peptide that disrupted the LIX1L-SEPT9 binding domain likewise destroyed actin architecture and reduced ECM compaction. In-vivo epistasis studies support these findings as mitral valve enlargement was exacerbated in DCHS1/LIX1L compound heterozygote mice compared to DCHS1 and LIX1L heterozygotes. Echocardiography of 11-month DCHS1/LIX1L compound heterozygote mice revealed pronounced MVP and increased left atrium diameter relative to DCHS1 and LIX1L heterozygotes. Filamentous SEPTIN9 localized to the actin ultrastructure and was also lost with disturbance of the DLS complex. Nuclei stretch and chromatin compaction were reduced in DCHS1/LIX1L compound heterozygote CFs. Further, histone H4 modifications were observed in DCHS1 KO mouse embryonic fibroblasts that indicate reductions in gene expression. Conclusion: DCHS1, through its interaction with the septin cytoskeleton, must regulate and stabilize actin dynamics in mitral VICs leading to proper valve development. Non-surgical interventions for MVP may be identified through establishing the downstream effects of DCHS1 mutations in our model.
Outcomes after Second Allogeneic Transplantation and Donor Lymphocyte Infusion for Relapse after a First Allogeneic Transplant
Authors: Christopher Lemieux, Lori S. Muffly, Sally Arai, Andrew R. Rezvani, Surbhi Sidana, Laura J. Johnston, Wen-Kai Weng, Parveen Shiraz, Matthew J. Frank, Everett H Meyer, Judith A Shizuru, David B. Miklos, Robert S. Negrin, Robert Lowsky
Journal: Blood (2020)
Translational Approach of Using Ex Vivo Cytotoxicity and Early Clinical Data to Predict Teclistamab Efficacious Therapeutic Range in Multiple Myeloma Patients
Authors: Suzette Girgis, Shun Xin Wang Lin, Kodandaram Pillarisetti, Arnob Banerjee, Jenna D. Goldberg, Shoba Shetty, Tara Stephenson, Brandi W. Hilder, Brett Hanna, Jennifer Smit, Homer Adams III, Yu-Nien (Tom) Sun, Jeffrey R. Infante, Yusri A Elsayed, Amarnath Sharma
Journal: Blood (2020)
Harnessing consumer smartphone and wearable sensors for clinical cancer research
Authors: Carissa A. Low
Journal: npj Digital Medicine (2020)

Abstract As smartphones and consumer wearable devices become more ubiquitous, there is a growing opportunity to capture rich mobile sensor data continuously, passively, and in real-world settings with minimal burden. In the context of cancer, changes in these passively sensed digital biomarkers may reflect meaningful variation in functional status, symptom burden, quality of life, and risk for adverse clinical outcomes. These data could enable real-time remote monitoring of patients between clinical encounters and more proactive, comprehensive, and personalized care. Over the past few years, small studies across a variety of cancer populations support the feasibility and potential clinical value of mobile sensors in oncology. Barriers to implementing mobile sensing in clinical oncology care include the challenges of managing and making sense of continuous sensor data, patient engagement issues, difficulty integrating sensor data into existing electronic health systems and clinical workflows, and ethical and privacy concerns. Multidisciplinary collaboration is needed to develop mobile sensing frameworks that overcome these barriers and that can be implemented at large-scale for remote monitoring of deteriorating health during or after cancer treatment or for promotion and tailoring of lifestyle or symptom management interventions. Leveraging digital technology has the potential to enrich scientific understanding of how cancer and its treatment affect patient lives, to use this understanding to offer more timely and personalized support to patients, and to improve clinical oncology outcomes.
Results from Lummicar-2: A Phase 1b/2 Study of Fully Human B-Cell Maturation Antigen-Specific CAR T Cells (CT053) in Patients with Relapsed and/or Refractory Multiple Myeloma
Authors: Shaji K. Kumar, Rachid C. Baz, Robert Z. Orlowski, Larry D. Anderson, Hong Ma, Alexandria Shrewsbury, Katrina A. Croghan, Mrinalini Bilgi, Ankit Kansagra, Prashant Kapoor, Zonghai Li, Jason Brayer
Journal: Blood (2020)
Effect of Intravenous Immunoglobulin on Infections in Multiple Myeloma (MM) Patients Receiving Daratumumab
Authors: Guido Lancman, Dahniel Sastow, Minira Aslanova, Erin Moshier, Hearn Jay Cho, Sundar Jagannath, Deepu Madduri, Samir Parekh, Shambavi Richard, Joshua Richter, Larysa Sanchez, Ajai Chari
Journal: Blood (2020)
Baseline Biomarkers of T-Cell Function Correlate with Clinical Responses to Odronextamab (REGN1979), and Loss of CD20 Target Antigen Expression Identified As a Mechanism of Treatment Resistance
Authors: Jurriaan Brouwer-Visser, Nathalie Fiaschi, Raquel P. Deering, Ankur Dhanik, Kamil J. Cygan, Wen Zhang, Se Jeong, Stephane Pourpe, Lauren Boucher, Sara Hamon, Max S. Topp, Rajat Bannerji, Johannes Duell, Ranjana Advani, Dina M. Flink, Aafia Chaudhry, Andres Sirulnik, Andrew J. Murphy, David M. Weinreich, George D. Yancopoulos, Gavin Thurston, Srikanth R. Ambati, Vladimir Jankovic
Journal: Blood (2020)
DNA methylation based glioblastoma subclassification is related to tumoral T-cell infiltration and patient survival
Authors: Joost Dejaegher, Lien Solie, Zoé Hunin, Raf Sciot, David Capper, Christin Siewert, Sofie Van Cauter, Guido Wilms, Johan van Loon, Nadine Ectors, Steffen Fieuws, Stefan M Pfister, Stefaan W Van Gool, Steven De Vleeschouwer
Journal: Neuro-Oncology (2020)

Histologically classified glioblastomas (GBM) can have different clinical behavior and response to therapy, for which molecular subclassifications have been proposed. We evaluated the relationship of epigenetic GBM subgroups with immune cell infiltrations, systemic immune changes during radiochemotherapy, and clinical outcome. 450K genome-wide DNA methylation was assessed on tumor tissue from 93 patients with newly diagnosed GBM, treated with standard radiochemotherapy and experimental immunotherapy. Tumor infiltration of T cells, myeloid cells, and Programmed cell death protein 1 (PD-1) expression were evaluated. Circulating immune cell populations and selected cytokines were assessed on blood samples taken before and after radiochemotherapy. Forty-two tumors had a mesenchymal, 27 a receptor tyrosine kinase (RTK) II, 17 RTK I, and 7 an isocitrate dehydrogenase (IDH) DNA methylation pattern. Mesenchymal tumors had the highest amount of tumor-infiltrating CD3+ and CD8+ T cells and IDH tumors the lowest. There were no significant differences for CD68+ cells, FoxP3+ cells, and PD-1 expression between groups. Systemically, there was a relative increase of CD8+ T cells and CD8+ PD-1 expression and a relative decrease of CD4+ T cells after radiochemotherapy in all subgroups except IDH tumors. Overall survival was the longest in the IDH group (median 36 mo), intermediate in RTK II tumors (27 mo), and significantly lower in mesenchymal and RTK I groups (15.5 and 16 mo, respectively). Methylation based stratification of GBM is related to T-cell infiltration and survival, with IDH and mesenchymal tumors representing both ends of a spectrum. DNA methylation profiles could be useful in stratifying patients for immunotherapy trials.
The pivotal roles of exosomes derived from endogenous immune cells and exogenous stem cells in myocardial repair after acute myocardial infarction
Authors: Yu-Yan Xiong, Zhao-Ting Gong, Rui-Jie Tang, Yue-Jin Yang
Journal: Theranostics (2020)

Acute myocardial infarction (AMI) is one of the leading causes of mortality around the world, and the inflammatory response plays a pivotal role in the progress of myocardial necrosis and ventricular remodeling, dysfunction and heart failure after AMI. Therapies aimed at modulating immune response after AMI on a molecular and cellular basis are urgently needed. Exosomes are a type of extracellular vesicles which contain a large amount of biologically active substances, like lipids, nucleic acids, proteins and so on. Emerging evidence suggests key roles of exosomes in immune regulation post AMI. A variety of immune cells participate in the immunomodulation after AMI, working together to clean up necrotic tissue and repair damaged myocardium. Stem cell therapy for myocardial infarction has long been a research hotspot during the last two decades and exosomes secreted by stem cells are important active substances and have similar therapeutic effects of immunomodulation, anti-apoptosis, anti-fibrotic and angiogenesis to those of stem cells themselves. Therefore, in this review, we focus on the characteristics and roles of exosomes produced by both of endogenous immune cells and exogenous stem cells in myocardial repair through immunomodulation after AMI.
DYRK1A Is Required to Alleviate Replication Stress in <i>KMT2A</i>-Rearranged Acute Lymphoblastic Leukemia
Authors: Christian Hurtz, Martin P. Carroll, Sarah K Tasian, Gerald Wertheim, Rahul S. Bhansali, Sung June Lee, Anil Kumar, Anne Lehman, Joseph Patrick Loftus, Grace Jeschke, John D. Crispino, Thierry Besson, Junwei Shi, Srividya Swaminathan
Journal: Blood (2020)
Efficacy and Safety of Blinatumomab for Relapsed or Refractory Pediatric ALL Patients: A Systematic Review
Authors: Anam Khan, Atif Irfan Khan, Sana Irfan Khan, Sobia Aamir, Usman Ali Akbar, Anum Waqar, Muhammad Ashar Ali, Abdul Jabbar Dar, Richi Kashyap, Saad Ur Rahman, Muhammad Ali Mirza, Qasim Khurshid, Faiz Anwer
Journal: Blood (2020)
Hedgehog signaling in gastrointestinal carcinogenesis and the gastrointestinal tumor microenvironment
Authors: Jinghui Zhang, Jiajun Fan, Xian Zeng, Mingming Nie, Jingyun Luan, Yichen Wang, Dianwen Ju, Kai Yin
Journal: Acta Pharmaceutica Sinica B (2020)

The Hedgehog (HH) signaling pathway plays important roles in gastrointestinal carcinogenesis and the gastrointestinal tumor microenvironment (TME). Aberrant HH signaling activation may accelerate the growth of gastrointestinal tumors and lead to tumor immune tolerance and drug resistance. The interaction between HH signaling and the TME is intimately involved in these processes, for example, tumor growth, tumor immune tolerance, inflammation, and drug resistance. Evidence indicates that inflammatory factors in the TME, such as interleukin 6 (IL-6) and interferon-γ (IFN-γ), macrophages, and T cell-dependent immune responses, play a vital role in tumor growth by affecting the HH signaling pathway. Moreover, inhibition of proliferating cancer-associated fibroblasts (CAFs) and inflammatory factors can normalize the TME by suppressing HH signaling. Furthermore, aberrant HH signaling activation is favorable to both the proliferation of cancer stem cells (CSCs) and the drug resistance of gastrointestinal tumors. This review discusses the current understanding of the role and mechanism of aberrant HH signaling activation in gastrointestinal carcinogenesis, the gastrointestinal TME, tumor immune tolerance and drug resistance and highlights the underlying therapeutic opportunities.
Safety and Efficacy of Idasanutlin in Patients (pts) with Hydroxyurea (HU)-Resistant/Intolerant Polycythemia Vera (PV): Results of an International Phase II Study
Authors: John Mascarenhas, Brian Higgins, Doreen Anders, Kate Burbury, Tarec Christoffer El-Galaly, Aaron T. Gerds, Vikas Gupta, Bruno Kovic, Margherita Maffioli, Ruben Mesa, Jeanne M. Palmer, Francesco Passamonti, Alessandro Rambaldi, David M Ross, Alessandro M. Vannucchi, Abdulraheem Yacoub
Journal: Blood (2020)
In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial
Authors: Tippi C. MacKenzie, Michela Frascoli, Renan Sper, Billie Rachael Lianoglou, Juan Gonzalez Velez, Christopher C. Dvorak, Sandhya Kharbanda, Elliott Vichinsky
Journal: Blood (2020)
Local microbleeding facilitates IL-6– and IL-17–dependent arthritis in the absence of tissue antigen recognition by activated T cells
Authors: Masaaki Murakami, Yuko Okuyama, Hideki Ogura, Shogo Asano, Yasunobu Arima, Mineko Tsuruoka, Masaya Harada, Minoru Kanamoto, Yukihisa Sawa, Yoichiro Iwakura, Kiyoshi Takatsu, Daisuke Kamimura, Toshio Hirano
Journal: The Journal of Experimental Medicine (2012)
NF1 Tumor Suppressor Gene Inactivation in Juvenile Myelomonocytic Leukemia: New Genetic Evidence and Consequences for Diagnostic Work-up
Authors: Senthilkumar Ramamoorthy, Dirk Lebrecht, Denny Schanze, Ina Schanze, Ilse Wieland, Michael H. Albert, Arndt Borkhardt, Dorine Bresters, Jochen Büchner, Albert Catala, Valerie De Haas, Michael Dworzak, Miriam Erlacher, Henrik Hasle, Kirsi Jahnukainen, Franco Locatelli, Riccardo Masetti, Jan Stary, Dominik Turkiewicz, Luca Vinci, Marcin W. Wlodarski, Ayami Yoshimi, Maria Hess, Melanie Boerries, Charlotte M. Niemeyer, Martin Zenker, Christian Flotho
Journal: Blood (2020)
Interim Analysis of the Phase 1b/2 Study of the BCL-2 Inhibitor Venetoclax in Combination with Standard Intensive AML Induction/Consolidation Therapy with FLAG-IDA in Patients with Newly Diagnosed or Relapsed/Refractory AML
Authors: Curtis Lachowiez, Marina Konopleva, Tapan M. Kadia, Naval Daver, Sanam Loghavi, Sa A Wang, Maria Adeoti, Sherry A. Pierce, Koichi Takahashi, Nicholas J. Short, Koji Sasaki, Gautam Borthakur, Ghayas C. Issa, William G. Wierda, Naveen Pemmaraju, Guillermo Montalban Bravo, Alessandra Ferrajoli, Nitin Jain, Lucia Masarova, Musa Yilmaz, Elias Jabbour, Guillermo Garcia-Manero, Steven M. Kornblau, Farhad Ravandi, Hagop M. Kantarjian, Courtney D. Dinardo
Journal: Blood (2020)
Antibodies to Hepatitis B Surface Antigen (HBsAb) Are Acquired after Allogeneic Stem Cell Transplantation in Non-B Hepatitis Virus-Infected Patients but Disappear over Time
Authors: Jinhua Ren, Tingting Xiao, Yingxi Weng, Rong Zheng, Yu Zhang, Jingjing Xu, Xiaofeng Luo, Zhihong Zheng, Zhizhe Chen, Jianda Hu, Ting Yang
Journal: Blood (2020)
Abstract 384: Predicting Drugs and Drug Targets Associated With Dilated Cardiomyopathy Using a Gene Expression-based Systems Medicine Platform
Authors: Henri Wathieu, Sivanesan Dakshanamurthy
Journal: Circulation Research (2020)

Among the genetic and environmental contributors to dilated cardiomyopathy (DCM), the pathogenesis of drug-induced DCM is heterogeneous, poorly understood, and difficult to predict. In this study, transcriptomic data from two recently published datasets were used to identify genes which are differentially expressed in DCM compared to cardiac tissue from non-failing donors. DCM-associated genes were then mapped using gene set enrichment analysis to associated biochemical pathways, gene ontology functions, and protein-protein interactions, creating a DCM-associated biological perturbation network. Corresponding multi-scale pharmacologic activity networks for over 9000 drugs were produced using public repositories of known drug-protein interactions. Drugs were then rank-ordered based on multi-scale statistical significance of concordance with the DCM perturbation network while accounting for direction of effect using a causal analysis algorithm. A predictive algorithm using overrepresentation analysis was developed using the SIDER database of adverse drug reactions (ADRs) to formulate ADR-protein and ADR-pathway associations, which were then compared to the drug-DCM association networks produced by our model. In addition to a demonstrated concordance between the rank lists for each dataset, this platform prioritized multiple drugs and metabolites which are known to be associated with DCM, such as doxorubicin, risperidone, and 4-hydroxynonenal, as well as associated mechanisms such as bile acid excess and inhibition of DNA topoisomerase II and mitochondrial complex III. Multi-scale concordance of drug and DCM networks highlighted the involvement of heat shock proteins and associated angiotensin II activity, supporting current evidence that its inhibition may be cardioprotective to drug-induced DCM. Our approach also recapitulated the roles of drugs, genes, and pathways that hold prognostic and therapeutic significance for DCM, such as the abilities of sulforaphane and fenofibrate to prevent DCM in animal models of type 2 diabetes. This platform can be used to predict a wide array of adverse drug reactions and their underlying mechanisms based on bioactivity data.
Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients
Authors: José Luis López Lorenzo, Susana Navarro, Ami J Shah, Maria Grazia Roncarolo, Julián Sevilla, Lucía Llanos, Begoña Pérez Camino de Gaisse, Sol Sanchez, Bertil Glader, May Chien, Oscar Quintana Bustamante, Brian C Beard, Kenneth M Law, Miriam Zeini, Grace Choi, Eileen Nicoletti, Juan A Bueren, Gayatri R Rao, Jonathan D Schwartz, José C Segovia
Journal: Blood (2020)