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JOURNAL ARTICLE
Franco Locatelli, Peter Lang, Donna Wall, Roland Meisel, Selim Corbacioglu, Amanda M Li, Josu de la Fuente, Ami J Shah, Ben Carpenter, Janet L Kwiatkowski, Markus Mapara, Robert I Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Rupert Handgretinger, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William E Hobbs, Haydar Frangoul
BACKGROUND: Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of the erythroid-specific enhancer region of BCL11A in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). METHODS: We conducted an open-label, single-group, phase 3 study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β0 /β0 , β0 /β0 -like, or non-β0 /β0 -like genotype...
April 24, 2024: New England Journal of Medicine
#2
JOURNAL ARTICLE
Victor J Collins, Katelyn R Ludwig, Ariana E Nelson, Soumya Sundara Rajan, Choh Yeung, Ksenia Vulikh, Kristine A Isanogle, Arnulfo Mendoza, Simone Difilippantonio, Baktiar O Karim, Natasha J Caplen, Christine M Heske
Disruption of DNA damage repair via impaired homologous recombination is characteristic of Ewing sarcoma (EWS) cells. We hypothesize that this disruption results in increased reliance on non-homologous end joining (NHEJ) to repair DNA damage. In this study, we investigated if pharmacological inhibition of the enzyme responsible for NHEJ, the DNA-PK holoenzyme, alters the response of EWS cells to genotoxic standard of care chemotherapy. We used analyses of cell viability and proliferation to investigate the effects of clinical DNA-PK inhibitors (DNA-PKi) in combination with six therapeutic or experimental agents for EWS...
April 24, 2024: Molecular Cancer Therapeutics
#3
RANDOMIZED CONTROLLED TRIAL
Andre Bizier, Arielle Jones, Michael Businelle, Krista Kezbers, Bettina B Hoeppner, Thomas P Giordano, Jessica M Thai, Jacqueline Charles, Audrey Montgomery, Matthew W Gallagher, Marshall K Cheney, Michael Zvolensky, Lorra Garey
BACKGROUND: Black adults who smoke and have HIV experience immense stressors (eg, racial discrimination and HIV stigma) that impede smoking cessation success and perpetuate smoking-related health disparities. These stressors also place Black adults who smoke and have HIV at an increased risk of elevated interoceptive stress (eg, anxiety and uncomfortable bodily sensations) and smoking to manage symptoms. In turn, this population is more likely to smoke to manage interoceptive stress, which contributes to worse HIV-related outcomes in this group...
April 24, 2024: JMIR Research Protocols
#4
JOURNAL ARTICLE
Niklas Klümper, Ngoc Khanh Tran, Stefanie Zschäbitz, Oliver Hahn, Thomas Büttner, Florian Roghmann, Christian Bolenz, Friedemann Zengerling, Constantin Schwab, Dora Nagy, Marieta Toma, Glen Kristiansen, Hendrik Heers, Philipp Ivanyi, Günter Niegisch, Camilla Marisa Grunewald, Christopher Darr, Arian Farid, Katrin Schlack, Mahmoud Abbas, Can Aydogdu, Jozefina Casuscelli, Theresa Mokry, Michael Mayr, Dora Niedersüß-Beke, Steffen Rausch, Dimo Dietrich, Jonas Saal, Jörg Ellinger, Manuel Ritter, Abdullah Alajati, Christoph Kuppe, Joshua Meeks, Francisco E Vera Badillo, J Alberto Nakauma-González, Joost Boormans, Kerstin Junker, Arndt Hartmann, Viktor Grünwald, Michael Hölzel, Markus Eckstein
PURPOSE: The anti-NECTIN4 antibody-drug conjugate enfortumab vedotin (EV) is approved for patients with metastatic urothelial cancer (mUC). However, durable benefit is only achieved in a small, yet uncharacterized patient subset. NECTIN4 is located on chromosome 1q23.3, and 1q23.3 gains represent frequent copy number variations (CNVs) in urothelial cancer. Here, we aimed to evaluate NECTIN4 amplifications as a genomic biomarker to predict EV response in patients with mUC. MATERIALS AND METHODS: We established a NECTIN4 -specific fluorescence in situ hybridization (FISH) assay to assess the predictive value of NECTIN4 CNVs in a multicenter EV-treated mUC patient cohort (mUC-EV, n = 108)...
April 24, 2024: Journal of Clinical Oncology
#5
JOURNAL ARTICLE
Jing Yang, Chen Yang, Jianju Feng, Fandong Zhu, Zhenhua Zhao
OBJECTIVE: This study aimed to explore the value of preoperative and postoperative computed tomography (CT)-based radiomic signatures and Δ radiomic signatures for evaluating the early efficacy of microwave ablation (MWA) for pulmonary malignancies. METHODS: In total, 115 patients with pulmonary malignancies who underwent MWA treatment were categorized into response and nonresponse groups according to relevant guidelines and consensus. Quantitative image features of the largest pulmonary malignancies were extracted from CT noncontrast scan images preoperatively (time point 0, TP0) and immediately postoperatively (time point 1, TP1)...
April 24, 2024: Journal of Computer Assisted Tomography
#6
COMPARATIVE STUDY
A D Navarro-Vergara, A S Portillo-Candia, C L Sánchez-Silva, R A Arréllaga-Alonso, A A Portillo-Vanni
INTRODUCTION: health promotion policy requires the identification of barriers to the adoption of public policies. Paraguay's national healthcare system is inequitable, expensive, and inefficient. The Ministry of Public Health and Social Welfare (MSPyBS) is the entity responsible for covering the needs of a significant portion of the population. In January 2022, the MSPyBS financed the purchase of titanium elastic nails through a National Public Tender for Osteosynthesis Materials (LPN 02/22) to provide them for free in the pediatric service...
2024: Acta Ortopédica Mexicana
#7
JOURNAL ARTICLE
Saurabh Rohatgi, Amol Dua, Andrea Diociasi, Karen Buch, Arman Avesta, Jeremy Ford, Rajiv Gupta
OBJECTIVE: Radiological imaging is pivotal in diagnosing idiopathic normal pressure hydrocephalus (iNPH), given the similarity of its symptoms to other neurodegenerative diseases. We aimed to correlate the Evans index (EI), callosal angle (CA), and the volume of the lateral ventricles measured before cerebrospinal fluid removal with the resultant outcomes in gait response. METHODS: In our retrospective study, we identified 42 patients with a diagnosis of iNPH. These patients underwent gait analysis, imaging, and lumbar puncture...
April 24, 2024: Journal of Computer Assisted Tomography
#8
JOURNAL ARTICLE
Wen Bi, Jingjing Li, Mengqiu Xiong, Lubanga Nasifu, Mingjuan Tan, Ping Tai, Qing Jin, Lingyun Zhang, Chengbin Zhu, Bangshun He
Objective: The purpose of this study was to evaluate the association between the single nucleotide polymorphisms (SNPs) ( EGR3 rs1996147; EGR4 rs3813226, rs6747506; ERBB3 rs2292238; and ERBB4 rs707284, rs7560730) and the risk of schizophrenia (SZ) in a Chinese population. Materials and Methods: We conducted a case-control study, including 248 patients with SZ and 236 healthy controls matched for age and sex. The Mass-array platform was used to detect all the genotypes of the SNPs. Results: The results revealed that the EGR3 rs1996147 AA genotype was associated with borderline decreased SZ risk (AA vs...
April 2024: Genetic Testing and Molecular Biomarkers
#9
JOURNAL ARTICLE
Brittany P Marshall, Beth G Ashinsky, Xavier E Ferrer, Jennifer A Kunes, Astia C Innis, Andrew J Luzzi, Lynn Ann Forrester, Kevin G Burt, Andy J Lee, Lee Song, Lauren E Lisiewski, Rajesh K Soni, Clark T Hung, William N Levine, David Kovacevic, Stavros Thomopoulos
Rotator cuff injuries result in more than 500,000 surgeries annually in the United States, many of which fail. These surgeries typically involve repair of the injured tendon and removal of the subacromial bursa, a synovial-like tissue that sits between the rotator cuff and the acromion. The subacromial bursa has been implicated in rotator cuff pathogenesis and healing. Using proteomic profiling of bursa samples from nine patients with rotator cuff injury, we show that the bursa responds to injury in the underlying tendon...
April 24, 2024: Science Translational Medicine
#10
JOURNAL ARTICLE
Konstantinos Feidantsis, Nikolas Panteli, Thomas Bousdras, Francesco Gai, Laura Gasco, Efthimia Antonopoulou
In the context of evaluating the impact of environmentally friendly and sustainably produced alternative protein sources in fish feed, the present study's aim was to examine the overall physiological stress response in one of the main fish species of European freshwater aquaculture, Oncorhynchus mykiss (rainbow trout), following the partial substitution of fish meal (FM) with a Tenebrio molitor (TM) (yellow mealworm) full-fat meal. In total, 222 rainbow trout individuals (115.2 ± 14.2 g) were allocated randomly into six tanks, three per dietary treatment, and were fed a formulated diet containing 60% yellow mealworm (TM60) compared to a control diet without insect meal (TM0)...
April 24, 2024: Journal of Animal Physiology and Animal Nutrition
#11
JOURNAL ARTICLE
Tomoka Kadowaki, Ayako Sasaki, Naomi Matsumoto, Toshiharu Mitsuhashi, Hideharu Hagiya, Soshi Takao, Takashi Yorifuji
BACKGROUND: Understanding the association between the immune response and the risk of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection has implications for forthcoming prevention strategies. We evaluated the association between antibody titers and the risk of infection for the general population during the Omicron-dominant phase. METHODS: This was a prospective cohort study of residents or people affiliated with institutions in Bizen City, which included 1,899 participants...
April 24, 2024: Journal of Infectious Diseases
#12
JOURNAL ARTICLE
R Mezencev, M Feshuk, L Kolaczkowski, G C Peterson, Q J Zhao, S Watford, J A Weaver
Absolute (ALW) and relative (RLW) liver weight changes are sensitive endpoints in repeat-dose rodent toxicity studies, and their changes are often used for quantitative assessment of health effects induced by hepatotoxic chemicals using the benchmark dose-response modeling (BMD) approach. To find biologically relevant liver weight changes to chemical exposures, we evaluated all data available for liver weight changes and associated liver histopathologic findings from the Toxicity Reference Database (ToxRefDB)...
April 24, 2024: Toxicological Sciences: An Official Journal of the Society of Toxicology
#13
JOURNAL ARTICLE
Erin Walsh, Amanda J Krause, Madeline Greytak, Alexander M Kaizer, Philip A Weissbrod, Kelli Liu, Tiffany Taft, Rena Yadlapati
BACKGROUND: Laryngopharyngeal symptoms such as cough, throat clearing, voice change, paradoxic vocal fold movement or laryngospasm are hyper-responsive behaviors resulting from local irritation (e.g., refluxate) and heightened sympathetic tone. Laryngeal recalibration therapy (LRT) guided by a speech-language pathologist (SLP) provides mechanical desensitization and cognitive recalibration to suppress hyper-responsive laryngeal patterns. This study aimed to assess symptom response to LRT among patients with chronic laryngopharyngeal symptoms symptoms undergoing evaluation of gastroesophageal reflux disease (GERD)...
April 24, 2024: American Journal of Gastroenterology
#14
JOURNAL ARTICLE
Zhenyu Liu, Yang Wu, Haibo Zhang, Gang Li, Zhijie Ding, Bin Hu
Text content analysis for depression detection using machine learning techniques has become a prominent area of research. However, previous studies focused mainly on analyzing the textual content, neglecting the fundamental factors driving text generation. Consequently, existing models face the challenge of poor generalization to out-of-domain data as they struggle to capture the crucial features of depression. To address this, we propose a novel computational perspective of "stimulus-response patterns" that brings us closer to the essence of clinical diagnosis of depression...
April 24, 2024: IEEE Journal of Biomedical and Health Informatics
#15
JOURNAL ARTICLE
Maria Lönn, Petra Svedberg, Jens Nygren, Håkan Jarbin, Katarina Aili, Ingrid Larsson
STUDY OBJECTIVES: To examine differences in sample characteristics and longitudinal sleep outcomes according to weighted blanket adherence. METHODS: Children with attention-deficit/hyperactivity disorder (ADHD) (n =94), mean age 9.0 (sd 2.2, range 6-14) participated in a 16-week sleep intervention with weighted blankets (WB). Children were classified as WB adherent (use of WB ≥ 4 nights/week) or non-adherent (use of WB ≤ 3 nights/week). Changes in objectively measured sleep by actigraphy, parent-reported sleep problems (Children's Sleep Habits Questionnaire (CSHQ)) and child-reported Insomnia Severity Index (ISI) were evaluated according to adherence with mixed effect models...
April 24, 2024: Journal of Clinical Sleep Medicine: JCSM: Official Publication of the American Academy of Sleep Medicine
#16
JOURNAL ARTICLE
Birgith Engelst Grove, Annette de Thurah, Per Ivarsen, Ann Katrine Kvisgaard, Niels Henrik Hjollund, Regine Grytnes, Liv Marit Valen Schougaard
BACKGROUND: In Denmark, outpatient follow-up for patients with chronic kidney disease (CKD) is changing from in-hospital visits toward more remote health care delivery. The nonuse of remote patient-reported outcomes (PROs) is a well-known challenge, and it can be difficult to explain which mechanisms of interventions influence the outcome. Process evaluation may, therefore, be used to answer important questions on how and why interventions work, aiming to enhance the implications for clinical practice...
April 24, 2024: JMIR Formative Research
#17
JOURNAL ARTICLE
Yuxin Ni, Ying Lu, Fengshi Jing, Qianyun Wang, Yewei Xie, Xi He, Dan Wu, Rayner Kay Jin Tan, Joseph D Tucker, Xumeng Yan, Jason J Ong, Qingpeng Zhang, Hongbo Jiang, Wencan Dai, Liqun Huang, Wenhua Mei, Yi Zhou, Weiming Tang
BACKGROUND: Sexual health influencers (SHIs) are individuals actively sharing sexual health information with their peers, and they play an important role in promoting HIV care services, including the secondary distribution of HIV self-testing (SD-HIVST). Previous studies used a 6-item empirical leadership scale to identify SHIs. However, this approach may be biased as it does not consider individuals' social networks. OBJECTIVE: This study used a quasi-experimental study design to evaluate how well a newly developed machine learning (ML) model identifies SHIs in promoting SD-HIVST compared to SHIs identified by a scale whose validity had been tested before...
April 24, 2024: JMIR Public Health and Surveillance
#18
JOURNAL ARTICLE
Frederic Berner, Michael Kovermann
The analysis of pressure induced changes in the chemical shift of proteins allows statements on structural fluctuations proteins exhibit at ambient pressure. The inherent issue of separating general pressure effects from structural related effects on the pressure dependence of chemical shifts has so far been addressed by considering the characteristics of random coil peptides on increasing pressure. In this work, chemically and pressure denatured states of the cold shock protein B from Bacillus subtilis (BsCspB) have been assigned in 2D 1H-15N HSQC NMR spectra and their dependence on increasing hydrostatic pressure has been evaluated...
April 24, 2024: Angewandte Chemie
#19
JOURNAL ARTICLE
Cynthia Gyamfi-Bannerman, Rebecca G Clifton, Alan T N Tita, Sean C Blackwell, Monica Longo, Jessica A de Voest, T Michael O'Shea, Sabine Z Bousleiman, Felecia Ortiz, Dwight J Rouse, Torri D Metz, George R Saade, Kara M Rood, Kent D Heyborne, John M Thorp, Geeta K Swamy, William A Grobman, Kelly S Gibson, Yasser Y El-Sayed, George A Macones
IMPORTANCE: The Antenatal Late Preterm Steroids (ALPS) trial changed clinical practice in the United States by finding that antenatal betamethasone at 34 to 36 weeks decreased short-term neonatal respiratory morbidity. However, the trial also found increased risk of neonatal hypoglycemia after betamethasone. This follow-up study focused on long-term neurodevelopmental outcomes after late preterm steroids. OBJECTIVE: To evaluate whether administration of late preterm (34-36 completed weeks) corticosteroids affected childhood neurodevelopmental outcomes...
April 24, 2024: JAMA
#20
JOURNAL ARTICLE
1,25(OH) 2 D 3 inhibits pancreatic stellate cells activation and promotes insulin secretion in T2DM.
Zhengyu Zhou, Lewen Zhang, Xun Wei, Aiqing Wang, Yudie Hu, Min Xiao, Yuxuan Zheng
PURPOSE: To evaluate the effect and mechanism of 1,25(OH)2 D3 on pancreatic stellate cells (PSCs) in type 2 diabetes mellitus (T2DM). METHODS: A mouse model of T2DM was successfully established by high-fat diet (HFD) /streptozotocin (STZ) and administered 1,25(OH)2 D3 for 3 weeks. Fasting blood glucose (FBG), glycated hemoglobin A1c (GHbA1c), insulin (INS) and glucose tolerance were measured. Histopathology changes and fibrosis of pancreas were examined by hematoxylin and eosin staining and Masson staining...
April 24, 2024: Endocrine
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