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https://www.readbyqxmd.com/read/28319446/good-laboratory-practice-preclinical-safety-studies-for-gsk2696273-mlv-vector-based-ex-vivo-gene-therapy-for-adenosine-deaminase-deficiency-severe-combined-immunodeficiency-in-nsg-mice
#1
Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez, Michela Vezzoli, Franck Chanut, Rhiannon Lowe, Draghici Elena, Melanie Nord, Paola Albertini, Patrizia Cristofori, Jane Richards, Hazel Staton, Jonathan Appleby, Alessandro Aiuti, Aisha V Sauer
GSK2696273 (autologous CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] enzyme) is a gamma-retroviral ex vivo gene therapy of bone marrow-derived CD34+ cells for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID). ADA-SCID is a severe monogenic disease characterized by immunologic and nonimmunologic symptoms. Bone-marrow transplant from a matched related donor is the treatment of choice, but it is available for only a small proportion of patients...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28315235/identification-and-quantitation-of-malonic-acid-biomarkers-of-in-born-error-metabolism-by-targeted-metabolomics
#2
Chandra Shekar R Ambati, Furong Yuan, Lutfi A Abu-Elheiga, Yiqing Zhang, Vivekananda Shetty
Malonic acid (MA), methylmalonic acid (MMA), and ethylmalonic acid (EMA) metabolites are implicated in various non-cancer disorders that are associated with inborn-error metabolism. In this study, we have slightly modified the published 3-nitrophenylhydrazine (3NPH) derivatization method and applied it to derivatize MA, MMA, and EMA to their hydrazone derivatives, which were amenable for liquid chromatography- mass spectrometry (LC-MS) quantitation. 3NPH was used to derivatize MA, MMA, and EMA, and multiple reaction monitoring (MRM) transitions of the corresponding derivatives were determined by product-ion experiments...
March 17, 2017: Journal of the American Society for Mass Spectrometry
https://www.readbyqxmd.com/read/28303569/efficacy-and-safety-of-mifepristone-buccal-misoprostol-for-early-medical-abortion-in-an-australian-clinical-setting
#3
Philip Goldstone, Clara Walker, Katherine Hawtin
BACKGROUND: In 2014, a composite pack containing mifepristone-buccal misoprostol, indicated for use to 63 days gestation replaced the existing regimen for early medical abortion (EMA) in Australia. AIMS: To provide updated efficacy and safety information for the use of mifepristone-buccal misoprostol for EMA in Australia, and assess the effect of patient age and gestational age on efficacy. MATERIALS AND METHODS: Observational cohort study of 15 008 women attending one of 16 Marie Stopes International clinics in Australia for an EMA (gestational age ≤ 63 days) between 1 March 2013 and 30 September 2015...
March 17, 2017: Australian & New Zealand Journal of Obstetrics & Gynaecology
https://www.readbyqxmd.com/read/28298264/ecological-momentary-assessment-in-behavioral-research-addressing-technological-and-human-participant-challenges
#4
Lora E Burke, Saul Shiffman, Edvin Music, Mindi A Styn, Andrea Kriska, Asim Smailagic, Daniel Siewiorek, Linda J Ewing, Eileen Chasens, Brian French, Juliet Mancino, Dara Mendez, Patrick Strollo, Stephen L Rathbun
BACKGROUND: Ecological momentary assessment (EMA) assesses individuals' current experiences, behaviors, and moods as they occur in real time and in their natural environment. EMA studies, particularly those of longer duration, are complex and require an infrastructure to support the data flow and monitoring of EMA completion. OBJECTIVE: Our objective is to provide a practical guide to developing and implementing an EMA study, with a focus on the methods and logistics of conducting such a study...
March 15, 2017: Journal of Medical Internet Research
https://www.readbyqxmd.com/read/28287857/assessment-of-immunomodulatory-activities-and-in-vitro-toxicity-of-new-quinolone-7-ethyl-9-ethyl-6-oxo-6-9-dihydro-1-2-5-selenadiazolo-3-4-h-quinoline-7-carboxylate
#5
Soňa Jantová, Ema Paulovičová, Lucia Paulovičová, Dominika Topoľská, Miroslav Pánik, Viktor Milata
Our previous studies on leukemia cells L1210 and cervical cancer HeLa cells revealed cytotoxic effects of the 7-ethyl 9-ethyl-6-oxo-6,9-dihydro[1,2,5]selenadiazolo[3,4-h]quinoline-7-carboxylate (E2h), a new synthetically prepared quinolone derivative, toward selected cancer cell lines. The aim of the present study was to examine the cytotoxicity of E2h toward next cell lines and tissues; that is, human cancer HL-60 and A549 cells, human non-cancer fibroblast BHNF-1 cells, and reconstructed human epidermis tissues...
March 13, 2017: Immunological Investigations
https://www.readbyqxmd.com/read/28287367/the-effects-of-contingency-contracts-and-performance-feedback-on-completing-data-entries-to-self-monitor-community-participation-of-people-with-physical-disabilities-an-ecological-momentary-study
#6
Chiaki Gonda-Kotani, Glen W White
This article examines the use of ecological momentary assessment (EMA) to analyze the effect of contingent contracts and performance feedback in completing data entries to self-monitor community participation of people with mobility-related disabilities. Two participants with mobility-related disabilities were assigned to self-monitor their community participation activities using a personal digital assistant (PDA), which had been preprogrammed to signal four times a day throughout the entire study. At each prompt, participants were asked to complete a brief survey on a PDA...
April 2017: Journal of Prevention & Intervention in the Community
https://www.readbyqxmd.com/read/28286687/lipomatous-extensively-vacuolated-ependymoma-with-signet-ring-cell-like-appearance-analysis-of-a-case-with-extensive-literature-review
#7
Miguel Fdo Salazar, Martha Lilia Tena-Suck, Alma Ortiz-Plata, Citlaltepetl Salinas-Lara, Daniel Rembao-Bojórquez
"Lipomatous" and "extensively vacuolated" are descriptive captions that have been used to portray a curious subset of ependymomas distinctively bearing cells with a large vacuole pushing the nucleus to the periphery and, thus, simulating a signet-ring cell appearance. Here, we would like to report the first ependymoma of this kind in a Latin American institution. A 16-year-old boy experienced cephalea during three months. Magnetic resonance imaging scans showed a left paraventricular tumour which corresponded to anaplastic ependymoma...
2017: Case Reports in Pathology
https://www.readbyqxmd.com/read/28286144/characterization-of-theileria-equi-genotypes-in-horses-in-israel-the-palestinian-authority-and-jordan
#8
Dafna Ketter-Ratzon, Sharon Tirosh-Levy, Yaarit Nachum-Biala, Tal Saar, Lara Qura'n, Doni Zivotofsky, Ziad Abdeen, Gad Baneth, Amir Steinman
Equine theileriosis caused by Theileria equi is endemic in the Middle East, where it causes a severe disease as well as widespread subclinical infection. The aim of this study was to evaluate the diversity of T. equi genotypes in Israel and the neighboring Palestinian Authority and Jordan. Blood samples from 355 horses from Israel, the Palestinian Authority and Jordan were tested for the prevalence of T. equi DNA. Two hundred and fourteen (60%) were found positive for T. equi infection by PCR. Of those, the 18S rRNA (1458bp) and the EMA-1 (745bp) genes of T...
March 4, 2017: Ticks and Tick-borne Diseases
https://www.readbyqxmd.com/read/28285812/-a-case-report-of-chordoid-glioma-with-unusual-features-immunohistochemical-and-molecular-findings-and-differential-diagnoses
#9
Arnault Tauziède-Espariat, Gilles Robert, Patricia de Cremoux, Marc Polivka
We report the case of a 63-year-old healthy patient who was admitted for surgery of a suprasellar tumor with extension to the optic chiasm responsible of visual disturbance. Histopathological examination revealed a tumoral proliferation composed of epithelioid cells without atypia arranged in cords in a mucinous matrix surrounded by some lymphocytic inflammatory infiltrates. On immunohistochemistry, the neoplastic cells strongly expressed GFAP and CD34, a weak expression of EMA, an expression of TTF1 without immunoreactivity for brachyury...
March 9, 2017: Annales de Pathologie
https://www.readbyqxmd.com/read/28281318/intracranial-myxoid-mesenchymal-tumors-with-ewsr1-creb-family-gene-fusions-myxoid-variant-of-angiomatoid-fibrous-histiocytoma-or-novel-entity
#10
Tejus A Bale, Angelica Oviedo, Harry Kozakewich, Caterina Giannini, Phani K Davineni, Keith Ligon, Sanda Alexandrescu
BACKGROUND: Intracranial myxoid mesenchymal tumor harboring EWSR1 fusions with CREB family of genes was recently described, and it resembles the myxoid variant of angiomatoid fibrous histiocytoma. We present 3 pediatric patients with intracranial EWSR1-rearranged myxoid mesenchymal neoplasm and provide a molecular genetic characterization of these tumors. METHODS: Clinical histories and imaging results were reviewed. Histology, immunohistochemistry, EWSR1, FUS, NR4A3 fluorescence in situ hybridization (FISH), and next generation sequencing (NGS) were performed...
March 9, 2017: Brain Pathology
https://www.readbyqxmd.com/read/28281136/ecological-momentary-assessment-of-dietary-lapses-across-behavioral-weight-loss-treatment-characteristics-predictors-and-relationships-with-weight-change
#11
Evan M Forman, Leah M Schumacher, Ross Crosby, Stephanie M Manasse, Stephanie P Goldstein, Meghan L Butryn, Emily P Wyckoff, J Graham Thomas
BACKGROUND: Adherence to dietary prescriptions is critical for successful weight loss and weight loss maintenance. However, research on specific instances of inadherence (lapses) is limited, and findings regarding the frequency, nature, and causes of lapses are mixed. Additionally, no studies have examined lapses over the course of a weight loss program. PURPOSE: In the context of a reduced calorie diet prescribed as part of a behavioral treatment, we aimed to characterize lapse occurrence, examine lapse frequency across treatment, examine predictors of lapses, and assess the relationship between lapses and weight loss...
March 9, 2017: Annals of Behavioral Medicine: a Publication of the Society of Behavioral Medicine
https://www.readbyqxmd.com/read/28280640/hhv8-ebv-coinfection-lymphoproliferative-disorder-rare-entity-with-a-favorable-outcome
#12
Dhouha Bacha, Beya Chelly, Houda Kilani, Lamia Charfi, Amel Douggaz, Samia Chatti, Emna Chelbi
HHV8/EBV-associated germinotropic lymphoproliferative disorder (GLD) is a challenging diagnosis given its rarity, the particular clinical presentation, and the lack of expression of markers usually used in establishing hematopoietic lineage. We report a new case of HHV8/EBV GLD in an immunocompetent 78-year-old woman. The diagnosis was made in an incidentally discovered lymphadenopathy. Histological examination showed a nodular lymphoid proliferation centered by aggregates of atypical plasmablastic cells admixed with small lymphoid cells...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/28279830/effects-of-partially-dismantling-the-cd4-binding-site-glycan-fence-of-hiv-1-envelope-glycoprotein-trimers-on-neutralizing-antibody-induction
#13
Ema T Crooks, Keiko Osawa, Tommy Tong, Samantha L Grimley, Yang D Dai, Robert G Whalen, Daniel W Kulp, Sergey Menis, William R Schief, James M Binley
Previously, VLPs bearing JR-FL strain HIV-1 Envelope trimers elicited potent neutralizing antibodies (nAbs) in 2/8 rabbits (PLoS Pathog 11(5): e1004932) by taking advantage of a naturally absent glycan at position 197 that borders the CD4 binding site (CD4bs). In new immunizations, we attempted to improve nAb responses by removing the N362 glycan that also lines the CD4bs. All 4 rabbits developed nAbs. One targeted the N197 glycan hole like our previous sera. Two sera depended on the N463 glycan, again suggesting CD4bs overlap...
March 6, 2017: Virology
https://www.readbyqxmd.com/read/28277869/disease-burden-of-systemic-light-chain-al-amyloidosis-a-systematic-literature-review
#14
Huamao Mark Lin, Xin Gao, Catherine E Cooke, Deborah Berg, Richard Labotka, Douglas V Faller, Brian Seal, Parameswaran Hari
A systematic literature review on AL amyloidosis was conducted in order to understand the disease burden, and identify unmet medical needs and knowledge gaps. MEDLINE, EMBASE and COCHRANE databases were searched for English language studies published in the last 10 years using search terms that focused on the clinical, economic, and patient-reported outcome (PRO) aspects of AL amyloidosis. There was a low yield of articles in the economic and PRO categories and additional searches were conducted in clinical conference proceedings, and using Google and Google Scholar...
February 21, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28277707/family-assessment-device-real-world-validity-in-urban-families-of-children-with-asthma
#15
Gillian G Leibach, Robin S Everhart
Ecological momentary assessment (EMA) was used to further validate the real-world predictability of the Family Assessment Device (FAD) among low income, racial and ethnic minority, urban families of children (7-12 years) with asthma. Caregivers completed self-report measures at baseline, as well as daily assessments of family functioning for 2 weeks through EMA delivered via smartphone. Concurrent validity was established with measures of caregiver perceived stress and positive and negative affect at baseline...
March 9, 2017: Journal of Family Psychology: JFP
https://www.readbyqxmd.com/read/28276811/new-therapeutic-options-for-ibs-the-role-of-the-first-in-class-mixed-%C3%A2%C2%B5-opioid-receptor-agonist-and-%C3%AE-opioid-receptor-antagonist-mudelta-eluxadoline
#16
Maura Corsetti, Peter Whorwell
Irritable bowel syndrome (IBS) is a prevalent functional gastrointestinal disorder which represents a major cost to healthcare services. IBS-D patients represent about one-third of the IBS population and are currently treated with antispasmodics, loperamide, bile acid sequestrants and antidepressants. Alosetron and rifaximin are also available in USA, ramosetron in Japan, Korea and Thailand and ondansetron as an off-label treatment. Areas covered: This article focuses on eluxadoline, a novel pharmacological agent that has recently been approved by both the FDA and EMA for treatment of patients with IBS-D...
April 2017: Expert Review of Gastroenterology & Hepatology
https://www.readbyqxmd.com/read/28276604/one-step-immunochromatographic-visual-assay-for-anti-transglutaminase-detection-in-organ-culture-system-an-easy-and-prompt-method-to-simplify-the-in-vitro-diagnosis-of-celiac-disease
#17
Marco Di Tola, Mariacatia Marino, Rossella Casale, Raffaele Borghini, Antonio Tiberti, Giuseppe Donato, Umberto Occhiuzzi, Antonio Picarelli
BACKGROUND: Anti-tissue transglutaminase (anti-tTG) and endomysium antibodies (EMA) are detectable in duodenal culture media of celiac disease (CD) patients. To improve the management of this organ culture system, we evaluated the anti-tTG occurrence by immunochromatographic assay (ICA). METHODS: A total of 103 CD patients and 41 disease controls underwent duodenal biopsy for the organ culture. In culture supernatants, IgA anti-tTG were tested by both enzyme-linked immunosorbent assay (ELISA) and ICA, IgA EMA were searched by indirect immunofluorescence analysis (iIFA)...
March 9, 2017: Journal of Clinical Laboratory Analysis
https://www.readbyqxmd.com/read/28276176/user-profiles-of-an-electronic-mental-health-tool-for-ecological-momentary-assessment-memind
#18
María Luisa Barrigón, Sofian Berrouiguet, Juan José Carballo, Covadonga Bonal-Giménez, Pablo Fernández-Navarro, Bernadette Pfang, David Delgado-Gómez, Philippe Courtet, Fuensanta Aroca, Jorge Lopez-Castroman, Antonio Artés-Rodríguez, Enrique Baca-García
Ecological momentary assessment (EMA) is gaining importance in psychiatry. This article assesses the characteristics of patients who used a new electronic EMA tool: the MEmind Wellness Tracker. Over one year, 13811 adult outpatients in our Psychiatry Department were asked to use MEmind. We collected information about socio-demographic data, psychiatric diagnoses, illness severity, stressful life events and suicidal thoughts/behavior. We compared active users (N = 2838) and non-active users (N = 10,973) of MEmind and performed a Random Forest analysis to assess which variables could predict its use...
March 9, 2017: International Journal of Methods in Psychiatric Research
https://www.readbyqxmd.com/read/28267446/characterisation-of-the-pore-structure-of-functionalised-calcium-carbonate-tablets-by-terahertz-time-domain-spectroscopy-and-x-ray-computed-microtomography
#19
Daniel Markl, Parry Wang, Cathy Ridgway, Anssi-Pekka Karttunen, Mousumi Chakraborty, Prince Bawuah, Pertti Pääkkönen, Patrick Gane, Jarkko Ketolainen, Kai Peiponen, J Axel Zeitler
Novel excipients are entering the market to enhance the bioavailability of drug particles by having a high porosity and thus providing a rapid liquid uptake and disintegration to accelerate subsequent drug dissolution. One example of such a novel excipient is functionalised calcium carbonate (FCC), which enables the manufacture of compacts with a bimodal pore size distribution consisting of larger inter-particle and fine intra-particle pores. Five sets of FCC tablets with a target porosity of 45% to 65% were prepared in 5% steps and characterised using terahertz time-domain spectroscopy (THz-TDS) and X- ray computed microtomography (XμCT)...
March 3, 2017: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28266870/evaluation-of-different-low-dose-multidetector-ct-and-cone-beam-ct-protocols-in-maxillary-sinus-imaging-part-i-an-in-vitro-study
#20
Abeer A Almashraqi, Eman A Ahmed, Nashwa S Mohamed, Imad H Barngkgei, Nader A Elsherbini, Esam S Halboub
OBJECTIVES: This in-vitro study aimed at assessing radiation dose and image quality of different low-dose MDCT and CBCT imaging protocols in comparison to standard MDCT protocol for maxillary sinus imaging. METHODS: Effective dose (E) and image quality of 10 MDCT (changing effective milliampere (EmAs) starting from 141.3 to 20) and 3 CBCT protocols (changing mAs and voxel size (VS)) were assessed throughout scanning an anthropomorphic head and neck Alderson Rando phantom...
March 7, 2017: Dento Maxillo Facial Radiology
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