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https://www.readbyqxmd.com/read/28530135/prolonged-expression-of-secreted-enzymes-in-dogs-after-liver-directed-delivery-of-i-sleeping-beauty-i-transposons-implications-for-non-viral-gene-therapy-of-systemic-disease
#1
Elena L Aronovich, Kendra Anne Hyland, Bryan C Hall, Jason B Bell, Erik R Olson, Myra Urness Rusten, David W Hunter, N Matthew Ellinwood, R Scott McIvor, Perry B Hackett
The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice including hemophilias A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis (MPS) types I and VII caused by α-L-iduronidase (IDUA) and β-glucuronidase (GUSB) deficiency, respectively. We recently reported modified approaches of the hydrodynamics-based procedure to deliver transposons to the liver in dogs. Using the transgenic canine reporter secreted alkaline phosphatase (cSEAP), we demonstrated transgenic protein in the plasma for up to six weeks post-infusion...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28528289/platelet-hyperactivation-apoptosis-and-hypercoagulability-in-patients-with-acute-pulmonary-embolism
#2
Samin Rezania, Michael A Puskarich, Daniela N Petrusca, Evandro M Neto-Neves, Matthew T Rondina, Jeffrey A Kline
Changes in systemic redox balance can alter platelet activation and aggregation. Acute pulmonary embolism (PE) is a systematic inflammatory disease associated with mechanical shear stress, increased thrombin, catecholamines, serotonin and hemolysis, which cumulatively can hyperactivate platelets and accelerate their turnover. We tested the hypothesis that platelets from patients with moderately severe PE will show hyperstimulation and a pre-apoptotic phenotype associated with microparticles (MPs) in plasma...
May 13, 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28520220/endothelial-damage-effects-of-circulating-microparticles-from-patients-with-stable-angina-is-reduced-by-aspirin-though-erk-p38-mapks-pathways
#3
Cheng Gong, Xue-Feng Shan, Xu-Lang Wang, Wei-Wei Dong, Zhe Li, D Xin-Hong Liu, Wei Zhang, Kun Xing, Feng-Jun Chang
INTRODUCTION: Platelet activation participate in the development of both coronary artery disease (CAD) and circulating microparticles (MPs). As a commonly used medicine for coronary heart disease, whether aspirin affects the function of MPs remains unclear. AIMS: This study was designed to test MPs from healthy subjects, stable angina (SA) patients before and after aspirin administration were obtained. MPs origins were tested by flow cytometry. Rat thoracic aortas were incubated with MPs (with or without aspirin) to determine the effects of MPs on expression of ERK1/2, JNKs and p38 MAPK...
May 18, 2017: Cardiovascular Therapeutics
https://www.readbyqxmd.com/read/28513999/microparticles-of-healthy-origins-improve-endothelial-progenitor-cell-dysfunction-via-microrna-transfer-in-an-atherosclerotic-hamster-model
#4
Nicoleta Alexandru, Eugen Andrei, Loredan Niculescu, Emanuel Dragan, Violeta Ristoiu, Adriana Georgescu
AIM: In this study we aimed: (1) to obtain and functionally characterize the cultures of late endothelial progenitor cells (EPCs) from the animal blood; (2) to investigate the potential beneficial effects of circulating microparticles (MPs) of healthy origins on EPC dysfunctionality in atherosclerosis as well as involved mechanisms. METHODS: Late EPCs were obtained and expanded in culture from peripheral blood isolated from two animal groups: hypertensive-hyperlipidemic (HH) and control (C) hamsters...
May 17, 2017: Acta Physiologica
https://www.readbyqxmd.com/read/28511172/donor-specific-anti-human-leukocyte-antigens-antibodies-acute-rejection-renal-function-and-histology-in-kidney-transplant-recipients-receiving-tacrolimus-and-everolimus
#5
Alexandra Ferreira, Claudia Felipe, Marina Cristelli, Laila Viana, Geovana Basso, Suelen Stopa, Juliana Mansur, Mayara Ivani, Adrieli Bessa, Priscila Ruppel, Wilson Aguiar, Erika Campos, Maria Gerbase-DeLima, Henrique Proença, Helio Tedesco-Silva, José Medina-Pestana
BACKGROUND: This analysis compared efficacy, renal function, and histology in kidney transplant recipients receiving tacrolimus (TAC) combined with everolimus (EVR) or mycophenolate (MPS). METHODS: This was a retrospective analysis from a randomized trial in kidney transplant recipients who received a single 3 mg/kg dose of rabbit antithymocyte globulin (r-ATG), TAC, EVR, and prednisone (PRED; r-ATG/EVR, n = 85), basiliximab (BAS), TAC, EVR, and PRED (BAS/EVR, n = 102) or BAS, TAC, MPS, and PRED (BAS/MPS, n = 101)...
May 17, 2017: American Journal of Nephrology
https://www.readbyqxmd.com/read/28501941/paracrine-release-of-il-2-and-anti-ctla-4-enhances-the-ability-of-artificial-polymer-antigen-presenting-cells-to-expand-antigen-specific-t-cells-and-inhibit-tumor-growth-in-a-mouse-model
#6
Lei Zhang, Limin Wang, Khawar Ali Shahzad, Tao Xu, Xin Wan, Weiya Pei, Chuanlai Shen
Accumulating evidence indicates that bead-based artificial antigen-presenting cells (aAPCs) are a powerful tool to induce antigen-specific T cell responses in vitro and in vivo. To date, most conventional aAPCs have been generated by coupling an antigen signal (signal 1) and one or two costimulatory signals, such as anti-CD28 with anti-LFA1 or anti-4-1BB (signal 2), onto the surfaces of cell-sized or nanoscale magnetic beads or polyester latex beads. The development of a biodegradable scaffold and the combined use of multiple costimulatory signals as well as third signals for putative clinical applications is the next step in the development of this technology...
May 13, 2017: Cancer Immunology, Immunotherapy: CII
https://www.readbyqxmd.com/read/28497321/predicting-cumulative-and-maximum-brain-strain-measures-from-hybridiii-head-kinematics-a-combined-laboratory-study-and-post-hoc-regression-analysis
#7
Brooklynn M Knowles, Christopher R Dennison
Due to growing concern on brain injury in sport, and the role that helmets could play in preventing brain injury caused by impact, biomechanics researchers and helmet certification organizations are discussing how helmet assessment methods might change to assess helmets based on impact parameters relevant to brain injury. To understand the relationship between kinematic measures and brain strain, we completed hundreds of impacts using a 50th percentile Hybrid III head-neck wearing an ice hockey helmet and input three-dimensional impact kinematics to a finite element brain model called the Simulated Injury Monitor (SIMon) (n = 267)...
May 11, 2017: Annals of Biomedical Engineering
https://www.readbyqxmd.com/read/28495847/inflammatory-responses-to-acute-elevations-of-carbon-dioxide-in-mice
#8
Stephen R Thom, Veena M Bhopale, Jingping Hu, Ming Yang
Health risks are described from elevated indoor air carbon dioxide (CO2), which often ranges from 1000 to 4000 parts per million (ppm), but mechanisms are unknown. Here, we demonstrate that mice exposed for 2 hours to 2000 or 4000 ppm CO2 exhibit, respectively, 3.4 + 0.9 (SE, n=6) and 4.1 + 0.7 (n=10)-fold elevations in circulating microparticles (MPs); neutrophil and platelet activation and vascular leak in brain, muscle and distal colon. Interleukin (IL)-1β content of MPs also increases after 2000 ppm by 3...
May 11, 2017: Journal of Applied Physiology
https://www.readbyqxmd.com/read/28494282/anthropogenic-fibres-in-the-baltic-sea-water-column-field-data-laboratory-and-numerical-testing-of-their-motion
#9
A Bagaev, A Mizyuk, L Khatmullina, I Isachenko, I Chubarenko
Distribution of microplastics particles (MPs) in the water column is investigated on the base of 95 water samples collected from various depths in the Baltic Sea Proper in 2015-2016. Fibres are the prevalent type of MPs: 7% of the samples contained small films; about 40% had (presumably) paint flakes, while 63% contained coloured fibres in concentrations from 0.07 to 2.6 items per litre. Near-surface and near-bottom layers (defined as one tenth of the local depth) have 3-5 times larger fibre concentrations than intermediate layers...
May 7, 2017: Science of the Total Environment
https://www.readbyqxmd.com/read/28489793/a-selective-screening-program-for-the-early-detection-of-mucopolysaccharidosis-results-of-the-find-project-a-2-year-follow-up-study
#10
Cristóbal Colón, J Victor Alvarez, Cristina Castaño, Luís G Gutierrez-Solana, Ana M Marquez, María O'Callaghan, Félix Sánchez-Valverde, Carmen Yeste, María-Luz Couce
The mucopolysaccharidoses (MPSs) are underdiagnosed but they are evaluated in few newborn screening programs, probably due to the many challenges remaining, such as the identification of late-onset phenotypes. Systematic screening at the onset of clinical symptoms could help to early identify patients who may benefit from specific treatments. The aim of this prospective study was to assess a novel selective screening program, the FIND project, targeting patients aged 0 to 16 years with clinical manifestations of MPS...
May 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28485265/massively-parallel-sequencing-mps-of-cell-free-fetal-dna-cffdna-for-trisomies-21-18-and-13-in-twin-pregnancies
#11
Erqiu Du, Chun Feng, Yuming Cao, Yanru Yao, Jing Lu, Yuanzhen Zhang
Massively parallel sequencing (MPS) technology has become increasingly available and has been widely used to screen for trisomies 21, 18, and 13 in singleton pregnancies. This study assessed the performance of MPS testing of cell-free fetal DNA (cffDNA) from maternal plasma for trisomies 21, 18, and 13 in twin pregnancies. Ninety-two women with twin pregnancies were recruited. The results were identified through karyotypes of amniocentesis or clinical examination and follow-up of the neonates. Fluorescent in-situ hybridization was used to examine the placentas postnatally in cases of false-positive results...
May 9, 2017: Twin Research and Human Genetics: the Official Journal of the International Society for Twin Studies
https://www.readbyqxmd.com/read/28478695/prevention-of-neurocognitive-deficiency-in-mucopolysaccharidosis-type-ii-mice-by-cns-directed-aav9-mediated-iduronate-sulfatase-gene-transfer
#12
Kanut Laoharawee, Kelly M Podetz-Pedersen, Tam T Nguyen, Laura B Evenstar, Kelley F Kitto, Zhenhong Nan, Carolyn A Fairbanks, Walter C Low, Karen F Kozarsky, R Scott McIvor
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare X-linked recessive lysosomal disorder caused by defective Iduronate-2-sulfatase (IDS) resulting in accumulation of heparan sulfate and dermatan sulfate glycosaminoglycans (GAGs). Enzyme replacement is the only FDA-approved therapy available for MPS II, but it is expensive and does not improve neurologic outcomes in MPS II patients. We conducted this study to evaluate the effectiveness of adeno-associated virus (AAV) vector encoding human IDS delivered intracerebroventricularly in a murine model of MPS II...
May 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28476725/mobile-phone-surveys-for-collecting-population-level-estimates-in-low-and-middle-income-countries-a-literature-review
#13
Dustin G Gibson, Amanda Pereira, Brooke A Farrenkopf, Alain B Labrique, George W Pariyo, Adnan A Hyder
BACKGROUND: National and subnational level surveys are important for monitoring disease burden, prioritizing resource allocation, and evaluating public health policies. As mobile phone access and ownership become more common globally, mobile phone surveys (MPSs) offer an opportunity to supplement traditional public health household surveys. OBJECTIVE: The objective of this study was to systematically review the current landscape of MPSs to collect population-level estimates in low- and middle-income countries (LMICs)...
May 5, 2017: Journal of Medical Internet Research
https://www.readbyqxmd.com/read/28476724/the-development-of-an-interactive-voice-response-survey-for-noncommunicable-disease-risk-factor-estimation-technical-assessment-and-cognitive-testing
#14
Dustin G Gibson, Brooke A Farrenkopf, Amanda Pereira, Alain B Labrique, George William Pariyo
BACKGROUND: The rise in mobile phone ownership in low- and middle-income countries (LMICs) presents an opportunity to transform existing data collection and surveillance methods. Administering surveys via interactive voice response (IVR) technology-a mobile phone survey (MPS) method-has potential to expand the current surveillance coverage and data collection, but formative work to contextualize the survey for LMIC deployment is needed. OBJECTIVE: The primary objectives of this study were to (1) cognitively test and identify challenging questions in a noncommunicable disease (NCD) risk factor questionnaire administered via an IVR platform and (2) assess the usability of the IVR platform...
May 5, 2017: Journal of Medical Internet Research
https://www.readbyqxmd.com/read/28476720/moving-the-agenda-on-noncommunicable-diseases-policy-implications-of-mobile-phone-surveys-in-low-and-middle-income-countries
#15
George W Pariyo, Adaeze C Wosu, Dustin G Gibson, Alain B Labrique, Joseph Ali, Adnan A Hyder
The growing burden of noncommunicable diseases (NCDs), for example, cardiovascular diseases and chronic respiratory diseases, in low- and middle-income countries (LMICs) presents special challenges for policy makers, due to resource constraints and lack of timely data for decision-making. Concurrently, the increasing ubiquity of mobile phones in LMICs presents possibilities for rapid collection of population-based data to inform the policy process. The objective of this paper is to highlight potential benefits of mobile phone surveys (MPS) for developing, implementing, and evaluating NCD prevention and control policies...
May 5, 2017: Journal of Medical Internet Research
https://www.readbyqxmd.com/read/28471177/topical-formulation-of-self-assembled-antiviral-prodrug-nanomicelles-for-targeted-retinal-delivery
#16
Abhirup Mandal, Kishore Cholkar, Varun Khurana, Ankit Shah, Vibhuti Agrahari, Rohit Bisht, Dhananjay Pal, Ashim K Mitra
Topical drug administration for back of the eye delivery is extremely challenging due to the presence of protection mechanisms and physiological barriers. Self-assembled polymeric nanomicelles have emerged as promising vehicles for drug delivery. Apart from serving as an inert nanocarrier for therapeutic agents, polymeric nanomicelles are known to bypass mononuclear phagocytic system (MPS) and efflux transporters thereby improving drug bioavailability. In this investigation, a highly efficacious biotinylated lipid prodrug of cyclic cidofovir (B-C12-cCDF) was formulated within polymeric nanomicelles as a carrier for targeted retinal delivery...
May 17, 2017: Molecular Pharmaceutics
https://www.readbyqxmd.com/read/28467847/spin-projected-matrix-product-states-versatile-tool-for-strongly-correlated-systems
#17
Zhendong Li, Garnet Kin-Lic Chan
We present a new wave function ansatz that combines the strengths of spin projection with the language of matrix product states (MPS) and matrix product operators (MPO) as used in the density matrix renormalization group (DMRG). Specifically, spin-projected matrix product states (SP-MPS) are constructed as [Formula: see text], where [Formula: see text] is the spin projector for total spin S and |ΨMPS((N,M))⟩ is an MPS wave function with a given particle number N and spin projection M. This new ansatz possesses several attractive features: (1) It provides a much simpler route to achieve spin adaptation (i...
May 16, 2017: Journal of Chemical Theory and Computation
https://www.readbyqxmd.com/read/28464912/ten-years-of-the-hunter-outcome-survey-hos-insights-achievements-and-lessons-learned-from-a-global-patient-registry
#18
REVIEW
Joseph Muenzer, Simon A Jones, Anna Tylki-Szymańska, Paul Harmatz, Nancy J Mendelsohn, Nathalie Guffon, Roberto Giugliani, Barbara K Burton, Maurizio Scarpa, Michael Beck, Yvonne Jangelind, Elizabeth Hernberg-Stahl, Maria Paabøl Larsen, Tom Pulles, David A H Whiteman
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease-specific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II...
May 2, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28460238/microplastics-pollution-after-the-removal-of-the-costa-concordia-wreck-first-evidences-from-a-biomonitoring-case-study
#19
Carlo Giacomo Avio, Lara Roberta Cardelli, Stefania Gorbi, David Pellegrini, Francesco Regoli
Microplastics (MPs) represent a matter of growing concern for the marine environment. Their ingestion has been documented in several species worldwide, but the impact of specific anthropogenic activities remains largely unexplored. In this study, MPs were characterized in different benthic fish sampled after 2.5 years of huge engineering operations for the parbuckling project on the Costa Concordia wreck at Giglio Island. Fish collected in proximity of the wreck showed a high ingestion of microplastics compared to both fish from a control area and values reported worldwide...
April 28, 2017: Environmental Pollution
https://www.readbyqxmd.com/read/28455159/metal-versus-plastic-stents-for-anastomotic-biliary-strictures-after-liver-transplantation-a-randomized-controlled-trial
#20
Fernanda Prata Martins, Gustavo Andrade De Paulo, Mônica L C Contini, Angelo Paulo Ferrari
BACKGROUND AND AIMS: biliary anastomotic stricture occurs in 15% to 20% of patients after deceased orthotopic liver transplantation (OLT). Usually treated endoscopically with multiple plastic stents (MPSs), although the use of fully covered self-expandable metal stents (cSEMSs) is emerging. This study aims to compare efficacy and safety of cSEMSs versus MPSs in these patients. METHODS: single center, open-label, randomized clinical trial. Patients were randomized to single cSEMSs for 6 months or to MPS placement, exchanged every 3 months during 1 year...
April 25, 2017: Gastrointestinal Endoscopy
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