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https://www.readbyqxmd.com/read/28916430/therapeutic-approaches-to-cftr-dysfunction-from-discovery-to-drug-development
#1
Hongyu Li, Emanuela Pesce, David N Sheppard, Ashvani K Singh, Nicoletta Pedemonte
Cystic fibrosis (CF) mutations have complex effects on the cystic fibrosis transmembrane conductance regulator (CFTR) protein. They disrupt its processing to and stability at the plasma membrane and function as an ATP-gated Cl(-) channel. Here, we review therapeutic strategies to overcome defective CFTR processing and stability. Because CF mutations have multiple impacts on the assembly of CFTR protein, combination therapy with several pharmacological chaperones is likely to be required to rescue mutant CFTR expression at the plasma membrane...
September 12, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28902616/mef2c-loss-of-function-mutation-associated-with-familial-dilated-cardiomyopathy
#2
Fang Yuan, Zhao-Hui Qiu, Xing-Hua Wang, Yu-Min Sun, Jun Wang, Ruo-Gu Li, Hua Liu, Min Zhang, Hong-Yu Shi, Liang Zhao, Wei-Feng Jiang, Xu Liu, Xing-Biao Qiu, Xin-Kai Qu, Yi-Qing Yang
BACKGROUND: The MADS-box transcription factor myocyte enhancer factor 2C (MEF2C) is required for the cardiac development and postnatal adaptation and in mice-targeted disruption of the MEF2C gene results in dilated cardiomyopathy (DCM). However, in humans, the association of MEF2C variation with DCM remains to be investigated. METHODS: The coding regions and splicing boundaries of the MEF2C gene were sequenced in 172 unrelated patients with idiopathic DCM. The available close relatives of the index patient harboring an identified MEF2C mutation and 300 unrelated, ethnically matched healthy individuals used as controls were genotyped for MEF2C...
September 13, 2017: Clinical Chemistry and Laboratory Medicine: CCLM
https://www.readbyqxmd.com/read/28899449/the-provider-perspective-investigating-the-effect-of-the-electronic-patient-reported-outcome-epro-mobile-application-and-portal-on-primary-care-provider-workflow
#3
Parminder K Hans, Carolyn Steele Gray, Ashlinder Gill, James Tiessen
Aim This qualitative study investigates how the Electronic Patient-Reported Outcome (ePRO) mobile application and portal system, designed to capture patient-reported measures to support self-management, affected primary care provider workflows. BACKGROUND: The Canadian health system is facing an ageing population that is living with chronic disease. Disruptive innovations like mobile health technologies can help to support health system transformation needed to better meet the multifaceted needs of the complex care patient...
September 13, 2017: Primary Health Care Research & Development
https://www.readbyqxmd.com/read/28899063/disruptive-innovation-in-neurovascular-disease
#4
Robert H Rosenwasser, Michael Lang, Stavropoula Tjoumakaris, Pascal Jabbour
No abstract text is available yet for this article.
September 1, 2017: Neurosurgery
https://www.readbyqxmd.com/read/28873011/response-to-blood-meal-in-the-fat-body-of-anopheles-stephensi-using-quantitative-proteomics-toward-new-vector-control-strategies-against-malaria
#5
Manish Kumar, Ajeet Kumar Mohanty, Sreelakshmi K Sreenivasamurthy, Gourav Dey, Jayshree Advani, Sneha M Pinto, Ashwani Kumar, Thottethodi Subrahmanya Keshava Prasad
Malaria remains a grand challenge for disruptive innovation in global health therapeutics and diagnostics. Anopheles stephensi is one of the major vectors of malaria in Asia. Vector and transmission control are key focus areas in the fight against malaria, a field of postgenomics research where proteomics can play a substantive role. Moreover, to identify novel strategies to control the vector population, it is necessary to understand the vector life processes at a global and molecular scale. In this context, fat body is a vital organ required for vitellogenesis, vector immunity, vector physiology, and vector-parasite interaction...
September 5, 2017: Omics: a Journal of Integrative Biology
https://www.readbyqxmd.com/read/28858998/an-integrative-review-of-21st-century-roles-responsibilities-characteristics-and-competencies-of-chief-nurse-executives-a-blueprint-for-the-next-generation
#6
REVIEW
Cecelia L Crawford, Anna Omery, Jerry Spicer
Executive nursing practice is experiencing "head-snapping change." Health care has transitioned from the managed care era to the disruptive innovation era. As chief nurse executives (CNEs) navigate evolving care delivery models, they must consider retooling their roles and responsibilities related to emergent models. This integrative review's purpose was to examine evidence for the roles, responsibilities, characteristics, and competencies of CNEs and system CNEs to better guide future generations of nurse executives...
October 2017: Nursing Administration Quarterly
https://www.readbyqxmd.com/read/28858267/novel-selective-estrogen-receptor-ligand-conjugates-incorporating-endoxifen-combretastatin-and-cyclofenil-combretastatin-hybrid-scaffolds-synthesis-and-biochemical-evaluation
#7
Patrick M Kelly, Niall O Keely, Sandra A Bright, Bassem Yassin, Gloria Ana, Darren Fayne, Daniela M Zisterer, Mary J Meegan
Nuclear receptors such as the estrogen receptors (ERα and ERβ) modulate the effects of the estrogen hormones and are important targets for design of innovative chemotherapeutic agents for diseases such as breast cancer and osteoporosis. Conjugate and bifunctional compounds which incorporate an ER ligand offer a useful method of delivering cytotoxic drugs to tissue sites such as breast cancers which express ERs. A series of novel conjugate molecules incorporating both the ER ligands endoxifen and cyclofenil-endoxifen hybrids covalently linked to the antimitotic and tubulin targeting agent combretastatin A-4 were synthesised and evaluated as ER ligands...
August 31, 2017: Molecules: a Journal of Synthetic Chemistry and Natural Product Chemistry
https://www.readbyqxmd.com/read/28851229/the-california-hiv-aids-research-program-history-impact-and-hiv-cure-initiative
#8
Lisa Loeb Stanga, Laura Packel, Tyler Martz, George Lemp
This Special Issue of AIDS Research and Human Retroviruses features results from the HIV Cure Initiative, funded by the California HIV/AIDS Research Program (CHRP). As a publicly-funded grant maker, CHRP has served for more than three decades as a unique resource for innovative researchers in California whose work seeks to address all aspects of the HIV epidemic and the communities affected by it. Early initiatives at CHRP pioneered what would become enduring cornerstones of HIV science: isolation of the virus; efficacy and toxicities of the first HIV treatments; the emergence of drug resistance; the first biospecimen banks for HIV-related research; the first community-based laboratory service for HIV diagnostic serology; and the first population-based longitudinal cohort study of persons living with HIV - The Gay Men's Health Study...
August 29, 2017: AIDS Research and Human Retroviruses
https://www.readbyqxmd.com/read/28838999/animacroxam-a-novel-dual-mode-compound-targeting-histone-deacetylases-and-cytoskeletal-integrity-of-testicular-germ-cell-cancer-cells
#9
Gustav Steinemann, Alexandra Dittmer, Weronika Kuzyniak, Björn Hoffmann, Mark Schrader, Rainer Schobert, Bernhard Biersack, Bianca Nitzsche, Michael Hoepfner
Novel approaches for the medical treatment of advanced solid tumors including testicular germ cell tumors (TGCT) are desperately needed. Especially, TGCT patients not responding to cisplatin-based therapy need therapeutic alternatives, as there is no effective medical treatment available for this particular subgroup. Here, we studied the suitability of the novel dual mode compound animacroxam for TGCT treatment. Animacroxam consists of a HDAC-inhibitory hydroxamate moiety coupled to a 4,5-diarylimidazole with inherent cytoskeleton disrupting potency...
August 24, 2017: Molecular Cancer Therapeutics
https://www.readbyqxmd.com/read/28826494/small-molecule-inhibition-of-apicomplexan-ftsh1-disrupts-plastid-biogenesis-in-human-pathogens
#10
Katherine Amberg-Johnson, Sanjay B Hari, Suresh M Ganesan, Hernan A Lorenzi, Robert T Sauer, Jacquin C Niles, Ellen Yeh
The malaria parasite Plasmodium falciparum and related apicomplexan pathogens contain an essential plastid organelle, the apicoplast, which is a key anti-parasitic target. Derived from secondary endosymbiosis, the apicoplast depends on novel, but largely cryptic, mechanisms for protein/lipid import and organelle inheritance during parasite replication. These critical biogenesis pathways present untapped opportunities to discover new parasite-specific drug targets. We used an innovative screen to identify actinonin as having a novel mechanism-of-action inhibiting apicoplast biogenesis...
August 18, 2017: ELife
https://www.readbyqxmd.com/read/28815609/the-academic-practice-gap-strategies-for-an-enduring-problem
#11
C L Huston, B Phillips, P Jeffries, C Todero, J Rich, P Knecht, S Sommer, M P Lewis
The academic-practice gap in nursing is well documented. Academe is criticized for producing nurses insufficiently prepared to fully participate in patient care. Practice settings are criticized for having unrealistic expectations of new graduates. This article is based on a review of the literature and an exploration of contemporary practices used to bridge academic and practice partnerships. Differences in outcome expectations for new graduates between academe and practice are outlined and consequences of the gap for new graduates, patients, and employers are discussed...
August 16, 2017: Nursing Forum
https://www.readbyqxmd.com/read/28796569/cost-effectiveness-of-kinase-inhibitors-for-hematologic-malignancies-a-systematic-and-critical-review
#12
Monia Marchetti
Several genetic disruptions lead to constitutive activation of those kinases leukemic cells depend on for survival and proliferation. Kinase inhibitors (KI) are major therapeutic innovations for chronic myeloid leukemia (CML), chronic lymphoid leukemia (CLL) and myelofibrosis (MF) providing a relevant improvement of quality-adjusted survival in patients with high-risk or refractory disease. CML patients are being treated with first-generation KI imatinib since many years, achieving expected survivals longer than 10 years...
August 21, 2017: Expert Review of Pharmacoeconomics & Outcomes Research
https://www.readbyqxmd.com/read/28795697/perovskite-based-photodetectors-materials-and-devices
#13
REVIEW
Huan Wang, Dong Ha Kim
While the field of perovskite-based optoelectronics has mostly been dominated by photovoltaics, light-emitting diodes, and transistors, semiconducting properties peculiar to perovskites make them interesting candidates for innovative and disruptive applications in light signal detection. Perovskites combine effective light absorption in the broadband range with good photo-generation yield and high charge carrier mobility, a combination that provides promising potential for exploiting sensitive and fast photodetectors that are targeted for image sensing, optical communication, environmental monitoring or chemical/biological detection...
August 10, 2017: Chemical Society Reviews
https://www.readbyqxmd.com/read/28780563/implementing-a-multi-modal-support-service-model-for-the-family-caregivers-of-persons-with-age-related-macular-degeneration-a-study-protocol-for-a-randomised-controlled-trial
#14
Bamini Gopinath, Ashley Craig, Annette Kifley, Gerald Liew, Jaye Bloffwitch, Kim Van Vu, Nichole Joachim, Rob Cummins, Julie Heraghty, Timothy Broady, Alison Hayes, Paul Mitchell
INTRODUCTION: Age-related macular degeneration (AMD) is a leading cause of blindness and low vision among older adults. Previous research shows a high prevalence of distress and disruption to the lifestyle of family caregivers of persons with late AMD. This supports existing evidence that caregivers are 'hidden patients' at risk of poor health outcomes. There is ample scope for improving the support available to caregivers, and further research should be undertaken into developing services that are tailored to the requirements of family caregivers of persons with AMD...
August 4, 2017: BMJ Open
https://www.readbyqxmd.com/read/28778033/induction-of-virus-resistance-by-exogenous-application-of-double-stranded-rna
#15
REVIEW
Neena Mitter, Elizabeth A Worrall, Karl E Robinson, Zhi Ping Xu, Bernard J Carroll
Exogenous application of double-stranded RNA (dsRNA) for virus resistance in plants represents a very attractive alternative to virus resistant transgenic crops or pesticides targeting virus vectors. However, the instability of dsRNA sprayed onto plants is a major challenge as spraying naked dsRNA onto plants provides protection against homologous viruses for only 5 days. Innovative approaches, such as the use of nanoparticles as carriers of dsRNA for improved stability and sustained release, are emerging as key disruptive technologies...
August 1, 2017: Current Opinion in Virology
https://www.readbyqxmd.com/read/28777969/method-validation-and-nanoparticle-characterization-assays-for-an-innovative-amphothericin-b-formulation-to-reach-increased-stability-and-safety-in-infectious-diseases
#16
Maraine Catarina Tadini, Ana Maria de Freitas Pinheiro, Daniel Blascke Carrão, Ana Luiza Scarano Aguillera Forte, Sofia Nikolaou, Anderson R M de Oliveira, Andresa Aparecida Berretta, Franciane Marquele-Oliveira
Drug Delivery Systems (DDS) of known drugs are prominent candidates towards new and more-effective treatments of various infectious diseases as they may increase drug bioavailability, control drug delivery and target the site of action. In this sense, the encapsulation of Amphotericin B (AmB) in Nanostructured Lipid Carriers (NLCs) designed with pH-sensible phospholipids to target infectious tissues was proposed and suitable analytical methods were validated, as well as, proper nanoparticle characterization were conducted...
October 25, 2017: Journal of Pharmaceutical and Biomedical Analysis
https://www.readbyqxmd.com/read/28765447/what-should-oral-health-professionals-know-in-2040-executive-summary
#17
Jane A Weintraub
The "Advancing Dental Education in the 21(st) Century" project assesses current status and trends to prepare for the future. Section 3 of the project asks what knowledge and skills should dental and advanced dental education learners have to provide dental care in 2040 and how should educators be preparing them. This executive summary of five background articles in this section focuses on predoctoral education, advanced dental education, the provision of medical services within dental practice, the incorporation of oral health services into primary care and medical practice, and interprofessional education and practice...
August 2017: Journal of Dental Education
https://www.readbyqxmd.com/read/28756433/point-of-care-testing-across-rural-and-remote-emergency-departments-in-australia-staff-perceptions-of-operational-impact
#18
Maria R Dahm, Euan McCaughey, Ling Li, Johanna Westbrook, Virginia Mumford, Juliana Iles-Mann, Andrew Sargeant, Andrew Georgiou
New South Wales (NSW) Health Pathology is implementing one of the world's largest managed PoCT services across rural and remote Emergency Departments (EDs) in New South Wales, Australia to improve patient access to care. The aim of this qualitative study was to gain a context-rich understanding of the operational impact of the NSW rollout of PoCT across rural and remote ED settings as experienced by frontline clinical staff. Clinical professionals (n=14) participated in interviews and focus groups in August 2015 at four rural and remote NSW EDs...
2017: Studies in Health Technology and Informatics
https://www.readbyqxmd.com/read/28749952/an-innovative-way-of-thinking-nuclear-waste-management-neutron-physics-of-a-reactor-directly-operating-on-snf
#19
Bruno Merk, Dzianis Litskevich, Mark Bankhead, Richard J Taylor
A solution for the nuclear waste problem is the key challenge for an extensive use of nuclear reactors as a major carbon free, sustainable, and applied highly reliable energy source. Partitioning and Transmutation (P&T) promises a solution for improved waste management. Current strategies rely on systems designed in the 60's for the massive production of plutonium. We propose an innovative strategic development plan based on invention and innovation described with the concept of developments in s-curves identifying the current boundary conditions, and the evolvable objectives...
2017: PloS One
https://www.readbyqxmd.com/read/28747537/john-sculley-s-noble-cause-disruptive-innovation-to-transform-healthcare
#20
EDITORIAL
Govind Rao
No abstract text is available yet for this article.
July 2017: PDA Journal of Pharmaceutical Science and Technology
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